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Health Risks from Dioxin and Related Compounds:
Evaluation of the EPA Reassessment
Committee on EPA's Exposure and Human Health
Reassessment of TCDD and Related Compounds,
National Research Council
ISBN: 0-309-66273-7, 268 pages, 6 x 9, (2006)
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THE NATIONAL ACADEMIES
Advisers 1o the Nation on Science, Engineering, and Medicine
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
Health Risks from
Dioxin and
Related Compounds
Evaluation o f the EPA Reassessment
Committee on EPA's Exposure and Human Health
Reassessment of TCDD and Related Compounds
Board on Environmental Studies and Toxicology
Division on Earth and Life Studies
N A T IO N A L RESEARCH C O U N C IL
OF THE NATIONAL ACADEMIES
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THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
The National Academy of Sciences is a private, nonprofit, self-perpetuating society
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Copyright © National Academy of Sciences. All rights reserved.
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Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
COMMITTEE ON EPA’S EXPOSURE AND HUMAN HEALTH
REASSESSMENT OF TCDD AND RELATED COMPOUNDS
Members
David L. Eaton (Chair), University of Washington, Seattle
Dennis M. Bier, Baylor College of Medicine, Houston, TX
Joshua T. Cohen, Tufts New England Medical Center, Boston, MA
Michael S. Denison, University of California, Davis
Richard T. Di Giulio, Duke University, Durham, NC
Norbert E. Kaminski, Michigan State University, East Lansing
Nancy K. Kim, New York State Department of Health, Troy
Antoine Keng Djien Liem, European Food Safety Authority, Parma, Italy
Thomas E. McKone, Lawrence Berkeley National Laboratory and School
of Public Health, University of California, Berkeley
Malcolm C. Pike, University of Southern California, Los Angeles
Alvaro Puga, University of Cincinnati Medical Center, Cincinnati, OH
Andrew G. Renwick, University of Southampton (emeritus),
Southampton, UK
David A. Savitz, Mount Sinai School of Medicine, New York, NY
Allen E. Silverstone, SUNY-Upstate Medical University, Syracuse, NY
Paul F. Terranova, University of Kansas Medical Center, Kansas City
Kimberly M. Thompson, Massachusetts Institute of Technology,
Cambridge
Gary M. Williams, New York Medical College, Valhalla
Yiliang Zhu, University of South Florida, Tampa
Staff
Suzanne van Drunick, Project Director
Kulbir Bakshi, Senior Program Officer for Toxicology
Ruth Crossgrove, Senior Editor
Jean Hampton, Senior Fellow
Cay Butler, Editor
Mirsada Karalic-Loncarevic, Research Associate
Bryan P. Shipley, Research Associate
Liza R. Hamilton, Senior Program Assistant
Alexandra Stupple, Senior Editorial Assistant
Sammy Bardley, Librarian
Sponsors
U.S. Environmental Protection Agency
U.S. Department of Agriculture
U.S. Department of Health and Human Services
v
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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BOARD ON ENVIRONMENTAL STUDIES AND TOXICOLOGY
Members
Jonathan M. Samet (Chair), Johns Hopkins University, Baltimore, MD
Ramón Alvarez, Environmental Defense, Austin, TX
John M. Balbus, Environmental Defense, Washington, DC
Thomas Burke, Johns Hopkins University, Baltimore, MD
Dallas Burtraw, Resources for the Future, Washington, DC
James S. Bus, Dow Chemical Company, Midland, MI
Costel D. Denson, University of Delaware, Newark
E. Donald Elliott, Willkie Farr & Gallagher LLP, Washington, DC
J. Paul Gilman, Oak Ridge National Laboratory, Oak Ridge, TN
Sherri W. Goodman, Center for Naval Analyses, Alexandria, VA
Judith A. Graham, American Chemistry Council, Arlington, VA
Daniel S. Greenbaum, Health Effects Institute, Cambridge, MA
William P. Horn, Birch, Horton, Bittner and Cherot, Washington, DC
Robert Huggett, Michigan State University (emeritus), East Lansing
James H. Johnson Jr., Howard University, Washington, DC
Judith L. Meyer, University of Georgia, Athens
Patrick Y. O’Brien, ChevronTexaco Energy Technology Company,
Richmond, CA
Dorothy E. Patton, International Life Sciences Institute, Washington, DC
Steward T.A. Pickett, Institute of Ecosystem Studies, Millbrook, NY
Danny D. Reible, University of Texas, Austin
Joseph V. Rodricks, ENVIRON International Corporation, Arlington, VA
Armistead G. Russell, Georgia Institute of Technology, Atlanta
Robert F. Sawyer, University of California, Berkeley
Lisa Speer, Natural Resources Defense Council, New York, NY
Kimberly M. Thompson, Massachusetts Institute of Technology,
Cambridge
Monica G. Turner, University of Wisconsin, Madison
Mark J. Utell, University of Rochester Medical Center, Rochester, NY
Chris G. Whipple, ENVIRON International Corporation, Emeryville, CA
Lauren Zeise, California Environmental Protection Agency, Oakland
Senior Staff
James J. Reisa, Director
David J. Policansky, Scholar
Raymond A. Wassel, Senior Program Officer for Environmental Sciences
and Engineering
Kulbir Bakshi, Senior Program Officer for Toxicology
Eileen N. Abt, Senior Program Officer for Risk Analysis
Vi
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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Karl E. Gustavson, Senior Program Officer
K. John Holmes, Senior Program Officer
Ellen K. Mantus, Senior Program Officer
Susan N.J. Martel, Senior Program Officer
Suzanne van Drunick, Senior Program Officer
Ruth E. Crossgrove, Senior Editor
vu
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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OTHER REPORTS OF THE BOARD ON
ENVIRONMENTAL STUDIES AND TOXICOLOGY
Assessing the Human Health Risks of Trichloroethylene: Key Scientific
Issues (2006)
New Source Review for Stationary Sources of Air Pollution (2006)
Human Biomonitoring for Environmental Chemicals (2006)
Fluoride in Drinking Water: A Scientific Review of EPA’s Standards (2006)
State and Federal Standards for Mobile-Source Emissions (2006)
Superfund and Mining Megasites—Lessons from the Coeur d’Alene River
Basin (2005)
Health Implications of Perchlorate Ingestion (2005)
Air Quality Management in the United States (2004)
Endangered and Threatened Species of the Platte River (2004)
Atlantic Salmon in Maine (2004)
Endangered and Threatened Fishes in the Klamath River Basin (2004)
Cumulative Environmental Effects of Alaska North Slope Oil and Gas
Development (2003)
Estimating the Public Health Benefits of Proposed Air Pollution
Regulations (2002)
Biosolids Applied to Land: Advancing Standards and Practices (2002)
The Airliner Cabin Environment and Health of Passengers and Crew
(2002)
Arsenic in Drinking Water: 2001 Update (2001)
Evaluating Vehicle Emissions Inspection and Maintenance Programs
(2001)
Compensating for Wetland Losses Under the Clean Water Act (2001)
A Risk-Management Strategy for PCB-Contaminated Sediments (2001)
Acute Exposure Guideline Levels for Selected Airborne Chemicals (4
volumes, 2000-2004)
Toxicological Effects of Methylmercury (2000)
Strengthening Science at the U.S. Environmental Protection Agency (2000)
Scientific Frontiers in Developmental Toxicology and Risk Assessment
(2000)
Ecological Indicators for the Nation (2000)
Waste Incineration and Public Health (1999)
Hormonally Active Agents in the Environment (1999)
Research Priorities for Airborne Particulate Matter (4 volumes, 1998
2004)
The National Research Council’s Committee on Toxicology: The First 50
Years (1997)
Carcinogens and Anticarcinogens in the Human Diet (1996)
Upstream: Salmon and Society in the Pacific Northwest (1996)
vm
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
Science and the Endangered Species Act (1995)
Wetlands: Characteristics and Boundaries (1995)
Biologic Markers (5 volumes, 1989-1995)
Review of EPA’s Environmental Monitoring and Assessment Program (3
volumes, 1994-1995)
Science and Judgment in Risk Assessment (1994)
Pesticides in the Diets of Infants and Children (1993)
Dolphins and the Tuna Industry (1992)
Science and the National Parks (1992)
Human Exposure Assessment for Airborne Pollutants (1991)
Rethinking the Ozone Problem in Urban and Regional Air Pollution
(1991)
Decline of the Sea Turtles (1990)
Copies of these reports may be ordered from the National Academies Press
(800) 624-6242 or (202) 334-3313
www.nap.edu
tx
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
A ck n o w led g m en ts
We are appreciative of the generous support provided by the U.S. Envi
ronmental Protection Agency and are especially grateful for the outstanding
assistance provided by Dr. William Farland. We are also grateful to Lisa
Matthews, EPA’s program manager, and for Dr. Richard Canady’s assistance
in facilitating invited speakers from the federal agencies.
Many people assisted the committee and National Research Council
staff in creating this report. We are grateful for the information and support
provided by the following:
Lesa L. Aylward, Summit Toxicology, L.L.P.
P. Michael Bolger, U.S. Food and Drug Administration
Gail Charnley, HealthRisk Strategies (on behalf of the Food Industry
Dioxin Working Group)
Richard W. Clapp, Boston University School of Public Health
Edmund A. C. Crouch, Cambridge Environmental Inc.
Christopher T. De Rosa, Agency for Toxic Substances and Disease Registry
Michael J. DeVito, U.S. Environmental Protection Agency
David W. Gaylor, Gaylor and Associates, LLC
David P. Goldman, U.S. Department of Agriculture
C.T. ‘Kip’ Howlett, Consultant
Russell E. Keenan, AMEC Earth & Environmental Inc.
Larry L. Needham, Centers for Disease Control and Prevention
Christopher J. Portier, National Institute of Environmental Health Sciences
Susan Schober, Centers for Disease Control and Prevention
Jay B. Silkworth, General Electric Company
Nigel Walker, National Institute of Environmental Health Sciences
The committee’s work also benefited from written and oral testimony
submitted by the public, whose participation is much appreciated.
xi
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
A ck n o w led g m en t o f R eview P articip an ts
This report has been reviewed in draft form by individuals chosen for
their diverse perspectives and technical expertise, in accordance with proce
dures approved by the National Research Council’s Report Review Com
mittee. The purpose of this independent review is to provide candid and
critical comments that will assist the institution in making its published re
port as sound as possible and to ensure that the report meets institutional
standards for objectivity, evidence, and responsiveness to the study charge.
The review comments and draft manuscript remain confidential to protect
the integrity of the deliberative process. We wish to thank the following
individuals for their review of this report:
Melvin Andersen, CIIT Centers for Health Research
John Doull, University of Kansas Medical Center
David Gaylor, Gaylor & Associates
Michael Holsapple, ILSI Health and Environmental Sciences
Daniel Krewski, University of Ottawa
Philip Landrigan, Mount Sinai School of Medicine
John A. Moore, Hollyhouse, Inc.
Stephen S. Olin, ILSI Research Foundation/Risk Science
Richard Peterson, School of Pharmacy, Harvard School of Public Health
Louise Ryan, Harvard School of Public Health
Steven Safe, Texas A&M University
Glenn Sipes, University of Arizona
Martin Van den Berg, Utrecht University
xm
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Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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x iv
ACKNOWLEDGMENTS
Noel Weiss, University of Washington
Lauren Zeise, California Environmental Protection Agency
Although the reviewers listed above have provided many constructive
comments and suggestions, they were not asked to endorse the conclusions
or recommendations, nor did they see the final draft of the report before its
release. The review of this report was overseen by William Halperin and
John Bailar. Appointed by the National Research Council, they were re
sponsible for making certain that an independent examination of this re
port was carried out in accordance with institutional procedures and that
all review comments were carefully considered. Responsibility for the final
content of this report rests entirely with the authoring committee and the
institution.
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
P reface
2,3,7,8-Tetrachlorodibenzo-p-dioxin (TCDD), also called dioxin, is
among the most toxic anthropogenic substance ever identified. TCDD and a
number of similar polychlorinated dioxins, dibenzofurans, and coplanar
polychlorinated biphenyls (dioxin-like compounds [DLCs]) have been the
subject of intense scientific research and frequently controversial environ
mental and health policies. Animal studies have demonstrated potent effects
of TCDD, other dioxins, and many DLCs on tumor development, birth
defects, reproductive abnormalities, immune dysfunction, dermatological
disorders, and a plethora of other adverse effects. Because of their persis
tence in the environment and their bioaccumulative potential, TCDD, other
dioxins, and DLCs are now ubiquitous environmental pollutants and are
detected at low concentrations in virtually all organisms at higher trophic
levels in the food chain, including humans. Inadvertent exposures of hu
mans through industrial accidents, occupational exposures to commercial
compounds (primarily phenoxyacid herbicides), and through dietary path
ways have led to a wide range of body burdens of TCDD, other dioxins, and
DLCs, and numerous epidemiological studies have attempted to relate ex
posures to a variety of adverse effects in humans.
Because of substantial policy and economic implications associated with
the regulation of TCDD, other dioxins, and DLCs in the environment, the
U.S. Environmental Protection Agency (EPA) began in the mid-1980s to
invest enormous efforts in risk assessment of these compounds. Many scien
tists in the dioxin research community participated in writing numerous
review chapters on various aspects of dioxin toxicology, chemistry, and en
vironmental fate. In September 1992, initial drafts of all background chapxv
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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xvi
PREFACE
ters of the EPA assessment underwent extensive peer review, followed by
extensive revision and additional review of some chapters. In September
1994, all the chapters, plus the first draft of a summary “risk characteriza
tion” chapter, were subjected to more peer review and public comment. In
1997 and 1998, additional modifications of the compiled information led to
the development of an “Integrated Summary and Risk Characterization”
document. This document, as well as additional information on toxic equiva
lency of DLCs, was revised and subsequently reviewed by EPA’s Science
Advisory Board (SAB) in November 2000. Recognizing the broad policy
implications of the dioxin reassessment, an Interagency Working Group
(IWG), consisting of representatives of seven federal agencies, was estab
lished in 2000 to foster information sharing, develop a common language
for dioxin science and science policy across governmental agencies and pro
grams, identify gaps and needs in dioxin risk assessment, and coordinate
risk management strategies. The IWG has provided input to EPA on the
draft dioxin reassessment and has been coordinating risk management is
sues on TCDD and other dioxins for the federal government since its incep
tion. After further revisions in response to SAB and other public comments,
in December 2003, EPA released a preliminary draft document titled Expo
sure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Compounds, referred to in this report as the
Reassessment.
In the summer of 2004, EPA requested the National Research Council
(NRC) to create “ an expert committee to review EPA’s draft reassessment of
the risks of dioxin and dioxin-like compounds.” In response, the NRC ap
pointed the Committee on EPA’s Exposure and Human Health Reassess
ment of TCDD and Related Compounds, which was charged, to the extent
possible, to review “EPA’s modeling assumptions, including those associated
with dose-response curve and points-of-departure dose ranges and associ
ated likelihood estimates for identified human health outcomes; EPA’s quan
titative uncertainty analysis; EPA’s selection of studies as a basis for its as
sessments and gaps in scientific knowledge.” The charge also requested that
the committee address two specific points of controversy: (1) the scientific
evidence for classifying dioxin as a human carcinogen, and (2) the validity
of the nonthreshold linear dose-response model and the cancer slope factor
calculated by EPA through the use of this model. The committee was also
asked to comment on the usefulness of toxic equivalency factors (TEFs) and
the uncertainties associated with their use in risk assessment of complex
mixtures. Finally, the committee was also asked to review the uncertainty
associated with the Reassessment’s approach to the analysis of food sam
pling and human dietary intake data.
The entire Reassessment consists of three parts totaling more than 1,800
pages of scientific review. Part I contains several volumes of a previous sci-
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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PREFACE
x v ii
entific review of information relating to sources and exposures to TCDD
and other dioxins in the environment, and Part II contains detailed reviews
of scientific information on the health effects of TCDD, other dioxins, and
DLCs. The information in Parts I and II were provided to the committee as
background, with the recognition that many chapters in these two volumes
have not been updated for several years. The committee was asked to focus
its review on Part III of the Reassessment, which represents an “integrated
summary and risk characterization for TCDD and related compounds.”
The committee held five meetings between November 22, 2004, and July
7, 2005. The first three meetings provided opportunity for public input. The
committee heard from scientists from the IWG, EPA, Food and Drug Admin
istration, Department of Agriculture, Agency for Toxic Substance and Disease
Registry, National Center for Health Statistics, and National Toxicology Pro
gram and from representatives from academia, environmental organizations,
and the regulated community. The committee was provided with written testi
mony and new scientific papers that have appeared since 2003 (and thus were
not available for consideration by EPA in the Reassessment).
It is important to recognize what the committee did not consider to be part
of its charge. Although the committee made every effort to consider critical new
studies that have appeared since the last revision of Part III of the Reassessment,
it did not conduct an exhaustive and detailed review of all scientific information
published on TCDD and other dioxins since 2003, and any information that
became available to the public after the date of the committee’s last meeting
(July 7, 2005) was not considered. The committee did not attempt to “redo” the
risk assessment—rather, it tried to provide constructive comments in areas in
which the scientific approaches or justifications were thought to need improve
ment, the expectation being that EPA might need to reconsider and revise its
approaches and documentation accordingly.
The final recommendations of the committee are offered to EPA with
the recognition and appreciation of the enormous amount of time and effort
that has been committed to the execution of this Reassessment for nearly 14
years. Although many of the comments are, not surprisingly, critical of cer
tain aspects or approaches taken by EPA, the committee was impressed over
all with the tremendous dedication and hard work that has gone into the
creation of the Reassessment. The committee hopes the report will be of
value in assisting EPA to make final changes to Part III that will allow the
timely release of a scientifically defensible document. The committee further
hopes that this review will help to guide all federal agencies in making ratio
nal and defensible health and environmental policies that adequately protect
human health and the environment from the adverse effects of TCDD, other
dioxins, and DLCs in the environment.
The Committee on EPA’s Exposure and Human Health Reassessment of
TCDD and Related Compounds was aided immensely by a number of in-
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
x v iii
PREFACE
dividuals. The committee, and especially the chair, would like to thank the
NRC study director Suzanne van Drunick for her tireless effort and good
humor in directing this project under substantial time constraints. We also
appreciate the organizational skills of Liza Hamilton for ensuring that our
meetings and travel arrangements went smoothly, and other NRC staff, in
cluding Bryan Shipley for his technical assistance, Ruth Crossgrove and Cay
Butler for their editorial assistance, Mirsada Karalic-Loncarevic for her refer
ence assistance, and Alexandra Stupple for her production assistance. The
committee is also grateful to Kulbir Bakshi, senior program officer; James
Reisa, director of the Board on Environmental Studies and Toxicology; and
Thomas Burke, professor and associate chair, Johns Hopkins University, for
their oversight of the study; and to Ann Yaktine, Food and Nutrition Board,
Institute of Medicine, for her contribution. I would like to thank all the com
mittee members for their hard work and their dedication to ensuring that the
report stands up to the basic charge that we “ ensure that the risk estimates ...
are scientifically robust.” I, the NRC staff, and the committee are indebted to
a number of individuals who presented background information, both orally
and in writing, that made the committee’s understanding of the issues more
complete. Thanks especially to Richard Canady, IWG on dioxin, for his assis
tance in helping to locate speakers and important background documents and
to William Farland for his outstanding assistance.
David L. Eaton, Chair
Committee on EPA’s Exposure and Human Health
Reassessment of TCDD and Related Compounds
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
A b b rev iatio n s
2-AAF: 2-acetylaminofluorene
AHF: altered hepatocelluar foci
AHR: aromatic hydrocarbon receptor
Ahr-/-: AHR null
AIC: Akaike’s information criterion
Anti-SRBC: anti-sheep red blood cell
ARNT: AHR nuclear translocator protein
ATSDR: Agency for Toxic Substances and Disease Registry
AUC: area under the curve
BMD: benchmark dose
BMDL: benchmark dose low
BMR: benchmark response
CB: chlorobiphenyl
CI: confidence intervals
CL: volume of blood cleared per unit time
CLB: cumulative lipid burden
COX: cyclooxygenase
COX-2: cyclooxygenase-2
CSF: cancer slope factor
CYP1A: cytochrome P450A1 protein
CYP1A1: cytochrome P4501A1 protein
CYP1A2: cytochrome P4501A2 protein
CYP1B1: cytochrome P4501B1 protein
DHHS: U.S. Department of Health and Human Services
DIM: diindolymethane
-V*
*A ¿*A
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Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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XX
ABBREVIATIONS
DLCs: dioxin-like compounds
DOD: U.S Department of Defense
DF: dioxins and furons
DFP: dioxins, furons, and PCBs
ED: effective dose
EGFR: epidermal growth factor receptor
EPA: U.S. Environmental Protection Agency
ER: estrogen receptor
FAO: Food and Agriculture Organization of the United Nations
FDA: U.S. Food and Drug Administration
FSH: follicle-stimulating hormone
GGT: y-glutamyl transpeptidase
GnRH: gonadotropin-releasing hormone
HAH: halogenated aromatic hydrocarbon
hCG: human chorionic gonadotropin
HpCDD: heptachlorodibenzo-p-dioxin
HepCB: heptachlorobiphenyl
HxCDD: hexachlorodibenzo-p-dioxin
HxCDF: hexachlorodibenzofuran
I3C: indole-3-carbinol
IARC: International Agency for Research on Cancer
ICZ: indolo-[3,2b]-carbazole
IOM: Institute of Medicine
IPCS: International Program of Chemical Safety
IWG: Interagency Working Group
JECFA: Joint Expert Committee on Food Additives
LABB: lifetime average body burden
LD: lethal dose
LED: lowest effective dose
LH: lutenizing hormone
LOAEL: lowest-observed-adverse-effect level
LOD: limit of detection
6-MCDF: 6-methyl-1,3,8-trichlorodibenzofuran
MOE: margin of exposure
mRNA: messenger ribonucleic acid
NAS: National Academy of Sciences
NCEA: National Center for Environmental Assessment
NIEHS: National Institute of Environmental Health Sciences
NIH: National Institutes of Health
NIOSH: National Institute for Occupational Safety and Health
NOAEL: no-observed-adverse-effect level
NOEL: no-observed-effect level
NRC: National Research Council
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Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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ABBREVIATIONS
NTP: National Toxicology Program
OCDF: octachlorodibenzofuran
OCDD: octachlorodibenzo-p-dioxin
PA: plasminogen activator
PAH: polycyclic aromatic hydrocarbon
PAI-1: plasminogen activator inhibitor-1
PBDD: polybrominated dibenzo-p-dioxin
PBDF: polybrominated dibenzofuran
PBPK: physiologically based pharmacokinetics
PCB: polychlorinated biphenyl
PCDD: polychlorinated dibenzo-p-dioxin
PCDF: polychlorinated dibenzofuran
PeCB: pentachlorobiphenyl
PeCDD: pentachlorodibenzo-p-dioxin
PeCDF: pentachlorodibenzofuran
PK: pharmacokinetics
POD: point of departure
PPAR: peroxisome proliferator activated receptor
ppt: parts per trillion
PR: progesterone receptor
QF: quality of fit
REP: relative potency
RfD: reference dose
RR: rate ratio
SAB: Science Advisory Board
SCF: Scientific Committee on Food
SD: standard deviation
SE: standard error
SMR: standardized mortality (morbidity) ratio
T3: triiodothyronine
T4: thyroxine
TCB: 2,2',5,5'-tetrachlorobiphenyl
TCDD: 2,3,7,8-tetrachlorodibenzo-p-dioxin
TCDF: 2,3,7,8-tetrachlorodibenzo furon
TEF: toxic equivalency factor
TEQ: toxic equivalent quotient
tPA: tissue plasminogen activator
2,4,5-T: 2,4,5-trichlorophenoxyacetic acid
TSH: thyroid-stimulating hormone
UED: upper effective dose
USDA: U.S. Department of Agriculture
WHO: World Health Organization
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C o n ten ts
PUBLIC SUMMARY
1
SUMMARY
11
1
INTRODUCTION
TCDD, Other Dioxins, and DLCs, 30
Toxic Equivalency Factors, 33
Exposure Characterization, 34
Health Effects, 38
Committee Charge and Response, 39
28
2
GENERAL CONSIDERATIONS OF UNCERTAINTY AND
VARIABILITY, SELECTION OF DOSE METRIC, AND
DOSE-RESPONSE MODELING
Hazard Classification, 47
Exposure Assessment, 48
Assessment of Other Dioxins and DLCs, 50
General Issues Related to Variability and Uncertainty
Associated with Selection of Dose Metric and Dose-Response
Modeling, 51
General Issues Related to Risk Characterization, 55
Selection of Dose Metric, 57
Dose-Response Modeling, 63
Conclusions and Recommendations, 73
xxm
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x x iv
CONTENTS
3
TOXIC EQUIVALENCY FACTORS
Dioxin-like Compounds, 75
Major Issues, Assumptions, and Uncertainties, 76
Key Studies and Publications to Be Included, 85
Conclusions and Recommendations, 86
75
4
EXPOSURE ASSESSMENT
Assessment Procedures, 90
Overview and Commentary on EPA’s Exposure
Characterization, 91
Committee Findings, 99
Conclusions and Recommendations, 105
90
5
CANCER
Qualitative Evaluation of Carcinogenicity, 108
Quantitative Considerations in Assessing TCDD, Other Dioxins,
and DLC Carcinogenicity, 121
Conclusions and Recommendations, 140
108
6
NONCANCER END POINTS
Immune Function, 144
Conclusions and Recommendations on the Immunotoxicity
of TCDD, Other Dioxins, and DLCs, 153
Reproduction and Development, 154
Other Noncancer End Points, 169
Conclusions and Recommendations on the Reproductive,
Developmental, and Other Noncancer End Points of
TCDD, Other Dioxins, and DLCs, 173
144
7
REVIEW OF RISK CHARACTERIZATION
Review, 175
Conclusions and Recommendations, 186
175
8
CONCLUSIONS AND RECOMMENDATIONS
188
Classification of TCDD as Carcinogenic to Humans, 188
Use of Low-Dose Linear Versus Threshold (Nonlinear)
Extrapolation Models for Quantitative Cancer Risk
Estimations, 190
Use of the 1% Response Level As a Point of Departure for
Low-Dose Risk Estimation, 190
Characterization of Uncertainty for Risk Estimates, 192
Use of Toxic Equivalency Factors for Risk Estimation of DLCs
and Mixture of DLCs, 193
Use of Body Burden As the Primary Dose Metric for Cross-Species
Extrapolation, 193
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CONTENTS
xxv
EPA’s Exposure Assessment for TCDD, Other Dioxins,
and DLCs in the United States, 194
EPA’s Evaluation of Immunotoxicity of TCDD, Other Dioxins,
and DLCs, 194
EPA’s Evaluation of Reproductive and Developmental Toxicity
of TCDD, Other Dioxins, and DLCs, 195
EPA’s Evaluation of Other Toxic End Points, 195
EPA’s Overall Approach to Risk Characterization, 196
REFERENCES
APPENDIXES
A Biographical Information on Committee Members, 227
B EPA’s 2005 Guidelines for Carcinogen Risk Assessment, 236
FIGURES
S-1 Conceptual illustration of the effect of the selection of
the point of departure and of the mathematical model
used to extrapolate below the point of departure on the risk
estimate, 5, 15
1-1 Benzene ring (a) with conjugated bonds and (b) with inner
ring depicting conjugated bonds, 31
1-2 Double benzene ring structures of (a) dioxins and
(b) furans, 31
1-3 Biphenyl ring structure of PCBs, 31
1- 4 Examples of toxic PCDDs, PCDFs, and PCBs of interest
in the Reassessment, 32
2- 1 Vmax, 69
5-1 Possible mechanism for TCDD hepatocarcinogenicity, 118
5-2 Range of plausible CSF values: Consideration of
parameter confidence intervals only, 140
TABLES
1-1 TEFs for Humans and Nonhuman Mammals, 35
1- 2 Summary of North American PCDD, PCDF, and PCB
TEQ WHO Concentrations in Environmental Media
and Food, 40
2- 1 Categories of Key Decisions EPA Faced in Characterizing
Cancer Risk, 52
2-2 Categories of Key Decisions EPA Faced in Characterizing
Noncancer Risk, 53
2-3 Components of a Systematic Review, 57
5-1 Dioxin Cancer Bioassays, 114
5-2 TCDD, Other Dioxins, and DLC Cancer Bioassays, 116
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xxvi
5-3
5-4
5-5
5-6
CONTENTS
Dioxin Rat Bioassays, 119
Hepatic Toxicity in TCDD Rat Bioassays, 126
EPA Inputs to CSF Estimates Using Epidemiological Data, 133
ED01, LED01, and UED01 Values, 135
BOXES
S-1 Statement of Task, 13
1-1 Statement of Task, 43
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Health Risks from
Dioxin and
Related Compounds
Evaluation o f the EPA Reassessment
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P ublic Su m m ary
HEALTH RISKS FROM TCDD, OTHER DIOXINS,
AND DIOXIN-LIKE COMPOUNDS
Evaluation of the EPA Reassessment
Dioxins and dioxin-like compounds (DLCs) are released into the en
vironment from several sources, including combustion, metal processing,
and chemical manufacturing and processing. The most toxic of these
compounds is TCDD, often simply called dioxin. Many other types of
dioxins, other than TCDD, and DLCs share most, if not all, of the toxic
characteristics of TCDD. In the past, occupational exposures to TCDD,
other dioxins, and DLCs occurred in a variety of industries, especially
those involved in the manufacture of trichlorophenol (used to make cer
tain herbicides) and PCBs. (PCBs contain some forms that are dioxin-like
and, when heated to high temperatures, may also be contaminated with
dibenzofurans, which are also dioxin-like.) Much of the knowledge about
the health effects of TCDD, other dioxins, and DLCs in humans comes
from studies of relatively highly exposed workplace populations. Wide
spread use of certain herbicides containing TCDD, other dioxins, and
DLCs, as well as some types of industrial emissions, resulted in local and
global contamination of air, soil, and water with trace levels of these
compounds. These trace levels built up in the food chain because TCDD,
other dioxins, and DLCs do not readily degrade. Instead, they persist in
the environment and accumulate in the tissues of animals. The general
1
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
public is exposed to TCDD, other dioxins, and DLCs primarily by eating
such foods as beef, dairy products, pork, fish, and shellfish.
The health effects of exposures to relatively high levels of dioxin be
came widely publicized due to the use of the herbicide called Agent Orange
in the Vietnam War. Agent Orange contained small amounts of TCDD as a
contaminant. Studies suggest that veterans and workers exposed occupa
tionally to TCDD, other dioxins, and DLCs experience an increased risk of
developing a potentially disfiguring skin lesion (called chloracne), liver dis
ease, and possibly cancer and diabetes.
Fortunately, background exposures for most people are typically much
lower than those seen in either Vietnam veterans or occupationally exposed
workers. The potential adverse effects of TCDD, other dioxins, and DLCs
from long-term, low-level exposures to the general public are not directly
observable and remain controversial. One major controversy is the issue of
estimating risks at doses below the range of existing reliable data. Another
controversy is the issue of appropriately assessing the toxicity of various
mixtures of these compounds in the environment.
In 2004, the U.S. Environmental Protection Agency (EPA), asked the
National Research Council (NRC) of the National Academies to review its
2003 draft document titled Exposure and Human Health Reassessment of
2,3,7,8-Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Compounds
(the Reassessment). This NRC report describes the Reassessment as very
comprehensive in its review and analysis of the extensive scientific literature
on TCDD, other dioxins, and DLCs. However, the NRC report finds sub
stantial room for improvement in the quantitative approaches used by EPA
to characterize risks. In particular, the committee recommends that EPA
more thoroughly justify and communicate its approaches to dose-response
modeling for health effects and make its criteria for selection of key data
sets more transparent. EPA should also improve how it handles and com
municates the substantial uncertainty that surrounds its various estimates
of health risks from low-level exposures to TCDD, other dioxins, and
DLCs. This NRC report provides a critical review of EPA’s Reassessment,
but the report is not a risk assessment and does not recommend exposure
levels for TCDD, other dioxins, or DLCs for regulatory consideration.
Rather the NRC report provides guidance to EPA on how the agency could
improve the scientific robustness and clarity of the Reassessment for its
ultimate use in risk management of TCDD, other dioxins, and DLCs in the
environment by federal, state, and local regulatory agencies.
Assessing Human Exposure to TCDD, Other Dioxins, and DLCs
People worldwide are exposed to background levels of TCDD, other
dioxins, and DLCs. Background exposures include those from the commer-
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PUBLIC SUMMARY
3
cial food supply, air, water, and soil. EPA’s 2003 draft Reassessment does
not identify many specific direct sources of human exposures to relatively
high levels of TCDD, other dioxins, or DLCs. EPA estimated background
concentrations based on studies conducted at various locations in North
America. Those studies examined a small number of locations and, hence,
may not fully characterize national variability. EPA derived its estimates of
TCDD, other dioxins, and DLCs in food from statistically based national
surveys, nationwide-sampling networks, food fat concentrations, and envi
ronmental samples of air, water, soil, and food.
According to recent estimates, background concentrations of TCDD,
other dioxins, and DLCs continue to decline. EPA’s estimates of releases of
these compounds to air, water, and land from reasonably quantifiable
sources in 2000 showed a decrease of 89% from its 1987 estimates. At least
one U.S. study determined that meat contains lower levels of TCDD, other
dioxins, and DLCs than samples from the 1950s through the 1970s. An on
going national study by the U.S. Department of Agriculture of the concen
trations of TCDD, other dioxins, and DLCs in beef, pork, and poultry
should allow for a time-trend analysis of food concentrations.
To assess the total magnitude of emissions of TCDD, other dioxins,
and DLCs, EPA used a “ bottom-up” approach that attempted to identify
all emission-source categories (such as combustion, metal processing, and
chemical manufacturing and processing) and then estimated the magnitude
of emissions for each category. The committee concludes that a “top-down”
approach would also provide useful information and could give rise to
significantly different estimates of the historical levels of emissions of
TCDD, other dioxins, and DLCs. A top-down approach would account for
measured levels in humans and the environment and consider the emission
sources required to account for these levels.
The committee also recommends that EPA set up an active database of
typical concentrations for TCDD, other dioxins, and DLCs present in food.
This database should be based on a collection of all available data and
updated on a regular basis with new data as they are published in the peerreviewed literature.
Cancer Risk and TCDD, Other Dioxins, and DLCs
The EPA Reassessment revisits EPA’s classification of TCDD, other
dioxins, and DLCs on their potential to cause cancer in humans. In 1985,
EPA classified TCDD as a “probable human carcinogen” based on the data
available and EPA’s classification criteria in place at the time. The Reassess
ment, which revisited this issue given the current evidence and a different
draft classification scheme, characterized TCDD as “carcinogenic to hu
mans.” In 2005, after completion of the Reassessment, EPA further revised
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
its cancer guidelines. In its charge, the NRC committee was specifically
asked to address “ the scientific evidence for classifying TCDD as a human
carcinogen.” 1 Referring to the definitions of chemical carcinogens in the
EPA’s current cancer guidelines, the NRC committee was split on whether
the evidence from available studies met all the criteria necessary for defini
tive classification of TCDD as “carcinogenic to humans,” although the
committee unanimously agreed on a classification for TCDD of at least
“likely to be carcinogenic to humans.” The committee believed that the
public health implications of the two terms appeared identical and for this
reason did not belabor the issue of classification. The committee concluded
that because the definition of “carcinogenic to humans” changed somewhat
from previous EPA guidelines and after submission of the Reassessment,
EPA should reevaluate its 2003 conclusion based on the criteria set out in
its 2005 cancer guidelines.
The committee agrees with EPA in classifying other dioxins and DLCs
as “ likely to be carcinogenic to humans.” However, because mixtures of
DLCs and other dioxins may include TCDD, EPA should reconsider its
classification of such mixtures as “likely to be carcinogenic to humans” if it
continues to classify TCDD as “carcinogenic to humans.”
Estimating Cancer Risks at Very Low Doses
Nearly all relevant cancer-risk data from human epidemiological stud
ies and experimental animal bioassays reflect doses much higher than those
typically experienced by humans from exposure to TCDD, other dioxins,
and DLCs in the general environment. Consequently, analysts must ex
trapolate well below the doses observed in the studies to consider typical
human exposure levels. This extrapolation involves two critical decisions:
(1) selecting a “ point of departure” (POD), which corresponds to the low
est dose associated with observable adverse effects within the range of data
from a study, and (2) selecting the mathematical model used to extrapolate
risk from typical human exposures that are well below the POD.
In general, EPA estimates the POD by setting it equal to the dose
producing the smallest positive effect observed in a study. The size of the
health effect it produces in the population determines the “ effective dose.”
For example, the 1% effective dose (referred to as the ED01) elicits an
additional 1% response and the ED05 elicits an additional 5% response
1The charge to the committee w as to evaluate EPA’s Reassessment of dioxins and D LCs.
Although other agencies, such as the International Agency for Research on Cancer (IARC),
have also done both qualitative and quantitative evaluations of dioxin carcinogenicity, the
committee focused solely on EPA’s Reassessment document, the associated scientific evidence,
and EPA’s definitions for carcinogen classification.
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PUBLIC SUMMARY
5
FIGURE S-1 Conceptual illustration of the effect of the selection of the point of
departure and the mathematical model used to extrapolate below the point of
departure on the risk estimate. Note that the 5% response rate is not drawn to
scale. If it were, the area of the extrapolation box would be much smaller. In this
illustration, the ED 0 5 has been selected as the point of departure for extrapolation
to lower doses.
above the “ background” response (the level of response that occurs in the
absence of any exposure). The response size depends on the difference
between the unexposed population and the largest response possible. For
example, consider the case of a 25% lifetime background risk of death from
cancer in an unexposed population and a highest possible cancer death rate
of 100%. In this case, the ED01 is the dose that increases the cancer death
rate by 1% of the difference between 100% and 25% , or by 0.75%. Thus,
the ED01 is the dose that increases the risk of dying from cancer from 25%
to 25.75%.
Estimating risks below the POD requires making assumptions about
how TCDD, other dioxins, and DLCs might cause cancer at lower expo
sures. For example, in the hypothetical illustration in Figure S-1, a biologi
cal mode of action implying that risk is proportional to dose would corre
spond to use of the dashed line below the POD. A biological mode of action
implying a sublinear dose-response relationship would correspond to the
shaded line below the POD.
The committee concludes that EPA’s decision to rely solely on a default
linear model lacked adequate scientific support. The report recommends
that EPA provide risk estimates using both nonlinear and linear methods to
extrapolate below PODs. If background exposures to humans result in
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
doses substantially less than the dose associated with the POD (the most
likely case in most instances but perhaps not for occupational exposures),
then an estimate of risk for typical human exposures to TCDD, other dioxins,
and DLCs would be lower in a sublinear extrapolation model than in the
linear model. Given the important regulatory implications of this assump
tion, the committee recommends that EPA communicate the scientific
strengths and weaknesses of both approaches so that the full range of uncer
tainty generated by modeling of the data is conveyed in the Reassessment.
The committee also concluded that EPA did not adequately quantify
the uncertainty associated with responses at the estimated value of the
POD. The estimated value of the response at a particular effective dose (like
the ED01) is typically uncertain for a variety of reasons related to the
challenge of conducting an epidemiological study or an animal study. For
example, in epidemiological studies, the number of enrolled subjects is
limited, it can be difficult to estimate the actual level of exposure, other
factors (such as smoking or exposure to other chemicals) can also cause
cancer, and so forth. The committee concludes that, although EPA dis
cussed many of these factors qualitatively, the agency should strive to more
comprehensively characterize the impact of these sources of uncertainty
quantitatively.
Estimating Noncancer Risk
To characterize the risks of adverse health effects other than cancer,
EPA typically identifies a dose, called the reference dose (RfD), below
which it anticipates no adverse effects from exposure even among sensitive
members of the population. EPA did not estimate an RfD for TCDD, other
dioxins, or DLCs in the Reassessment. The committee suggests that esti
mating an RfD would provide useful guidance to risk managers to help
them (1) assess potential health risks in that portion of the population with
intakes above the RfD, (2) assess risks to population subgroups, such as
those with occupational exposures, and (3) estimate the contributions to
risk from the major food sources and other environmental sources of TCDD,
other dioxins, and DLCs for those individuals with high intakes.
Given the existing data, the committee concurs with the conclusion in
EPA’s Reassessment that TCDD, other dioxins, and DLCs are likely to be
human immunotoxicants at “ some dose level.” However, the report finds
this conclusion inadequate. The committee recommends that EPA add a
section or paragraph to its Reassessment on the immunotoxicology of
TCDD, other dioxins, and DLCs in the context of the biological mecha
nisms responsible for health effects relevant to assessing the likelihood of
such effects occurring in humans at relatively low levels of exposure. The
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PUBLIC SUMMARY
7
risk characterization should provide some insight about the level of risk
given actual exposures.
Studies show that TCDD, other dioxins, and DLCs cause embryonic
and fetal development and reproduction problems in rodents and some
other species. However, the fetal rodent clearly shows more susceptibility
to adverse effects of TCDD, other dioxins, and DLCs than the adult rodent.
Given the lack of comparable human data, the committee recommends that
EPA more thoroughly address how animal pregnancy models might relate
to human reproductive and developmental toxicity and risk information.
The committee further recommends that, in areas with substantial
amounts of human clinical data and epidemiological data, EPA establish
formal, evidence-based approaches, including but not limited to those for
assessing the quality of the study and study design for classifying and
statistically reviewing all available data.
Communicating Variability and Uncertainty in Risk Estimates
Risk assessors must make many choices as they develop models to
characterize risks, including selecting appropriate data sets for low-dose
extrapolation, dose-response models, PODs, and so forth. Because risk
estimates reflect numerous sources of uncertainty and alternative assump
tions, EPA’s Reassessment should include a detailed discussion of variabil
ity (the range of risks reflecting true differences among members of the
population due to, for example, genetic and age differences) and uncer
tainty (the range of plausible risk estimates arising because of limitations in
knowledge). Although EPA addressed many sources of variability and un
certainty qualitatively, the committee noted that the Reassessment would
be substantially improved if its risk characterization included more quanti
tative approaches. Failure to characterize variability and uncertainty thor
oughly can convey a false sense of precision in the conclusions of the risk
assessment.
Estimating Toxicity of DLCs and Mixtures in the Environment
Risk managers base their decisions about cleanup and control of chemi
cals, such as TCDD, other dioxins, and DLCs, in the environment on
assessment of the risks. Because of the common mode of action in produc
ing health effects, EPA’s Reassessment assessed the cumulative toxicity of
the compounds. The approach taken by EPA and international public health
organizations relies on assigning each compound (dioxins, other than
TCDD, and DLCs) a “toxic equivalency factor,” which is an estimate of the
toxicity of the compound relative to TCDD. For example, a particular DLC
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
thought to result in one-tenth the risk of TCDD for the same level of
exposure would be assigned a toxicity equivalency factor of 0.1.
Because some mixtures may contain little or no measurable TCDD but
relatively large amounts of other dioxins and/or DLCs, the toxic equiva
lency factor plays a critical role in determining the mixture’s overall esti
mated toxicity (which is called the toxic equivalency quotient). Estimation
of TEFs is a critically important part of the risk assessment of environmen
tal mixtures of TCDD, other dioxins, and DLCs, because any environmen
tal sample typically contains a dozen or more similar substances, but often
very little TCDD. Also, TCDD, other dioxins, and DLCs break down at
different rates in the environment and are eliminated at different rates in
humans. Thus, although analysts may reasonably estimate the relative po
tency value for a given compound based on toxicity tests, the compound’s
contribution to total risk in an environmental (or biological) sample may
change over a period of many years. This change may occur because the
relative concentration in a sample may change with time, even though the
potency remains constant, and the estimated risk in a given sample depends
on both potency and concentration.
Even with the inherent uncertainties, the committee concludes that the
toxic equivalency factor methodology provides a reasonable, scientifically
justifiable, and widely accepted method to estimate the relative potency of
DLCs. However, the committee noted that the Reassessment should ac
knowledge the need for better uncertainty analysis of the toxicity values
and should provide at least some initial uncertainty analysis of overall
toxicity of environmental samples.
CONCLUDING REMARKS
The committee appreciates the dedication and hard work that went
into the creation of the Reassessment and commends EPA for its detailed
evaluation of an extremely large volume of scientific literature (particularly
Parts I and II of the Reassessment). The NRC report focused its review on
Part III of the Reassessment and offers its recommendations with the inten
tion of helping to guide EPA in its efforts to make and implement environ
mental policies that adequately protect human health and the environment
from the potential adverse effects of TCDD, other dioxins, and DLCs. The
committee recognizes that it will require a substantial amount of effort for
EPA to incorporate all the changes recommended in this NRC report. Nev
ertheless, the committee encourages EPA to finalize the current Reassess
ment as quickly, efficiently, and concisely as possible after addressing the
major recommendations in this report. The committee notes that new ad
vances in the understanding of TCDD, other dioxins, and DLCs could
require reevaluation of key assumptions in the EPA risk assessment docu-
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PUBLIC SUMMARY
ment. The committee recommends that EPA routinely monitor new scien
tific information related to TCDD, other dioxins, and DLCs, with the
understanding that future revisions should provide risk assessment based
on the current state-of-the-science. However, the committee also recognizes
the importance of stability in regulatory policy to the regulated community
and thus suggests that EPA establish criteria for identifying when compel
ling new information warrants science-based revisions in its risk assess
ment. The committee finds that the recent dose-response data released by
the National Toxicology Program after submission of the Reassessment
represent good examples of new and compelling information that warrants
consideration in a revised risk assessment.
COMM ITTEE’S KEY FINDINGS
The committee identified three areas that require substantial improve
ment in describing the scientific basis for EPA’s dioxin risk assessment to
support a scientifically robust risk characterization:
• Justification of approaches to dose-response modeling for cancer
and noncancer end points.
• Transparency and clarity in selection of key data sets for analysis.
• Transparency, thoroughness, and clarity in quantitative uncertainty
analysis.
The following points represent Summary recommendations to address
the key concerns:
• EPA should compare cancer risks by using both a linear model and a
nonlinear model consistent with a receptor-mediated mechanism of action
and by using epidemiological data and the new NTP animal bioassay data.
The comparison should include upper and lower bounds, as well as central
estimates of risk. EPA should clearly communicate this information as part
of its risk characterization.
• EPA should identify the most important data sets to be used for
quantitative risk assessment for each of the four key end points (cancer,
immunotoxicity, reproductive effects, and developmental effects). EPA
should specify inclusion criteria for the studies (animal and human) used
for derivation of the benchmark dose (BMD) for different noncancer effects
and potentially for the development of RfD values and discuss the strengths
and limitations of those key studies; describe and define (quantitatively to
the extent possible) the variability and uncertainty for key assumptions
used for each key end-point-specific risk assessment (choices of data set,
POD, model, and dose metric); incorporate probabilistic models to the
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
extent possible to represent the range of plausible values; and assess goodness-of-fit of dose-response models for data sets and provide both upper
and lower bounds on central estimates for all statistical estimates. When
quantitation is not possible, EPA should clearly state it and explain what
would be required to achieve quantitation.
• When selecting a BMD as a POD, EPA should provide justification
for selecting a response level (e.g., at the 10%, 5%, or 1% level). The effects
of this choice on the final risk assessment values should be illustrated by
comparing point estimates and lower bounds derived from selected PODs.
• EPA should continue to use body burden as the preferred dose met
ric but should also consider physiologically based pharmacokinetic model
ing as a means to adjust for differences in body fat composition and for
other differences between rodents and humans.
The committee encourages EPA to calculate RfDs as part of its effort to
develop appropriate margins of exposure for different end points and risk
scenarios.
Copyright © National Academy of Sciences. All rights reserved.
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Su m m ary
Dioxins are a class of chemicals, and the most toxic of these com
pounds is 2,3,7,8-tetrachlorodibenzo-p-dioxin (commonly referred to as
TCDD or dioxin). There are many forms of dioxins and “ dioxin-like com
pounds” (DLCs) that share most, if not all, of the toxic potential of TCDD,
although nearly all are considerably less potent. Included in the list of DLCs
are chlorinated forms of dibenzofurans and certain polychlorinated biphe
nyls (PCBs).
Combustion, metal processing, chemical manufacturing and process
ing, and other sources emit TCDD, other dioxins, and DLCs into the envi
ronment. Unlike PCBs, TCDD and other dioxins have never been intention
ally produced. TCDD, other dioxins, and DLCs persist and bioaccumulate
in the environment, which means that they break down slowly and build up
through the food chain. Human exposure to TCDD, other dioxins, and
DLCs occurs primarily from eating foods, such as beef, dairy products, fish,
shellfish, and pork. In recent years, efforts to reduce the amount of TCDD,
other dioxins, and DLCs in the environment have resulted in reductions in
measured concentrations in the environment and in human blood.
TCDD, other dioxins, and DLCs share a common mode of action in
producing toxic effects in humans and animals. They bind to a specific
receptor, called the aromatic hydrocarbon receptor or Ah receptor; such
binding is a necessary, but not sufficient, step toward producing adverse
health effects.
A few industrial accidents and occupational exposures to substantial
amounts of TCDD, other dioxins, and DLCs have provided opportunities1
11
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
to assess the toxicity of these compounds to humans. Several episodes of
high-level human exposure to TCDD have been found to cause a specific
type of persistent, potentially disfiguring skin lesion called chloracne. In
2004, the media widely publicized the suspected intentional poisoning of
Viktor Yushchenko with TCDD after he developed chloracne during the
Ukraine presidential campaign. In contrast to the undisputed high-dose
effects of chloracne, the potential adverse effects of TCDD, other dioxins,
and DLCs in humans after long-term, low-level environmental exposures
remain controversial. The major controversies include how to classify the
potential of these compounds to cause cancer in humans (as either “carci
nogenic to humans” or “likely to be carcinogenic to humans” ), how to
estimate the potential health risks at very low doses typical of actual popu
lation exposures, and how to assess the toxicity of each of the compounds
and various mixtures of them in the environment.
TCDD, other dioxins, and DLCs have been regulated extensively
worldwide. In the early 1980s, the U.S. Environmental Protection Agency
(EPA) and other organizations, such as the World Health Organization
(WHO), began collecting and evaluating scientific information about the
sources, fate, and effects of the compounds. In 1985, EPA produced an
initial assessment of the human health risks from environmental exposure
to TCDD. Later, as new scientific information became available, EPA
reassessed the human health risks in an open process involving participa
tion of numerous scientists external to the agency, a series of public
meetings, and peer review.
An Interagency Working Group (IWG) made up of representatives of
seven federal agencies was established in 2000 to coordinate federal strate
gies for risk management of TCDD, other dioxins, and DLCs. Members of
the IWG, EPA’s Science Advisory Board, and the public commented on
earlier drafts of EPA’s dioxin risk assessment, and after further revisions,
EPA released the 2003 draft document titled Exposure and Human Health
Reassessment of Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Com
pounds (referred to as the Reassessment). The IWG recommended further
review of the new document, and in 2004, EPA asked the National Re
search Council (NRC) to convene an expert committee to review indepen
dently EPA’s 2003 draft Reassessment and to determine whether EPA’s risk
estimates are scientifically robust and whether there is clear delineation of
all substantial uncertainties and variabilities (Box S-1).
This report presents the committee’s conclusions and recommenda
tions. In general, the committee recommends that EPA substantially aug
ment its Reassessment to improve the transparency about assumptions used
to estimate risk and how these assumptions affect estimates. The committee
also recommends that EPA re-estimate the risks using several assumptions
and communicate the uncertainty in these estimates to the public.
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13
BOX S-1 Statement of Task
The National Academies' National Research Council will convene an expert
committee that will review EPA's 2003 draft reassessment of the risks of dioxins
and dioxin-like compounds to assess whether EPA's risk estimates are scientifical
ly robust and whether there is a clear delineation of all substantial uncertainties
and variability. To the extent possible, the review will focus on EPA's modeling
assumptions, including those associated with the dose-response curve and points
of departure; dose ranges and associated likelihood estimates for identified human
health outcomes; EPA's quantitative uncertainty analysis; EPA's selection of stud
ies as a basis for its assessments; and gaps in scientific knowledge. The study will
also address the following aspects of the EPA reassessment: (1) the scientific
evidence for classifying dioxin as a human carcinogen; and (2) the validity of the
non-threshold linear dose-response model and the cancer slope factor calculated
by EPA through the use of this model. The committee will also provide scientific
judgment regarding the usefulness of toxicity equivalence factors (TEFs) in the risk
assessment of complex mixtures of dioxins and the uncertainties associated with
the use of TEFs. The committee will also review the uncertainty associated with
the reassessment's approach regarding the analysis of food sampling and human
dietary intake data and, therefore, human exposures, taking into consideration the
Institute of Medicine's report Dioxin and Dioxin-Like Compounds in the Food Sup
ply: Strategies to Decrease Exposure. The committee will focus particularly on the
risk characterization section of EPA's reassessment report and will endeavor to
make the uncertainties in such risk assessments more fully understood by deci
sion makers. The committee will review the breadth of the uncertainty and variabil
ity associated with risk assessment decisions and numerical choices—for exam
ple, modeling assumptions, including those associated with the dose-response
curve and points of departure. The committee will also review quantitative uncer
tainty analyses, as feasible and appropriate. The committee will identify gaps in
scientific knowledge that are critical to understanding dioxin reassessment.
CARCINOGENIC CLASSIFICATION
In 1985, EPA classified TCDD as a “probable human carcinogen”
based on the data available at the time, but the latest Reassessment (2003)
stated that TCDD was better characterized as “ carcinogenic to humans.”
EPA and the International Agency for Research on Cancer (IARC), an arm
of WHO, have established criteria for qualitatively classifying chemicals
into various carcinogenic categories based on the weight of scientific evi
dence from animal, human epidemiological, and mechanism or mode-ofaction studies. In 1997, an expert panel convened by IARC concluded that
the weight of scientific evidence for dioxin carcinogenicity in humans sup
ported its classification as a Class 1 carcinogen— “carcinogenic to humans.”
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
In 2001, the U.S. National Toxicology Program (NTP) upgraded its classi
fication of dioxin to “known to be a human carcinogen.”
After reviewing EPA’s 2003 Reassessment and other scientific information
and in light of EPA’s recently revised 2005 Guidelines for Carcinogen Risk
Assessment (cancer guidelines), the committee concludes that the classification
of TCDD as “carcinogenic to humans”—a designation suggesting the greatest
degree of certainty about carcinogenicity—versus “likely to be carcinogenic to
humans”—the next highest designation—is somewhat subjective and depends
largely on the definition and interpretation of the criteria used for classification.
The true weight of evidence lies on a continuum, with no obvious point or
“bright line” that readily distinguishes those two categories.
Referring to the specific definitions in EPA’s 2005 cancer guidelines for
qualitative classification of chemical carcinogens, the NRC committee was
split on whether the evidence met all the criteria necessary for classification
of TCDD as “carcinogenic to humans,” although the committee unani
mously agreed on a classification of at least “likely to be carcinogenic to
humans.” The committee concludes that the weight of epidemiological evi
dence supporting classification of TCDD as a human carcinogen is not
“ strong.” The committee points out, however, that the human data avail
able from occupational studies show a modest positive association between
relatively high concentrations of TCDD in the body and increased mortality
from all cancers. Animal studies and mechanistic data provide additional
support for classifying TCDD as a human carcinogen.
The committee concludes that the distinction between those two quali
tative categories of cancer risk classification depends more on semantics
than on science and that the public health implications of the two terms
appeared identical, and for these reasons the committee did not focus much
attention on the issue of classification. To the extent that EPA can be
consistent with regulatory requirements, the committee recommends that
EPA focus its energies and resources on more carefully quantifying risks
and uncertainties for TCDD, other dioxins, and DLCs rather than on
whether its carcinogenicity is probable or proven. Because the 2005 cancer
guidelines’ definition of “carcinogenic to humans” has changed since EPA
completed its 2003 Reassessment, the committee recommends that EPA
reevaluate its conclusion that TCDD satisfies the criteria for designation as
either “carcinogenic to humans” or “ likely to be a human carcinogen”
based on the criteria set out in EPA’s 2005 cancer guidelines.
The committee agrees with EPA in classifying dioxins, other than
TCDD, and DLCs as “likely to be carcinogenic to humans.” However,
because mixtures of DLCs may also contain dioxins, including TCDD, EPA
should reconsider its classification of such mixtures as “ likely to be carcino
genic to humans” if it continues to classify dioxin as “carcinogenic to
humans.”
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SUMMARY
15
FIGURE S-1 Conceptual illustration of the effect of the selection of the point of
departure and of the mathematical model used to extrapolate below the point of
departure on the risk estimate. Note that the 5% response rate is not drawn to
scale. If it were, the area of extrapolation box would be much smaller.
ESTIMATING CANCER RISK
Because nearly all data (both human epidemiological studies and ex
perimental animal bioassays) relevant to cancer risk are for doses much
higher than those to which the general human population is typically ex
posed, analysts must extrapolate below the doses observed when estimating
risks. This extrapolation depends on first fitting a dose-response curve to
the observed data from a given study and choosing a “ point-of-departure”
(POD) dose, which corresponds to the lowest dose associated with adverse
effects within the range of the data from the experiment or study. The POD
dose is an incremental “ effect” observed; for example, analysts would call a
POD corresponding to a 5% increase in effects (above no exposure) a 5%
effective dose or an ED05.
Estimating risks below the POD may require extrapolating down to
background levels of exposure. See Figure S-1 for a conceptual illustration
of a dose-response extrapolation to background levels using the 5% re
sponse rate and ED05 as the POD. This extrapolation must be based on
assumptions about how TCDD, other dioxins, and DLCs might cause can
cer. Thus, the selection of the type of mathematical model used to extrapo
late below the POD is a critical decision in the cancer risk assessment
process. In the 2003 Reassessment, EPA chose to extrapolate below the
POD with a “ linear” model, which assumes that the biological response
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
increases proportionally with the level of exposure starting at a dose of
zero. Risk estimates based on this approach are generally higher than those
based on alternative “nonlinear” assumptions, where the biological re
sponse does not vary proportionally with the dose. However, EPA took the
position that scientific data were inadequate to rule out its default linear
assumption.
Selection of the POD also is an important choice in cancer risk assess
ment modeling because it determines the range of extrapolation below the
observed data range. For example, more extrapolation below the POD is
necessary when using a POD equal to the ED10 than when using a POD
equal to the ED01. However, using an ED01 requires more data because the
analyst must be able to detect a 1% increase in effects instead of a larger
increase (e.g., a 5% increase for an Ed05).
After reviewing EPA’s 2003 Reassessment and additional scientific
data published since completion of the Reassessment, the committee
unanimously agreed that the current weight of scientific evidence on the
carcinogenicity of dioxin is adequate to justify the use of nonlinear meth
ods consistent with a receptor-mediated response to extrapolate below
the POD. The committee points out that data from NTP released after
EPA generated the 2003 Reassessment provide the most extensive infor
mation collected to date about TCDD carcinogenicity in test animals, and
the committee found the NTP results to be compelling. The committee
concludes that EPA should reevaluate how it models the dose-response
relationships for TCDD, other dioxins, and DLCs. Specifically, the com
mittee determined that the scientific evidence is consistent with receptormediated responses and favors the use of a nonlinear model over the
default linear assumption to extrapolate below the POD for dioxin-re
lated cancer risk. The committee recognizes that a linear response at doses
below the POD cannot be entirely excluded, especially if background
exposures are not orders of magnitude below the POD and additivity of
risk from other types of chemicals is considered.
Because the committee concludes that the data support the hypothesis
that the dose-response relationship for dioxin and cancer is sublinear, it
recommends that EPA include a nonlinear model for cancer risk estimates
but also use the current linear models for comparative purposes. EPA should
then describe the scientific strengths and weaknesses of each approach to
inform risk managers about the importance of these assumptions. The
committee recognizes that additional evidence about dioxin carcinogenicity
will continue to develop and concludes that EPA should proceed with
completing its quantitative cancer risk assessment and include the recent
NTP data and appropriate nonlinear dose-response models.
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SUMMARY
17
ESTIMATING NONCANCER RISK
To characterize the risks of adverse health effects other than cancer at
very low doses, EPA typically identifies a dose called the reference dose
(RfD) below which it anticipates no adverse effects from exposure, even
among sensitive members of the population. To estimate the RfD, EPA
usually starts with a benchmark dose (BMD), which is the level of exposure
in an epidemiological study or an animal experiment that produces a cer
tain specified level of response. For example, the BMD might be defined as
the level of exposure at which 5% of exposed animals or people exhibit a
specific type of adverse effect. EPA then calculates the RfD by dividing the
BMD by a series of uncertainty factors intended to take into account several
sources of uncertainty. These sources of uncertainty include extrapolation
from animals to humans (allowing for a more sensitive response in humans
than that in the test animals), extrapolation within the population (allow
ing for more sensitive members of the human population), and database
sufficiency considerations (allowing for the possibility that more data might
reveal more sensitive effects).
EPA did not estimate an RfD for TCDD, other dioxins, and DLCs in
the Reassessment. However, the committee noted that defining an RfD
would provide useful guidance to risk managers to help them (1) assess
potential health risks in that portion of the population with intakes above
the RfD, (2) assess risks to population groups, such as those with occupa
tional exposures, and (3) estimate the risk contributions of the major food
sources and other environmental sources for those individuals with high
intakes. Alternatively, EPA could undertake risk characterization for differ
ent adverse effects by comparing noncancer dose-response data to relevant
human exposure data in the calculation of margins of exposure (MOEs),1
as was done in the Reassessment. Such MOEs, accompanied by a descrip
tion of associated uncertainties, could provide risk managers with informa
tion that would help to inform their decisions. Although EPA concluded
that calculating RfDs would not provide useful information, the committee
concludes that the information might be useful if EPA also considered that
the use of body burden (estimate of the total amount of chemical in the
body at steady state for a defined rate of exposure) as a dose metric would
already take into account some of the uncertainty factors that EPA would
typically use to adjust the BMD or POD in estimating an RfD. Estimates of
background exposures in the United States also appear to have continued to
decline, in part due to enhanced analytical detection.
1EPA defines M O E as the lowest “ E D 1 0 or other point of departure divided by the actual
or projected environmental exposure of interest” (http://www.epa.gov/iris/gloss8.htm#m).
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
The committee concludes that EPA did not adequately justify the use of
the 1% response level (the ED01) as the POD for analyzing epidemiological
or animal bioassay data for both cancer and noncancer effects. The com
mittee recommends that EPA more explicitly address the importance of the
selection of the POD and its impact on risk estimates by calculating risk
estimates using alternative assumptions (e.g., the ED05).
The committee commends EPA’s extensive dose-response modeling ef
forts of a large number of data sets, particularly those for noncancer effects,
but remains concerned about selection of the final model for computing the
POD. It is critical that the model used for determining a POD fit the data
well, especially at the lower end of the observed responses. Whenever fea
sible, mechanistic and statistical information should be used to estimate the
shape of the dose-response curve at lower doses. At a minimum, EPA
should use rigorous statistical methods to assess model fit, and to control
and reduce the uncertainty of the POD caused by a poorly fitted model. The
overall quality of the study design is also a critical element in deciding
which data sets to use for quantitative modeling.
UNCERTAINTY AND VARIABILITY IN RISK ESTIMATES
Risk assessors must make many choices as they develop models to
characterize risks. Some of the initial choices are selecting appropriate data
sets for low-dose extrapolation, selecting appropriate dose-response mod
els, selecting critical end points, and selecting an appropriate POD (e.g.,
ED01 versus ED05). Risk estimates routinely reflect numerous sources of
both uncertainty (which describes the range of plausible risk estimates
arising because of limitations in knowledge) and variability (which de
scribes the range of risks arising because of true differences—for example,
genetic and age differences among members of the population). Failure to
fully characterize uncertainty and variability can convey a false sense of
precision in the conclusions of the risk assessment. EPA should include a
detailed discussion of both uncertainty and variability.
Overall, the committee concludes that EPA addressed many sources of
uncertainty and variability qualitatively, but it did not adequately quantify
either the uncertainty or the variability of many. In the case of its cancer
risk estimates, EPA should provide quantitative estimates corresponding to
(1) central, upper-bound, and lower-bound estimates of the POD; (2) the
use of different plausible POD values; (3) different plausible mathematical
functions fit to the observed epidemiological data; and (4) different as
sumptions for estimating historical exposures among subjects in the epide
miological studies. In the case of the noncancer risk estimates, EPA should
characterize the uncertainty associated with (1) fitting a dose-response rela
tionship to the available data and (2) selecting a POD. If necessary, EPA
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SUMMARY
should acknowledge that the information available remains insufficient to
support a meaningful point estimate.
EPA’s discussion of epidemiological studies in Part III of the Reassess
ment, Integrated Summary and Risk Characterization for TCDD and
Related Compounds, should clearly specify inclusion criteria for those
studies used as a basis to support quantitative risk estimates. The commit
tee notes that EPA could substantially improve the transparency and man
agement of the uncertainties and complexities of the risk assessment for
TCDD, other dioxins, and DLCs by creating an ongoing process for clearly
identifying and updating the key assumptions that support the quantita
tive risk assessment.
ESTIMATING TOXICITY OF DLCS AND MIXTURES
Many DLCs and dioxins, other than TCDD, present in the environ
ment are capable of producing toxicological effects similar or identical to
those of TCDD. Substantial efforts have been aimed at simplifying estima
tion of risk for these compounds and for mixtures of them. EPA and inter
national public health organizations have tended to take the approach of
assigning each compound (dioxins, other than TCDD, and DLCs) a toxic
equivalency factor (TEF), which represents a scaling factor for estimating
the toxicity of the compound relative to TCDD. For example, a substance
with a TEF of 0.1 is estimated to be 10% as toxic as dioxin per unit mass.
Estimation of TEFs is a critically important part of the risk assessment of
environmental mixtures of TCDD, other dioxins, and DLCs, because any
environmental sample typically contains a dozen or more similar substances,
but often very little TCDD. TCDD, other dioxins, and DLCs break down at
different rates in the environment and have different elimination rates in
humans. Thus, although analysts may reasonably estimate the relative po
tency value for a given compound based on toxicity tests, the compound’s
contribution to total risk in an environmental (or biological) sample may
change over a period of many years. This change may occur because the
relative concentration in a sample may change with time, even though the
potency remains constant, and the estimated risk in a given sample depends
on both potency and concentration. Because these mixtures may contain
little or no TCDD but relatively large amounts of low-potency dioxins and/
or DLCs, TEFs are a critical factor in determining the mixture’s overall
estimated toxicity. Analysts refer to the aggregate weighting by TEF of a
mixture as the mixture’s toxic equivalent quotient (TEQ).
The recent NTP studies on TCDD and several other dioxins and DLCs
provide additional evidence in support of the TEF approach. Uncertainty
about the validity of the approach led the NTP to specifically test the TEF
value for one particular PCB (126) in its analyses, and the results showed
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
excellent agreement between the predicted TEF for PCB 126 and the value
observed in the NTP experiment.
Overall, even given the inherent uncertainties, the committee agrees
that the TEF method is reasonable, scientifically justifiable, and widely
accepted for the estimation of the relative toxic potency of TCDD, other
dioxins, and DLCs. The TEF approach has also been used in other contexts.
WHO’s International Programme on Chemical Safety used the approach in
assessing the risks of different polycyclic aromatic hydrocarbons (PAHs)
relative to benzo(a)pyrene as an indicator PAH.
The committee concludes that the recent NTP results, released after
EPA completed its 2003 Reassessment, provide important additional sup
port for the TEF approach. However, EPA should acknowledge the need
for better uncertainty analysis of the TEF values and should, as a follow-up
to the Reassessment, establish a task force to begin to address this uncer
tainty by developing “consensus probability density functions” for dioxins
and DLCs. The committee recommends that EPA clearly address TEF un
certainties in the Reassessment.
SCALING DATA FROM ANIMAL STUDIES
For risk assessments that rely on experimental animal data, determining
the most appropriate way to scale the data from the animal model (usually
rats and mice) to humans is another important risk assessment choice. Nu
merous options for choosing dose metrics exist, and they can yield results
different from the traditional daily dose metric based on per unit of body
weight. For highly persistent chemicals like TCDD, other dioxins, and DLCs,
substantial differences in the rates of elimination from the body will result in
very different amounts of chemical accumulated in the body over time, even
with the same daily dose rate expressed in body weight or body surface area
units. In the 2003 Reassessment, EPA used an estimate of the total amount of
chemical in the body at steady state for a defined rate of exposure, called the
body burden, as the dose metric to adjust for differences in body weight (or
surface area) and in elimination rates.
The committee agrees with EPA’s conclusion that use of body burden as
the dose metric appears to be the most reasonable and pragmatic approach
for dioxin risk assessment, but EPA should address important uncertainties
quantitatively in more detail when possible. One such uncertainty, not quan
titatively addressed in EPA’s 2003 Reassessment, relates to species differences
in body fat expressed as a percentage of total body weight. Differences in
body fat content have a potentially large impact on dioxin concentrations
present in nonfatty tissues, including such organs as the liver.
Large errors may also arise from trying to estimate the overall body
burden TEQs for humans based on intake TEFs from rats. The errors result
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SUMMARY
from uncertainties in the differences in how long TCDD, other dioxins, and
DLCs persist in humans and rodents and uncertainties about how these
compounds concentrate in tissues.
The committee recommends that EPA’s Reassessment use basic physi
ologically based pharmacokinetic (PBPK) models to estimate the differences
between humans and rodents in the relationship between total body burden
at steady state, as calculated from the intake, half-life, bioavailability, and
tissue concentrations, and use the results to modify the estimated human
equivalent intakes. The committee also recommends that EPA provide a
clear evaluation of the impact of using body burden as the dose metric,
relative to other possible options such as intake, on the final risk estimates.
HUMAN EXPOSURE TO TCDD, OTHER DIOXINS, AND DLCS
Estimating human exposure levels, including those representative of
background levels (e.g., typical dietary intake levels) and levels resulting
from specific exposure scenarios (e.g., accidental, occupational, and highly
exposed communities), is a critical component of any chemical risk assess
ment. The extensive environmental persistence of TCDD, other dioxins,
and DLCs and their global environmental distribution create many possible
sources and routes of exposure to these compounds, and determining typi
cal background rates of exposure is difficult. EPA’s 2003 Reassessment
addresses exposure to TCDD, other dioxins, and DLCs in terms of sources,
environmental fate, environmental media concentrations, food concentra
tions, background exposures, and potentially highly exposed populations.
To assess total dioxin and DLC emissions, EPA used a “ bottom-up”
approach in which it attempted to identify all source categories and then
estimated the emissions for each category. However, a “top-down” ap
proach that attempts to account for measured levels and considers the
emission sources required to account for those levels would provide useful
additional information. Such alternative approaches may give rise to sig
nificantly different estimates of the historical levels of dioxin and DLC
emissions. Both approaches come with uncertainties, and EPA could benefit
substantially from using the approaches simultaneously to set plausible
bounds on historical trends and current levels in emissions.
The committee also recommends that EPA more explicitly define its
procedures for addressing analytical measurements that fall below the limit
of detection in environmental and exposure media samples. Consideration
of the detection limits is important in assessing background exposure esti
mates. Typically, samples that contain small or no amounts of dioxin
(“nondetects” ) are given a value of 50% of the lowest level measurable by
the instrument (the detection limit). For example, if the detection limit was
1 part per billion (ppb), a sample that contained 0.1 ppb would be assigned
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
a value of 0.5 ppb (half of the detection limit), or 5 times greater than its
actual value. If the detection limit decreased to 0.05 ppb, the actual value of
0.1 ppb would be reported. In addition, as analytical detection limits im
prove, the estimates of contaminants in background environmental samples
become more accurate as nondetect samples become fewer and the range of
uncertainty is narrowed.
Although beyond the scope of the review of the EPA Reassessment, the
committee notes that it would be useful for EPA to set up a compoundspecific, active database of typical concentrations for the range of TCDD,
other dioxins, and DLCs present in dietary and other environmental sources.
This database should undergo regular updates to capture new data as they
appear in the peer-reviewed literature. Such a database should include clear
requirements of data quality and traceability (chemical analysis, representa
tive and targeted sampling, representative of consumer exposure, presenta
tion of data, and handling and presentation of “nondetects” ). Chapter 4
provides several additional recommendations about the exposure assess
ment section of the 2003 Reassessment.
IMM UNOTOXICITY OF TCDD, OTHER DIOXINS, AND DLCS
TCDD, other dioxins, and DLCs have well-known effects on the im
mune systems of experimental animals. Chemically induced alterations in
immune function could result in various adverse health outcomes because
the immune system plays a critical role in fighting off infections, killing
cancer cells at early stages, and implementing numerous other health-pro
tective functions.
In light of the large database showing that TCDD, other dioxins, and
DLCs produce immunotoxic responses in laboratory animal studies, com
bined with sparse human data, the committee agrees with EPA’s conclusion
that these compounds are potential human immunotoxicants.
However, EPA’s conclusion that dioxins, other than TCDD, and DLCs
are immunotoxic at “ some dose level” is inadequate. At a minimum, EPA
should add a section or paragraph that discusses the immunotoxicology of
these compounds in the context of current Ah receptor biology. EPA should
also include some discussion about the implications of using genetically
homogeneous inbred mice to characterize immunotoxicological risk in the
genetically variable human population.
REPRODUCTIVE AND DEVELOPMENTAL TOXICITY OF TCDD,
OTHER DIOXINS, AND DLCS
Reproduction and embryonic and fetal development are sensitive end
points from rodent exposure to TCDD, other dioxins, and DLCs. Although
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SUMMARY
the fetal rodent consistently appears to be more susceptible to adverse
effects of these compounds than the adult rodent, comparable human data
do not exist, and the susceptibility of humans to these end points is less well
determined.
EPA’s 2003 Reassessment comprehensively covers developmental and
reproductive toxicity of TCDD, other dioxins, and DLCs in several models.
One rodent model included TCDD administration during pregnancy and
thus tested the disruption of development of the pups and their reproduc
tive function later in life. The 2003 Reassessment presented a comprehen
sive overview of the pregnancy model, but it did not provide an adequate
discussion of the doses used in the studies or the relationships of animal
studies to human reproductive and developmental toxicity. The committee
recommends that EPA more thoroughly address how the effective doses
used in the animal pregnancy models relate to human reproductive and
developmental toxicity and risk information, including TEFs and TEQs.
The 2003 Reassessment also did not provide an adequate discussion of
other models (e.g., effects of TCDD on ovulation in adult rats).
OTHER TOXIC END POINTS
Although TCDD, other dioxins, and DLCs have received wide recogni
tion for their potential to cause cancer, birth defects, reproductive disor
ders, immunotoxicity, and chloracne, animal and human studies have dem
onstrated other potential toxic end points, including liver disease, thyroid
dysfunction, lipid disorders, neurotoxicity, cardiovascular disease, and
metabolic disorders, such as diabetes.
The committee agrees that EPA has in general adequately addressed the
available data on the likelihood that exposure to TCDD, other dioxins, and
DLCs is a significant risk factor for other toxic end points. EPA cautiously
stated its overall conclusions about noncancer risks due to TCDD, other
dioxins, and DLCs exposures and acknowledged the uncertainty of sus
pected relationships. Nonetheless, the committee notes that EPA did not
uniformly address the limitations of individual human studies. Similarly,
EPA did not discuss the broad 95% confidence intervals accompanying
some reported statistically significant effects in the context of the uncer
tainty (and, perhaps, individual variability) that these broad confidence
limits imply. Conversely, the 2003 Reassessment highlights statistically non
significant effects in some cases, suggesting an implied potential for unob
served detrimental effects without a supporting presentation of a firm evi
dence base. The committee recommends that EPA establish formal principles
and mechanisms for evidence-based classification and systematic statistical
review, including meta-analysis when possible, for available human, clini
cal, and noncancer end-point data.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
New studies of the effects of dioxin on the developing vascular system
suggest a potentially sensitive target for TCDD, other dioxins, and DLCs.
The committee recommends that EPA identify this area as an important
data gap in the understanding of the potential adverse effects of these
compounds.
EPA’S OVERALL APPROACH TO RISK CHARACTERIZATION
Risk characterization is the culminating step in risk assessment. It
should attempt to pull together all the relevant scientific information on
toxicity and exposure for a coherent, quantitative understanding of poten
tial health risks and on the uncertainties that surround the estimates of risk.
Ideally, the risk characterization component of a risk assessment provides
risk managers with a user-friendly synopsis of the scientific basis that un
derpins an agent’s potential impact on public health under defined expo
sure conditions and scenarios.
As discussed previously, selection of the default linear extrapolation
approach for carcinogenicity emerged as one of the most critical decisions
in the 2003 Reassessment. The committee concludes that EPA did not
support its decision adequately to rely solely on this default linear model
and recommends that EPA add a scientifically rigorous evaluation of a
nonlinear model that is consistent with receptor-mediated responses and
uses the recent NTP cancer bioassay studies. The committee determined
that the available data support the use of a nonlinear model, which is
consistent with receptor-mediated responses and a potential threshold, with
subsequent calculations and interpretation of MOEs. EPA’s sole use of the
default assumption of linearity and selection of the ED01 as the only POD to
quantify cancer risk does not provide an adequate quantitative character
ization of the overall range of uncertainty associated with the final esti
mates of cancer risk.
Because EPA decided not to derive an RfD, its traditional noncancer
metric, or any other alternative for noncancer effects, the 2003 Reassess
ment does not provide important detailed risk characterization information
about noncancer risks. Typically, when EPA estimates an RfD, the risk
characterization will include (1) estimates of the proportion of the popula
tion with intakes above the RfD; (2) detailed assessment of population
groups, such as those with occupational exposures; and (3) contributions of
the major food sources and other environmental sources for those individu
als with high intakes. If a nonlinear model consistent with a threshold were
used for cancer risk assessment, these same types of risk characterization
details could also be provided for cancer risk. The lack of such a focus in
the risk characterization section of the 2003 Reassessment results in a risk
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SUMMARY
characterization that is difficult to follow and does not provide clear guid
ance with respect to noncancer end points.
The committee recommends that EPA revise its risk characterization
chapter to clearly describe the following:
1. The effects seen at the lowest body burdens that are the primary
focus for any risk assessment—the “ critical effects.”
2. The modeling strategy used for each noncancer effect modeled, pay
ing particular attention to the critical effects, and the selection of a point of
comparison based on the biological significance of the effect; if the ED01 is
retained, then the biological significance of the response should be defined
and the precision of the estimate given.
3. The precision and uncertainties associated with the body burden
estimates for the critical effects at the point of comparison, including the
use of total body burden rather than modeling steady-state concentrations
for the relevant tissue.
4. The committee encourages EPA to calculate RfDs as part of its effort
to develop appropriate margins of exposure for different end points and
risk scenarios, including the proportions of the general population and of
any identified groups that might be at increased risk (See Table A-1 in the
Reassessment, Part III Appendix, for the different effects; appropriate expo
sure information would need to be generated.) Interpretation of the calcu
lated values should take into consideration the uncertainties in the POD
values and intake estimates.
5. Consideration of individuals in susceptible life stages or groups (e.g.,
children, women of childbearing age, and nursing infants) who might re
quire estimation of a separate MOE using specific exposure data.
6. Distributions that provide clear insights about the uncertainty in
the risk assessments, along with discussion of the key contributors to the
uncertainty.
The committee recommends that EPA substantially revise the risk char
acterization section of Part III of the Reassessment to include a more com
prehensive risk characterization and discussion of the uncertainties sur
rounding key assumptions and variables.
CONCLUDING REMARKS
The committee appreciates the dedication and hard work that went
into the creation of the Reassessment and commends EPA for its detailed
evaluation of an extremely large volume of scientific literature (particularly
Parts I and II of the Reassessment). This NRC report focuses its review on
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Part III of the Reassessment and offers its recommendations with the inten
tion of helping to guide EPA in its efforts to make and implement environ
mental policies that adequately protect human health and the environment
from the potential adverse effects of TCDD, other dioxins, and DLCs. The
committee recognizes that it will require a substantial amount of effort for
EPA to incorporate all the changes recommended in this report. Neverthe
less, the committee encourages EPA to finalize the current Reassessment as
quickly, efficiently, and concisely as possible after addressing the major
recommendations in this report. The committee notes that new advances in
the understanding of TCDD, other dioxins, and DLCs could require re
evaluation of key assumptions in EPA’s risk assessment document. The
committee recommends that EPA routinely monitor new scientific informa
tion related to TCDD, other dioxins, and DLCs, with the understanding
that future revisions may be required to maintain a risk assessment based
on the current state-of-the-art science. However, the committee also recog
nizes that stability in regulatory policy is important to the regulated com
munity and therefore suggests that EPA establish criteria for identifying
when compelling new information would warrant science-based revisions
in its risk assessment. The committee finds that the recent dose-response
data released by the NTP after submission of the Reassessment are good
examples of new and compelling information that warrants consideration
in a revised risk assessment.
COMM ITTEE’S KEY FINDINGS
The committee identified three areas that require substantial improve
ment in describing the scientific basis for EPA’s dioxin risk assessment to
support a scientifically robust risk characterization:
• Justification of approaches to dose-response modeling for cancer
and noncancer end points.
• Transparency and clarity in selection of key data sets for analysis.
• Transparency, thoroughness, and clarity in quantitative uncertainty
analysis.
The following points represent Summary recommendations to address
the key concerns:
• EPA should compare cancer risks by using nonlinear models con
sistent with a receptor-mediated mechanism of action and by using epide
miological data and the new NTP animal bioassay data. The comparison
should include upper and lower bounds, as well as central estimates of
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27
SUMMARY
risk. EPA should clearly communicate this information as part of its risk
characterization.
• EPA should identify the most important data sets to be used for
quantitative risk assessment for each of the four key end points (cancer,
immunotoxicity, reproductive effects, and developmental effects). EPA
should specify inclusion criteria for the studies (animal and human) used
for derivation of the benchmark dose (BMD) for different noncancer effects
and potentially for the development of RfD values and discuss the strengths
and limitations of those key studies; describe and define (quantitatively to
the extent possible) the variability and uncertainty for key assumptions
used for each key end-point-specific risk assessment (choices of data set,
POD, model, and dose metric); incorporate probabilistic models to the
extent possible to represent the range of plausible values; and assess goodness-of-fit of dose-response models for data sets and provide both upper
and lower bounds on central estimates for all statistical estimates. When
quantitation is not possible, EPA should clearly state it and explain what
would be required to achieve quantitation.
• When selecting a BMD as a POD, EPA should provide justification
for selecting a response level (e.g., at the 10%, 5%, or 1% level). The effects
of this choice on the final risk assessment values should be illustrated by
comparing point estimates and lower bounds derived from selected PODs.
• EPA should continue to use body burden as the preferred dose met
ric but should also consider physiologically based pharmacokinetic model
ing as a means to adjust for differences in body fat composition and for
other differences between rodents and humans.
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1
In tro d u ctio n
The U.S. Environmental Protection Agency (EPA) and other organiza
tions, such as the World Health Organization (WHO), began assessing the
potential risks to human health from exposure to 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD, commonly referred to as dioxin) decades ago.
Early studies suggested very high toxicity at very low doses in test animals
and potential carcinogenicity. The history of dioxin risk assessment is com
plicated and contentious (Thompson and Graham 1997). In 1985, EPA
produced an initial assessment of the human health risks from environmen
tal exposure to dioxin. Three years later, EPA, other federal agencies, and
the scientific community began developing a broad research program to
identify the biological response mechanisms and to explore other key scien
tific issues related to dioxin. In light of significant advances in the scientific
understanding of the mechanisms of dioxin toxicity, new studies of dioxin’s
carcinogenic potential in humans, and increased evidence of other adverse
health effects primarily after the 1985 assessment, EPA announced in 1991
that it would conduct a scientific reassessment of the health risks of human
exposure to TCDD and related compounds, that is, dioxins, other than
TCDD, and dioxin-like compounds (DLCs). The reassessment would re
spond to emerging scientific knowledge of the biological, human health,
and environmental effects of TCDD, other dioxins, and DLCs.
EPA conducted the reassessment process as an open and participatory
exercise, involving chapter authorship by scientists outside the agency, a
series of public meetings and peer-review workshops, and reviews by EPA’s
Science Advisory Board (SAB). EPA’s National Center for Environmental
28
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INTRODUCTION
29
Assessment (NCEA) headed the reassessment efforts with participation of
scientific experts in EPA, the National Institutes of Health’s (NIH) N a
tional Institute of Environmental Health Sciences (NIEHS), and other fed
eral agencies and scientific experts in the private sector and academia. EPA
sponsored open meetings in 1991 and 1992 to inform the public about the
assessment, receive public comments on plans and activities of the reassess
ment process, and obtain additional relevant scientific information. Peer
review workshops were convened in 1992 and 1993 to review initial drafts
of all background chapters. The workshops were followed by extensive
revision and additional review of some chapters. In 1994, EPA released for
public review all the chapters plus the first draft of a summary risk charac
terization chapter, received public comments on the drafts, and submitted
the documents to the SAB for review.
In 1995, the SAB, commenting on the 1994 draft assessment, proposed
several substantive and contingent recommendations, including revision of
the chapter on dose-response modeling for TCDD, development of a chap
ter on dioxin toxic equivalency factors (TEFs), and an external peer review
of redrafted or new chapters, including the chapter on risk characterization.
The SAB also recommended that EPA involve outside scientists from the
public and private sectors to help determine approaches for revising what
was then called Chapter 9: “Risk Characterization of TCDD and Related
Compounds.”
In 1996, EPA initiated interaction with a group of 40 stakeholders
from the public and private sectors to gather input on approaches for
conducting the risk characterization revision. EPA met regularly with the
group to ensure ongoing input as recommended by the SAB and shared
with them the initial post-SAB revision of the draft risk characterization.
EPA, with NIEHS, revised Chapter 8, developed a new Chapter 9 on
TEFs, and revised the former Chapter 9 and renamed it as a free-standing
report, “Part III—Integrated Summary and Risk Characterization for
2.3.7.8- Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Compounds.”
The dioxin report consisted of two other parts: “Part I—Estimating Expo
sure to Dioxin-Like Compounds” and “Part II—Health Assessment for
2.3.7.8- Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Compounds.”
All three parts are collectively referred to as the Reassessment.
On February 24, 1997, the Federal Register announced the public ex
ternal peer review and 60-day comment period of the revised Chapter 8,
“Dose-Response Modeling for 2,3,7,8-TCDD.” On June 12, 2000, the
Federal Register announced a similar peer review and public comment
period on the revised Part III—Integrated Summary and Risk Characteriza
tion and the revised TEFs Chapter 9 in Part II.
Recognizing the broad policy implications of the dioxin reassessment,
the National Science and Technology Council established an interagency
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
working group on dioxin (IWG) in the summer of 2000 to ensure a coordi
nated federal approach to dioxin-related health, food, and environmental
issues. Specifically, the IWG was charged with fostering information shar
ing, developing a common language for dioxin science and science policy
across governmental agencies and programs, identifying gaps and needs in
the dioxin risk assessment, and facilitating coordination of risk manage
ment strategies. The IWG includes representatives from the following fed
eral agencies: U.S. Department of Health and Human Services, U.S. Depart
ment of Agriculture, U.S. Department of State, U.S. Department of Veterans
Affairs, U.S. Department of Defense, the Executive Office of the President,
and EPA.
In the winter of 2000, the SAB held a 3-day public review of Part III of
the Reassessment and additional information on the toxic equivalence of
dioxins, other than TCDD, and DLCs. In the spring of 2001, the SAB
recommended that EPA proceed expeditiously to complete and release its
report, taking appropriate note of the SAB’s findings and recommendations
and public comments. In response, EPA revised its draft Reassessment and
submitted it to the IWG in late 2003, requesting input about the need and
benefit of further review. EPA appropriations language for fiscal year 2003
also called for an IWG evaluation of the need for further review and pro
vided specific issues to consider. The IWG recommended that the National
Academies’ National Research Council (NRC) review the draft Reassess
ment. The scope of work for the NRC review and interagency agreements
for funding were developed through the IWG in the spring of 2004. Ulti
mately, the NRC review would seek to inform and assure the risk charac
terization of TCDD, other dioxins, and DLCs and to benefit EPA in finaliz
ing its Reassessment.
TCDD, OTHER DIOXINS, AND DLCS
The Reassessment addresses a limited number of chemical compounds
within three subclasses of the halogenated aromatic hydrocarbons (HAHs):
the polychlorinated dibenzo-p-dioxins (PCDDs), the polychlorinated dibenzofurans (PCDFs), and the polychlorinated biphenyls (PCBs). These com
pounds contain the basic aromatic structure of a benzene ring, a hexagonal
carbon structure with conjugated double bonds connecting the carbons
(Figure 1-1). PCDDs and PCDFs have tricyclic (triple-ring) structures con
sisting of two benzene rings, with varying numbers of chlorines, connected
by an oxygenated ring, with the oxygenated ring of PCDDs having two
oxygen atoms (a dioxin, Figure 1-2a) and the oxygenated ring of PCDFs
having a single oxygen atom (a furan, Figure 1-2b). PCBs have a variable
number of chlorines attached to a biphenyl group (two benzene rings with
a carbon-to-carbon bond between carbon 1 on the first ring and carbon 1'
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INTRODUCTION
31
FIGURE 1-1 Benzene ring (a) with conjugated bonds and (b) with inner ring
depicting conjugated bonds.
FIGURE 1-2 Double benzene ring structures of (a) dioxins and (b) furans.
FIGURE 1-3
on the second ring) (Figure 1-3). Examples of some PCDDs, PCDFs, and
PCBs of interest are shown in Figure 1-4. Each chemical compound from
any of these subclasses is referred to as a congener. Brominated or mixed
halogenated congeners within these classes of compounds or within other
chemical classes, such as the polyhalogenated naphthalenes, benzenes,
azobenzenes, and azoxybenzenes, have not been evaluated as extensively
and are not addressed in the Reassessment. TCDD, the most studied and
one of the most toxic members of these classes of compounds, is the desig
nated reference chemical for the Reassessment and for other related litera-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
2,3,7 B-Tetrachlorodibenzo-p-dioxin
2.3.7.8-TetraehlorodibenzofLiran
1,2,3,7,8-Pentachlorodibe nzo-p-dioxm
2,3,4,7,8-Pe ntach iorodibe nzofu ra n
3,3 ,4,4 ,5,5 -Hexachlorobipheny]
3,3',4,4',5-Pentachlorobiphenyl
FIGURE 1-4 Examples of toxic PCDDs, PCDFs, and PCBs of interest in the
Reassessment.
ture. PCDDs and PCDFs are tricyclic aromatic compounds with similar
physical and chemical properties—properties shared by specifically config
ured, or coplanar (a flat configuration), dioxin-like PCBs. The Reassess
ment uses the terms “ dioxins” and “ dioxin-like compounds” in reference to
any individual or any mixture of the addressed chemicals. These are general
terms that describe chemicals that share defined similarities, including
chemical structure and biological and toxicological character. However, of
the several hundred HAH congeners, only 29 are considered to have signifi
cant toxicity and to induce a common battery of toxic responses through
similar biological modes of action. The evaluation of dioxin-like congeners
within the Reassessment focuses on dioxins and DLCs with TCDD-like
toxicity and those generally considered the most associated with environ
mental and human health risks. These chemicals include PCDDs and PCDFs
that retain chlorine substitutions at positions 2, 3, 7, and 8 on the benzene
rings (see Figure 1-2). The remaining evaluated TCDD-like congeners in
clude the PCBs with four or more chlorines in the lateral positions (3, 3', 4,
4', 5, or 5'), with established TCDD-like environmental and biological
behaviors, and particularly the mono- and non-ortho PCBs—that is, PCBs
with one or no, respectively, chlorine substitution in the ortho position (2,
2', 6, or 6') on the benzene rings (see Figure 1-3). Studies show that the
TCDD-like toxicity of the PCB congener increases with a larger number of
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INTRODUCTION
chlorines in the lateral positions and one or no chlorines in the ortho
position. Also, when a PCB has only one or no substitution in the ortho
position, the atoms of the PCB congener can line up in a coplanar or flat
configuration, making these the most toxic of the TCDD-like PCBs. Evalu
ation of the chemical congeners addressed in the Reassessment is consid
ered sufficient to characterize environmental chlorinated dioxins (Reassess
ment, Part I, p. 1-5, lines 4 to 6).
Experimental evidence indicates that TCDD acts by way of binding to
an intracellular protein, the aromatic hydrocarbon receptor (AHR), a
ligand-dependent transcription factor that functions in partnership with a
second protein, the AHR nuclear translocator protein (ARNT) to stimulate
alterations in gene expression that result in toxic and biological effects.
AHR is present throughout the animal kingdom, including invertebrates
like the fruit fly and the clam. In addition to AHR binding, several other
molecular events are necessary for AHR-dependent biological and toxic
effects to occur, and there are significant species differences in those events,
so quantitative cross-species comparisons based only on AHR binding may
not provide accurate or dependable information about their AHR respon
siveness or the possible AHR-dependent responses. The invertebrate AHR
does not bind xenobiotic ligands (that is, TCDD, other dioxins, and DLCs),
and it is not associated with toxic end points, suggesting that the role of
AHR as a mediator of toxic or adaptive responses might be a function
acquired during vertebrate evolution and superimposed on an endogenous
physiological role.
TOXIC EQUIVALENCY FACTORS
TCDD, other dioxins, and DLCs are generally present as complex
mixtures in environmental, food, and biological matrices, including hu
mans and other animals. To address the complexity of risk assessment for
TCDD-related compounds, the concept of TEFs is used in the Reassess
ment. The TEF concept originated as a method for evaluating health risks
associated with closely related chemicals with similar mechanisms of action
but different potencies. The criteria for including a chemical in the TEF
concept are described in detail in the Reassessment, Part II, Chapter 9. The
TEF of each chemical congener is determined by evaluating available con
gener-specific data (primarily in vivo data), and the congener is then as
signed an “ order of magnitude” estimate of relative toxicity compared with
the prototypical and most potent HAH, 2,3,7,8-TCDD. By using those
factors, the toxicity of a mixture is expressed in terms of its total toxic
equivalent quotient (TEQ), which is the amount of TCDD that it would
take to equal the combined toxic effect of all contributing congeners within
the mixture. The TEF value of each congener within a mixture is multiplied
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
by its concentration, and the products (TEQs) are summed to yield the total
TEQ of the mixture, which is the estimate of the total toxicity of the
mixture.
In 1997, a team of experts convened by the WHO European Center for
Environment and Health and the International Program on Chemical Safety
(IPCS) evaluated a large database of experimental data of the relative po
tencies for PCDDs, PCDFs, and dioxin-like PCBs to establish consensus
TEF values for these compounds in mammals, birds, and fish. Human and
mammalian TEFs were constructed by an approach that gave more weight
to in vivo toxicity data than to in vitro data. Moreover, among the different
in vivo studies available for establishing TEFs, the basis for selecting the
most relevant in vivo toxicity study was the length of exposure, with chronic
exposures ranking highest and acute exposures lowest in relevance. The
team concluded that an additive TEF model served as the most feasible risk
assessment method for complex mixtures of TCDD-like PCDDs, PCDFs,
and PCBs. Although several TEF/TEQ schemes exist for TCDD-related
compounds, the Reassessment recommends using the international WHO
TEF scheme of values, proposed and published by WHO (IPCS 1998a), to
assign toxic equivalency for the Reassessment. Table 1-1 presents WHO
TEFs established for humans and mammals for 7 PCDDs, 10 PCDFs, and
12 dioxin-like PCBs. TEF assignments continue to evolve in accordance
with emerging science and iteration, and WHO recommended revisiting
TEF values every 5 years, with review in 2005. For additional information
on the TEF/TEQ approach, see Chapter 3 of the Reassessment, Part II.
To facilitate evaluation of human health risks and regulatory control of
exposure to mixtures of TCDD, other dioxins, and DLCs, EPA, using all
available data, has incorporated the TEF concept and method into the risk
assessment process since 1987. The Reassessment considers the application,
limitations, and uncertainties when using TEFs. Part II, Chapter 9, of the
Reassessment describes the application of the TEF method for TCDD, other
dioxins, and DLCs and addresses the uncertainties in detail. Overall, the
use of the TEF method is currently the most reliable and best evaluated
approach for assessing the potential toxic potency of complex mixtures of
DLCs. TEFs/TEQs are addressed in further detail in Chapter 3.
EXPOSURE CHARACTERIZATION
EPA classifies sources of TCDD, other dioxins, and DLCs into five
categories—(1) combustion; (2) metal smelting, refining, and processing;
(3) chemical manufacturing and processing; (4) biological and photochemi
cal processes; and (5) reservoir sources. Combustion sources include incin
eration of various types of waste (municipal solid, sewage sludge, medical,
and hazardous), burning of fuels (coal, wood, and petroleum products),
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INTRODUCTION
TABLE 1-1 TEFs for Humans and Nonhuman Mammals
PCDD Congeners
W H O TEF
2 ,3,7,8-T C D D
1,2,3,7,8-PeC D D
1,2,3,4 ,7 ,8 -H xC D D
1,2,3,7 ,8 ,9 -H xC D D
1,2,3,6 ,7 ,8 -H xC D D
1 ,2,3,4,6,7,8-H p C D D
1 ,2 ,3 ,4 ,6 ,7 ,8 ,9 -O C D D
1
1
0.1
0.1
0.1
0.01
0.0001
PC D F Congeners
W H O TEF
2 ,3 ,7 ,8 -T C D F
1,2,3,7,8-P eC D F
2,3,4,7,8-P eC D F
1 ,2 ,3 ,4 ,7 ,8 -H x C D F
1 ,2 ,3 ,7 ,8 ,9 -H x C D F
1 ,2 ,3 ,6 ,7 ,8 -H x C D F
2 ,3 ,4 ,6 ,7 ,8 -H x C D F
1 ,2 ,3 ,4 ,6 ,7 ,8 -H p C D F
1 ,2 ,3 ,4 ,7 ,8 ,9 -H p C D F
1 ,2 ,3 ,4 ,6 ,7 ,8 ,9 -O C D F
0.1
0.05
0.5
0.1
0.1
0.1
0.1
0.01
0.01
0.0001
PC B Congeners
W H O TEF
IUPAC N um ber
Structure
77
81
105
114
118
123
126
156
157
167
169
189
3 ,3 ',4 ,4 '-T C B
3,4,4',5-T C B
2,3 ,3 ',4 ,4 '-P eC B
2,3,4,4',5-P eC B
2 ,3 ’,4,4',5-P eC B
2',3 ,4 ,4 ',5 -P eC B
3,3',4,4',5-P eC B
2 ,3 ,3 ',4 ,4 ',5 -H x C B
2 ,3 ,3 ',4 ,4 ',5 '-H x C B
2 ,3 ',4 ,4 ',5 ,5 '-H x C B
3 ,3 ',4 ,4 ',5 ,5 '-H x C B
2 ,3 ,3 ',4 ,4 ',5 ,5 '-H p C B
0.0001
0.0001
0.0001
0.0005
0.0001
0.0001
0.1
0.0005
0.0005
0.00001
0.01
0.0001
Abbreviations: PeCDD, pentachlorodibenzo-p-dioxin; H xCD D , hexachlorodibenzo-p-dioxin;
H pCD D , heptachlorodibenzo-p-dioxin; O CD D , octachlorodibenzo-p-dioxin; TCD F, tetrachlorodibenzofuran; PeCDF, pentachlorodibenzofuran; H xC D F, hexachlorodibenzofuran;
H pCDF, heptachlorodibenzofuran; O CD F, octachlorodibenzofuran; TCB, tetrachlorobiphe
nyl; PeCB, pentachlorobiphenyl; H xCB, hexachlorobiphenyal; H pCB, heptachlorobiphenyl.
SOURCE: IPCS 1998a. Reprinted with permission; copyright 1998, World Health Organization.
Copyright © National Academy of Sciences. All rights reserved.
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36
HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
forest fires and open burning of waste materials, and high-temperature
processes (e.g., cement kiln operations). Combustion sources produce
PCDDs, PCDFs, and limited amounts of PCBs (commercially manufactured
in large quantities from about 1930 until 1977). Metallurgical operations
(e.g., iron ore sintering, steel production, and scrap metal recovery) can
produce PCDDs and PCDFs, which are also formed as by-products of
chemical processing (e.g., manufacture of chlorine-bleached wood pulp and
phenoxy herbicides). PCDDs and PCDFs can also be formed under such
environmental conditions as composting via microorganism action on chlo
rinated phenolic compounds. Studies have also reported that these chemi
cals form during photolysis of highly chlorinated phenols, such as pentachlorophenol, although it has been demonstrated only under laboratory
conditions. Four of the source categories (combustion, metallurgical pro
cessing, chemical manufacturing and processing, and biological and photo
chemical processes) are collectively referred to as contemporary formation
sources. In contrast, reservoir sources are not considered in the quantitative
inventory of contemporary formation sources because they involve the re
circulation of previously formed compounds that have already partitioned
into air, water, soil, sediment, and biota. However, the Reassessment recog
nizes that the contribution of reservoir sources to human exposure may be
significant, perhaps contributing half or more of total background TEQ
exposure. For any given time period, releases from both contemporary
formation sources and from reservoir sources determine the overall amount
of TCDD, other dioxins, and DLCs released to the accessible environment.
The Reassessment gives an inventory of environmental releases of
PCDDs, PCDFs, and TCDD-like PCBs for the United States based on two
reference years, 1987 and 1995. An updated inventory for reference year
2000 was published in 2005 and was included in the committee’s review.
EPA’s best estimate of releases of these compounds to air, water, and land
from reasonably quantifiable sources in 2000 was approximately 1,500 g
TEQdf-WHO ([DF] dioxins and furans), representing an 89% decrease
from a 1987 best estimate of 14,000 g TEQ df-WHO. U.S. environmental
releases of PCDDs and PCDFs occur from an expansive variety of sources
but are dominated by releases to the air from combustion sources. The
decrease in estimated releases of PCDDs and PCDFs from 1987 to 2000 is
largely attributed to reductions in air emissions from municipal and medi
cal waste incinerators; further reductions are anticipated. Three types of
combustion sources contributed approximately 70% of all quantifiable
environmental releases in 1995: municipal waste incinerators, backyard
burning of refuse in barrels, and medical waste incinerators, representing
38%, 19%, and 14% of total environmental releases, respectively. A num
ber of investigators have proposed that the U.S. inventory underestimates
releases from contemporary formation sources partly because of the lack of
Copyright © National Academy of Sciences. All rights reserved.
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INTRODUCTION
37
sufficient data from sources that can emit TCDD and related compounds,
such as land fires; unquantifiable or poorly quantifiable sources, such as
agricultural burning; and the possibility of unknown sources. Additional
observations in the Reassessment regarding sources are concerns about
insufficient data or estimates from nonpoint sources (e.g., urban stormwater
runoff and rural soil erosion) and the likelihood that total nonpoint-source
releases are substantially larger than point-source releases. Evidence also
indicates that current emissions of TCDD, other dioxins, and DLCs to the
U.S. environment result principally from anthropogenic activities, as
supported by correlations in the rise in environmental levels of these com
pounds and a period of rapid increase in industrial activities, lack of signifi
cant natural sources, and observations of higher body burdens in industri
alized versus less industrialized countries. PCDDs, PCDFs, and PCBs share
similar properties, including lipophilicity, hydrophobicity, and resistance to
degradation. Consequently, these intrinsically stable compounds are found
throughout the world in practically all environmental media, including air,
water, soil, sediment, food, and food products. The amount of time re
quired for a chemical to lose one-half of its original concentration, known
as its half-life, varies by substance. The chemical half-lives of mixtures
change with time, as the shorter-lived substances disappear and the propor
tion with longer half-lives increases. (For further discussion on chemical
half-lives, see commentary (Part II, Volume 2, Chapter 2).
The Reassessment defines background exposure to TCDD, other diox
ins, and DLCs as exposure that would occur in an area without known
point sources of the contaminants. Background exposure includes exposure
via the commercial food supply, air, or soil but not any significant occupa
tional exposure. Background exposure estimates are based on the monitor
ing of data from environmental sites and other media void of known con
taminant sources and on pharmacokinetic models using body burden data
from nonoccupationally exposed populations. High concentrations, mea
sured in parts per trillion (ppt) and higher, are found in soil, sediments, and
biota because of their recalcitrant nature and their physical-chemical prop
erties. Low concentrations, measured in parts per quadrillion (ppq) and
picograms per cubic meter (pg/m3), are found in water and air, respectively.
Estimates for background concentrations of DLCs in environmental
media and in food are based on studies conducted at various locations in
North America. The number of locations examined for environmental me
dia estimates in those studies was small, and it is not known whether the
estimates adequately reflect the full range of variation across the United
States. Food estimates were derived from statistically based national sur
veys, nationwide sampling networks, food fat concentration measurements,
samples collected from retail stores, and samples obtained from biohabitat.
PCDD, PCDF, and PCB TEQ-WHO concentrations in environmental
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38
HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
media and food are presented in Table 1-2. Measurable quantities of these
in environmental media and food in the United States were found to be
similar to quantities measured in Europe. Evidence from Europe suggests a
decline in dioxin and furan concentrations in food products during the
1990s. Although no systematic study of temporal trends in dioxin concen
trations in food has been conducted in the United States, at least one study
determined that meat now contains lower concentrations of TCDD, other
dioxins, and DLCs than samples from the 1950s through the 1970s con
tained. The U.S. Department of Agriculture is conducting a nationwide
survey of dioxin concentrations in beef, pork, and poultry that should
allow for a time-trend analysis.
The average PCDD, PCDF, and PCB tissue concentration for the gen
eral adult U.S. population in the late 1990s, based on EPA’s estimate, was
25 ppt TEQdfp-WHO ([DFP] dioxins, furans, and PCBs), lipid basis (Reas
sessment, Part III, p. 4-15). This estimate suggests average tissue concentra
tions have declined from the estimated 55 ppt in the late 1980s and early
1990s. Because new emissions of TCDD, other dioxins, and DLCs have
been declining since the 1970s, it is reasonable to expect that concentra
tions in food, human diet, and, ultimately, human tissue have also declined
during this time.
The Reassessment acknowledges that characterization of national back
ground concentrations of TCDD, other dioxins, and DLCs in tissue is
uncertain because current data are not statistically representative of general
populations. Also, tissue concentrations are a function of age and year of
birth.
HEALTH EFFECTS
On a global scale, exposure to TCDD, other dioxins, and DLCs result
ing from accidental, occupational, or incidental exposure through dermal
contact, inhalation, or ingestion has been associated with adverse effects on
human health. In the early 1900s, workers involved in distilling, processing,
or producing chlorine-based chemicals presented with symptoms character
istic of those currently associated with TCDD poisoning, including severe
cases of chloracne and various degrees of fatigue. Soil contaminated with
TCDD at 300 ppb caused the 1983 evacuation of the town of Times Beach,
Missouri, and allegedly was responsible for the deaths of local animals and
for a variety of human and animal illnesses. In a January 2003 press release,
the Institute of Medicine announced that reexamination of six studies of
herbicide-exposed veterans revealed sufficient evidence of an association
between herbicide defoliants, or their contaminants, which included TCDD,
sprayed by U.S. forces in Vietnam and the risk of developing chloracne,
chronic lymphocytic leukemia, and soft tissue sarcoma.
Copyright © National Academy of Sciences. All rights reserved.
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INTRODUCTION
39
Some studies suggest that exposure of chemical workers to very high
concentrations of TCDD, other dioxins, and/or PCBs (body burdens of
100-1,000 times background) are associated with an increased incidence of
cancer (Flesch- Janys et al. 1995; Hooiveld et al. 1998; Steenland et al.
1999). Other studies of highly exposed populations suggest that TCDD,
other dioxins, and PCBs can have reproductive and developmental effects
(Eskenazi et al. 2000; Kogevinas 2001; Revich 2002; Vreugdenhill et al.
2002a; Pesatori et al. 2003). The long-term effects of low-level exposure to
TCDD, other dioxins, or DLCs normally experienced by the general popu
lation are not known, nor is the clinical significance of biochemical
biomarkers, such as enzyme induction at or near background-level expo
sures. Focal points of research include organ and organ-system effects and
elucidation of the cellular mechanisms through which these effects occur.
COMMITTEE CHARGE AND RESPONSE
In May 2004, EPA asked the NRC to review the revised draft reassess
ment titled Exposure and Human Health Reassessment of 2,3,7,8Tetrachlorodibenzo-p-dioxin (TCDD) and Related Compounds (2003 ver
sion, publicly released in October 2004) and to assess whether EPA’s risk
estimates are scientifically robust and whether there is a clear delineation of
all substantial uncertainties and variability (see Box 1-1 for the complete
statement of task). In response, the NRC formed the Committee on EPA’s
Exposure and Human Health Reassessment of TCDD and Related Com
pounds, a panel of 18 members that included experts in exposure assess
ment; food exposure pathways; pharmacokinetics; physiologically based
pharmacokinetic modeling; benchmark dose modeling; dose-response mod
eling; molecular and cellular aspects of receptor-mediated responses; toxi
cology with specialties in cancer, reproduction, development, and immu
nology; epidemiology; reproductive physiology and medicine; pediatric
biology and medicine; statistics; risk assessment (both qualitative and quan
titative); and uncertainty analysis (see Appendix A for details).
The committee held three public meetings in Washington, DC, to col
lect information, meet with researchers and decision makers, and accept
testimony from the public. The committee met two additional times, in
executive session, to complete its report. Although the committee reviewed
all three parts of the Reassessment, it focused primarily on Part III—
Dioxin: Integrated Summary and Risk Characterization for 2,3,7,8-Tetrachlorodibenzo-p-Dioxin (TCDD) and Related Compounds, as directed by
the study charge. The committee also considered new peer-reviewed studies
published since Part III of the Reassessment was last revised and before the
committee held its final meeting in July 2005. However, because the com
mittee was charged to review EPA’s Reassessment, conducting a compre-
Copyright © National Academy of Sciences. All rights reserved.
Copyright © National Academy of Sciences. All rights reserved.
Media
PCDDs, PCDFs"7
References^
PCBs"7
References^
Mean Total
PCDD,
PCDF, PCBs
Urban soil, ppt
n = 270
9.3 ± 10.2"
Range = 2 to 21
EPA 1985, 1996a, 2000a;
Nestrick et al. 1986;
Birmingham 1990;
Pearson et al. 1990 ; NIH
1995; Rogowski et al.
1999
n = 99
2.3
EPA 2000a
11.6
Rural soil, ppt
n = 354
2.7"
Range = 0.11 to 5.7
EPA 1985, 1996a, 2000a;
Birmingham 1990;
Pearson et al. 1990; Reed
et al. 1990; MRI 1992;
van Oostdam and Ward
1995; Tewhey Assoc. 1997;
Rogowski et al. 1999;
Rogowski and Yake 1999
n = 62
0.59
EPA 2000a
3.3
Sediment, ppt
n = 11
5.3 ± 5.8"
Range = <1 to 20
Cleverly et al. 1996
n = 11
0.53 ± 0.69c
Cleverly et al. 1996
5.8
Urban air, pg/nC
n = 106
0.12 ± 0.094"
Range = 0.03 to 0.2
CDEP 1988, 1995; Hunt
et al. 1990; Hunt and
Maisel 1990; Maisel and
Hunt 1990; OHEPA 1995;
Smith et al. 1989, 1990
n = 53
0.0009e1
H off et al. 1992
0.12
Health Risks from Dioxin and Related Compounds: Evaluation of the EP A Reassessm ent
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O
TABLE 1-2 Summary of North American PCDD, PCDF, and PCB TEQ WHO Concentrations in Environmental
Media and Food (Whole Weight Basis)
Copyright © National Academy of Sciences. All rights reserved.
Rural air, pg/m^
n = 60
0.013"
Range = 0.004 to 0.02
CDEP 1995; OHEPA
1995; Cleverly et al. 2000
Freshwater fish
and shellfish, ppt
n = 222
1.0"
EPA 1992; Fiedler et al.
1997; Jensen et al. 2000;
Jensen and Bolger 2001
Marine fish and
shellfish, ppt
n = 158
0.26"
Fiedler et al. 1997; Jensen
et al. 2000
Water, ppq
n = 236
0.00056 ± 0.00079
Meyer et al. 1989 ; Jobb
et al. 1990
Milk, ppt
n = 8 composites
0.018/
Lorber et al. 1998
Dairy, ppt
n = 8 composites
0.12/
Based on data from
Lorber et al. 1998
Eggs, ppt
n = 15 composites
0.081/
Hayward and Bolger 2000
Beef, ppt
n = 63
0.18 ± 0.11
Range = 0.11 to 0.95
Winters et al. 1996a
Cleverly et al. 2000
0.014
n = 1 composite
of 10 samples
plus 6
composites of
1.2 e’f
Mes and Weber
1989; Mes et al.
1991, Schecter
et al. 1997
2.2
n = 1 composite
of 13 samples
plus 6 composites
0.2 5e’f
Mes et al. 1991;
Schecter et al.
1997
0.57
s
—
0.00056
n = 8 composites
0.0088
Lorber et al. 1998
0.027
n = 8 composites
0.058
Based on data from
Lorber et al. 1998
0.18
n = 1 8 plus 6
composites of
0.10e^
Mes and Weber
1989; Mes et al.
1991; Schecter et.
1997
0.13
n = 63
0.084
Winters et al.
1996b
0.26
continued
Health Risks from Dioxin and Related Compounds: Evaluation of the EP A Reassessm ent
http://www.nap.edU/catalog/11688.html
n = 53
0.00071
-k
h~i
K>
Copyright © National Academy of Sciences. All rights reserved.
Media
PCDDs, PCDFsJ
References^
PCBsJ
References^
Mean Total
PCDD,
PCDF, PCBs
Pork, ppt
n = 78
0.28 ± 0.28
Range = 0.15 to 1.8
Lorber et al. 1997
n = 78
0.012
Lorber et al. 1997
0.29
Poultry, ppt
n = 78
0.068 ± 0.070
Range 0.03 to 0.43
Ferrano et al. 1997
n = 78
0.026
Ferrario et al. 1997
0.094
Vegetable fats, ppt
n = 30
0.056 ± 0.24,;
Versar 1996
n = 5 composites
0.037f
Mes et al. 1991
0.093
^Values are the arithmetic mean TEQs, in ppt, and standard deviations. Nondetects were set to one-half the limit of detection, except for soil,
PCDDs, and PCDFs in vegetable fats for which nondetects were set to zero.
full list of references is found in Part I, Vol. 2, Chapter 3 of the Reassessment NAS review draft (December 2003).
T h e values for environmental media are means of the data but lack the spatial representativeness to be considered true national means.
T>ased on data from Canadian air, as reported by Hoff et al. (1992). Not used in U.S. background exposure estimates in Part I, Vol. 2, Chapter 4 of
the Reassessment NAS review draft (December 2003).
T h e values for fish lack the statistical significance to be considered true means; the values of the other food groups were derived from statistically
based surveys and can be considered true national means. The PCCD and PCDF concentrations are species-specific ingestion-weighted average
values.
/Standard deviations could not be calculated because of limitations of the data (composite analyses).
^Congener-specific PCB data are sparse.
¿TEQ calculated from Versar (1996) by setting nondetects to zero.
SOURCE: EPA 2003a.
Health Risks from Dioxin and Related Compounds: Evaluation of the EP A Reassessm ent
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-k
TABLE 1-2 Continued
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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INTRODUCTION
43
BOX 1-1 Statement of Task
The National Academies' National Research Council will convene an expert com
mittee that will review EPA's 2003 draft reassessment of the risks of dioxins and
dioxin-like compounds to assess whether EPA's risk estimates are scientifically
robust and whether there is a clear delineation of all substantial uncertainties and
variability. To the extent possible, the review will focus on EPA's modeling as
sumptions, including those associated with the dose-response curve and points of
departure; dose ranges and associated likelihood estimates for identified human
health outcomes; EPA's quantitative uncertainty analysis; EPA's selection of stud
ies as a basis for its assessments; and gaps in scientific knowledge. The study will
also address the following aspects of the EPA reassessment: (1) the scientific
evidence for classifying dioxin as a human carcinogen; and (2) the validity of the
non-threshold linear dose-response model and the cancer slope factor calculated
by EPA through the use of this model. The committee will also provide scientific
judgment regarding the usefulness of toxicity equivalence factors (TEFs) in the risk
assessment of complex mixtures of dioxins and the uncertainties associated with
the use of TEFs. The committee will also review the uncertainty associated with
the reassessment's approach regarding the analysis of food sampling and human
dietary intake data, and, therefore, human exposures, taking into consideration the
Institute of Medicine's report Dioxin and Dioxin-Like Compounds in the Food Sup
ply: Strategies to Decrease Exposure. The committee will focus particularly on the
risk characterization section of EPA's reassessment report and will endeavor to
make the uncertainties in such risk assessments more fully understood by deci
sion makers. The committee will review the breadth of the uncertainty and variabil
ity associated with risk assessment decisions and numerical choices, for example,
modeling assumptions, including those associated with the dose-response curve
and points of departure. The committee will also review quantitative uncertainty
analyses, as feasible and appropriate. The committee will identify gaps in scientific
knowledge that are critical to understanding dioxin reassessment.
hensive and thorough review of all TCDD-related materials published since
2003, reassessing TEF values, and re-creating the risk assessment were
outside of the scope of the statement of task.
The present report is the product of the efforts of the entire NRC
committee and underwent extensive, independent, external review overseen
by the N R C’s Report Review Committee. It specifically addresses and is
limited to the statement of task as agreed upon by the NRC and EPA.
The remaining chapters of this report comprise the findings of the
Committee on EPA’s Exposure and Human Health Reassessment of TCDD
and Related Compounds. Chapter 2 provides conceptual text on how to
address variability and uncertainty in risk assessment. Chapter 3 evaluates
the usefulness and uncertainties of TEFs in the risk assessment of complex
mixtures of TCDD, other dioxins, and DLCs and discusses various ap-
Copyright © National Academy of Sciences. All rights reserved.
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44
HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
proaches to dose metrics. Chapter 4 addresses exposure characterization in
terms of sources, environmental fate, environmental media concentrations,
food concentrations, background exposures, and potentially highly exposed
populations. Chapter 5 reviews EPA’s assessment of the carcinogenicity of
TCDD other TCDD, other dioxins, and DLCs, including the qualitative
characterization of their carcinogenicity, the validity of the nonthreshold
linear dose-response model, and the use of the animal bioassay and epide
miological data to quantify the dose response. Chapter 6 reviews EPA’s
assessment of noncancer end points, including immune function, reproduc
tion, and development. Chapter 7 focuses on risk characterization. Chapter
8 summarizes the committee’s conclusions and recommendations and suc
cinctly addresses each component of the statement of task.
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2
G en eral C o n sid e ra tio n s o f U n certain ty
a n d V ariability, Selection o f D o se
M e tric , an d D o se -R e sp o n se M o d e lin g
Health risk assessments now typically include discussion of variability
(real differences) and uncertainty (fundamental lack of knowledge) and
often use probabilistic risk assessment methods to characterize variability
and uncertainty in the estimates of risks. Prior National Research Council
(NRC) reports and U.S. Environmental Protection Agency (EPA) docu
ments make clear the need for these characterizations; for example, they
emphasize that
uncertainty forces decision-makers to judge how probable it is that risks
will be overestimated or underestimated for every member of the exposed
population, whereas variability forces them to cope with the certainty that
different individuals will be subjected to risks both above and below any
reference point one chooses (NRC 1994, p. 237)
and that
[i]n successive versions of its cancer guidelines, EPA expressed increasing
emphasis on a full examination of uncertainties, with the recognition that
both qualitative and quantitative approaches to uncertainty assessment
are important and can (applied appropriately) help clarify the nature of
assessment findings. The use of sophisticated uncertainty tools also in
volves substantial issues of science and mathematics, as well as specialized
issues such as the appropriate presentation and characterization of proba
bilistic estimates in the decision making context where appropriate. (EPA
2004a, p. 49)
Significant uncertainties remain in understanding human health risks
from 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD), other dioxins, and di45
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46
HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
oxin-like compounds (DLCs), in spite of very large investments in data
collection and research.
Variability among members of the population is an important consider
ation in understanding risks. Variability results from the wide range of
environmental sources and human interactions with them, as well as from
physiological and genetic differences that might influence the relative sus
ceptibility of humans and other species to adverse health effects from expo
sure. For example, sources of variability associated with human health
outcomes include the inherent genetic diversity of human populations,
which currently remain difficult to address quantitatively. Abundant evi
dence demonstrates complex gene-environment interactions for many com
plex human diseases, immune system dysfunction, and other disorders in
which TCDD, other dioxins, and DLCs might be implicated.
Adding more complexity, the risks from TCDD, other dioxins and
DLCs continue to change over time because of changing exposures, and
understanding of the risks continues to evolve with the collection of more
data. Any assessment reflects the snapshot of the information available at
that time, and analysts should recognize that additional information might
later reveal evidence that differs from prior assumptions.
One of the charges to the committee emphasized reviewing the Reas
sessment1 “to assess whether EPA’s risk estimates are scientifically robust
and whether there is a clear delineation of all substantial uncertainties and
variability.” Risk assessment in the case of TCDD, other dioxins, and DLCs
represents a formidable task because of the size of the available database
and the complexity of numerous issues. EPA collated and presented a mas
sive database on TCDD, other dioxins, and DLCs, on which the committee
commented specifically in the chapters that follow. This chapter identifies
the major categories of decisions that analysts generally make when devel
oping risk estimates in the context of the four traditional steps of risk
assessment: hazard identification and classification, exposure assessment,
dose-response assessment, and risk characterization (NRC 1983). The Re
assessment deals with complexities in the risk assessment of TCDD, other
dioxins and DLCs by making specific choices as described in this chapter,
but EPA could alternatively use a probabilistic approach. Typically, risk
assessments should address uncertainties that derive from conceptuali
zations and fundamental choices among competing options in a way that
clearly identifies the quantitative impacts of alternatives. When there are
two or more plausible interpretations, a risk assessment should make clear
^-The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
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GENERAL CONSIDERATIONS
47
that such alternatives give rise to uncertainty. To this end, a risk assessment
should identify the key uncertainties (those that drive the risk estimates)
and make clear how selection of specific alternative assumptions influences
the risk assessment results.
In general, the choice of individual or population risk metric that is
modeled influences the appropriate characterization of variability and un
certainty in risk (Thompson and Graham 1996). The Reassessment strives
to present a comprehensive baseline risk assessment intended to cover all
potential sources. This generic approach results in limited discussions of
variability and uncertainty. The committee found that the lack of a specific
context and absence of a focused exposure assessment that would link
sources to potential health effects in individuals, or in the population,
severely limited both EPA’s and the committee’s abilities to appropriately
characterize variability and uncertainty in risk estimates related to exposure
to TCDD, other dioxins, and DLCs.
HAZARD CLASSIFICATION
In the context of the Reassessment, EPA faced the decisions of assign
ing a hazard classification for TCDD, and for other dioxins and DLCs,
including mixtures. Hazard classification typically focuses on characteriz
ing the weight of the evidence with respect to potential health effects. For
cancer risk, the cancer guidelines (EPA 2005a, also see Appendix B) outline
specific criteria for classifying substances into the following categories:
1. Carcinogenic to humans
2. Likely to be carcinogenic to humans
3. Suggestive evidence of carcinogenic potential
4. Inadequate evidence to assess carcinogenic potential
5. Not likely to be carcinogenic to humans
The charge to the committee stated that it should address “ the scientific
evidence for classifying dioxin as a human carcinogen.”
The committee believes that the scientific evidence on cancer causation
usually falls within a continuum, and classification often artificially places
apparent bright lines (e.g., in distinguishing a “known human carcinogen”
from a “likely human carcinogen” ). In Chapter 5, the committee reviews
and comments on EPA’s decisions with respect to its determinations of
cancer classification.
With respect to noncancer end points, the committee notes that EPA
does not use a rigorous approach for evaluating evidence from studies and
the weight of their evidence in the Reassessment. The committee finds that
EPA’s lack of systematic evaluation and classification of the noncancer
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
evidence left significant ambiguity about the basis for some of EPA’s deci
sions implied in the report (e.g., the decision not to identify a critical effect
or to develop a reference dose [RfD]). The Reassessment provides an exten
sive catalog of studies but does not synthesize the significant insights or
provide clear assessments of the key uncertainties in a way that allows the
reader to determine the impact of various choices made.
In general, the use of a rigorous evaluation process for noncancer haz
ards would lead to improved characterization of noncancer risks. In the
context of the Reassessment and any future iterations of this analysis, the
committee suggests that EPA focus its efforts on improving its quantitative
characterization of the risks, including noncancer risks, and not devote
substantial effort to further carcinogen classification for TCDD, other di
oxins, and DLCs, as discussed in Chapter 5.
EXPOSURE ASSESSMENT
EPA provided the committee with an updated exposure inventory (EPA
2005b), which provides an extensive review of the existing database of
exposure data for TCDD, other dioxins, and DLCs. The review also pro
vides a useful qualitative review of the level of confidence in the data for
various sources, although the Reassessment does not quantitatively charac
terize the uncertainty associated with low-confidence data. Although the
Reassessment (Part III, p. 4-6) specifically mentions the possibility of un
known sources causing underestimation of releases from contemporary
sources, it does not attempt to correct the incomplete accounting of sources
in historical data or adjust current data to address anticipated discoveries of
other sources. Thus, EPA implicitly assumed that the exposure assessment
sufficiently captures the exposure sources so that any additional new sources
identified would not significantly alter its estimates. The committee dis
cusses this choice in more detail in Chapter 4 and suggests additional
analyses that might further explore the impacts of this assumption.
The updated exposure inventory devotes considerable attention to docu
menting how the nature and magnitude of dominant exposure sources
changed over time. The substantial amount of new evidence of significant
declines in measured concentrations of TCDD, other dioxins, and DLCs
over the past several decades reflects EPA’s specific management efforts
targeted at reducing exposure from some sources (e.g., pulp and paper
mills, medical and municipal waste incineration, and ball clay2 ). Referring
2The term ball clay originated from an early English mining practice of rolling the highly
plastic clay into balls weighing 30 to 50 lb. Ball-clay uses historically included serving as a
supplement in animal feeds (as in chicken feed). In 1996, as a result of investigations into the
source of contamination with T C D D and other dioxins in chicken fat, investigators measured
relatively high levels of TC D D and related compounds in ball clay (FDA 1997).
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GENERAL CONSIDERATIONS
49
specifically to TCDD, EPA notes that “ dioxin levels in the environment
have been declining since the 1970s..., and it is reasonable to expect that
levels in food, human intake, and ultimately, human tissue have also de
clined over this period. The changes in tissue levels are likely to lag the
decline seen in environmental levels, and the changes in tissue levels cannot
be assumed to occur proportionally with declines in environmental levels”
(Reassessment, Part III, p. 4-16). Changing concentrations in the environ
ment over time provides another substantial uncertainty in risk assessment,
because EPA must decide whether to use specific “ snapshot-in-time” con
centrations for risk assessment or whether to extrapolate or average such
changing concentrations over time. Given the timing of the updated expo
sure inventory, it was not clear to the committee how EPA intends to use
the updated inventory information in the context of estimating current
exposures.
Another area of uncertainty lies in determining what constitutes back
ground exposures in the general population. EPA carefully defines “ back
ground” in a prominent footnote (Reassessment, Part III, p. 1-1), and the
committee concurs that this approach is appropriate and is clearly pre
sented in the Reassessment. However, the uncertainty associated with po
tential discoveries of “new sources” will remain an issue that EPA may need
to analyze further. For example, the Reassessment added a chapter on ball
clays in the latest iteration.
Yet another area of uncertainty is determination of background levels
when many samples lie below the analytical limit of detection. This issue
arises in any exposure assessment, and several widely used options address
it (e.g., assume all nondetects are true zeroes, assign a value of either V2 or
1 times the detection limit, or fit a distribution to the data). The committee
noted that EPA did not pick a single consistent approach (see the note to the
summary table at the bottom of Part III, p. 4-32) or provide a clear quanti
tative indication of the importance of the choice of strategy for dealing with
nondetects, which creates inconsistencies in the Reassessment. The commit
tee recommends that EPA clearly and quantitatively explore how different
strategies for dealing with nondetects affect exposure assessment results, as
discussed in Chapter 4. If these alternative approaches produce very differ
ent results, then EPA should further consider the implications of specific
options.
Another major source of uncertainty stems from the selection of a dose
metric. The Reassessment could provide exposure estimates for a wide
range of dose metrics and averaging times to support the spectrum of
possible dose-response assessment choices. This important issue is discussed
in more detail below. The Reassessment also provides little insight about
bioavailability, an issue that frequently falls between the domains of the
exposure assessment and dose-response assessment.
Finally, the Reassessment provides very little information about the
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
amount of individual variability in exposure. EPA describes how average
daily toxic equivalent quotient (TEQ) varies as a function of age (Reassess
ment, Part III, pp. 4-16, 4-17, and 4-35), although it does not provide a
measure of the variability around these estimates (that is, the population
distribution of exposures within each age group). EPA’s description may
implicitly give the impression of very limited variability within the popula
tion, which may not be the case. However, the Reassessment provides some
good examples of other parameters that may influence interindividual vari
ability. For example, considering the variability in total fat consumption,
the Reassessment suggests that TCDD intakes in the general population
could extend to levels at least three times higher than the mean (Reassess
ment, Part III, p. 4-19). The exposure assessment also demonstrates that
TCDD intake for children based on age-specific food consumption and
average food concentrations exceeds adult intake estimates on a bodyweight basis (although their intake on a mass basis is lower) (Reassessment,
Part III, p. 4-35). These examples also illustrate the difficulties that arise in
choosing an appropriate overall averaging time for exposure.
ASSESSMENT OF OTHER DIOXINS AND DLCS
The challenge of characterizing the risks from complex mixtures also
leads to important choices. EPA’s use of a TEQ approach represents the
prevailing strategy (in the United States and internationally). In Chapter 3,
the committee provides an in-depth evaluation of EPA’s use of toxic equiva
lency factors (TEFs) and TEQs. This issue also represents an important area
of uncertainty in the overall risk assessment. The Reassessment states that
“ despite the uncertainties in the TEF methodology, the use of this method
ology decreases the overall uncertainty of the risk assessment” (Reassess
ment, Part III, p. 1-10). Although that may be true, EPA should quantita
tively support the argument with some comparisons or data. The
Reassessment also notes that “ TEFs are the result of scientific judgment of
a panel of experts who used all of the available data, and they are selected
to account for uncertainties in the available data and to avoid underesti
mating risk. In this sense, they can be described as public-health conserva
tive values” (Reassessment, Part III, p. 1-5). The committee recommends
that EPA quantify the extent to which the TEF estimation process may be
health protective. In addition, because TEFs continue to evolve (see Chap
ter 3), EPA must continue to choose which TEF values to use and which
congeners to include. Such choices will influence exposure estimates as well
as the uncertainties associated with those estimates.
The Reassessment acknowledges the difficulty of comparing different
human-exposure data sets because some do not include coplanar polychlo
rinated biphenyls in the estimation of TEQ values. The Reassessment clearly
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GENERAL CONSIDERATIONS
51
states that TCDD per se is not the main contributor to TEQ levels in human
lipids (Part III, Table 4-5). The Reassessment uses the calculation of body
burden at steady state, its associated assumptions given in the Reassessment
(Part III, section 1.3), best estimates of current adult intakes, and the as
sumption of 25% body fat to calculate the TEQ concentration in human
lipids. The resulting estimate is about one-half the level currently measured
in human lipids. The Reassessment suggests that this discrepancy arises
from the presence of an historical body burden and lipid concentration, but
it does not consider other possibilities.
GENERAL ISSUES RELATED TO VARIABILITY AND
UNCERTAINTY ASSOCIATED WITH SELECTION OF DOSE
M ETRIC AND DOSE-RESPONSE MODELING
EPA makes a number of assumptions about the appropriate dose met
ric and mathematical functions to use in the Reassessment’s dose-response
analysis (see “ Selection of Dose Metric” and “Dose-Response Modeling” in
this chapter for specific issues related to dose metric and dose-response
modeling). The Reassessment does not adequately comment on the extent
to which each of these assumptions could affect the resulting risk estimates.
EPA discussed various dose metrics and selected one particular metric
based on its judgment. However, EPA did not quantitatively describe how
this particular selection affected its estimates of exposure and therefore
provided no overall quantitative perspective on the relative importance of
the selection.
EPA faced numerous choices with respect to developing quantitative
models for characterizing cancer risk from exposure to TCDD, other diox
ins, and DLCs (summarized in Table 2-1) and for characterizing noncancer
effects (summarized in Table 2-2). The Reassessment characterizes the risk
of cancer at background and incremental intakes by using a cancer slope
factor (CSF), and it recommends the use of a margin of exposure (MOE) for
both noncancer and cancer end points (Reassessment, Part III, p. 6-12). The
committee did not find EPA’s justification sufficient for why it used differ
ent methods to characterize risk for end points that have the same basic
underlying mode of action. The committee noted that the Reassessment
should also quantitatively characterize the impact of this choice.
The Reassessment concludes that setting an RfD is not appropriate
because of the relatively high background levels compared with effect levels
and suggests that setting an RfD provides little value for evaluating possible
risk management options if average background exposure exceeds the RfD
(Reassessment, Part III, p. 6-14). As discussed in Chapter 7, this decision
conflicts with the choices made by other international regulatory bodies
(e.g., European Scientific Committee on Food, Food and Agricultural Orga-
Copyright © National Academy of Sciences. All rights reserved.
K)
Copyright © National Academy of Sciences. All rights reserved.
TABLE 2-1 Categories of Key Decisions EPA Faced in Characterizing Cancer Risk
Basis for
Quantification
Epidemiological
Data Set
Bioassay Data Set
Dose-Response
Model
Dose Metric
•
•
•
•
•
•
•
•
Epidemiological
and bioassay data
Epidemiological
data
Bioassay data
Other
•
Choose from
individual
studies
Use multiple
studies
•
Choose from
individual studies
Use multiple
studies
•
•
•
Low-dose
linear
Nonlinear
Multiple
Other
•
•
•
•
Abbreviations: ED, effective dose; LED, lower confidence limit on ED.
Average daily
dose
Area under the
curve
Lifetime average
body burden
Peak
Other
Point of
Departure
•
•
•
•
•
e d 0i
ED o s
ED io
LED0 i
Other
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TABLE 2-2 Categories of Key Decisions EPA Faced in Characterizing Noncancer Risk
Copyright © National Academy of Sciences. All rights reserved.
Basis for Quantification
Epidemiological Data Set
Bioassay Data Set
POD
•
•
•
•
•
•
•
•
•
•
•
•
•
Epidemiological and
bioassay data
Epidemiological data
Bioassay data
Other
Choose from individual
studies
Choose from
individual studies
Dose
Metric
LOAEL
NOAEL
ED0 f
e d 05
EDfo
BMD
Other
•
•
•
•
•
BB
ADD
AUC
Peak
Other
Critical Effect Choice
•
Reproductive and
developmental
Immunotoxicity
Neurotoxicity
Central nervous
system
Diabetes
Enzymatic change
Other
•
•
•
•
•
•
Additional Categories
Exposure
Route
•
•
•
•
Ingestion
Inhalation
Multiple
Other
Exposure Time
•
Depends on individual studies
Type of
Dosing
DRD
U.F. (Database)
U.F. (Interspecies)
U.F. (Intraspecies)
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
•
Single
Multiple
Yes
No
•
10
3
1
Chemical-specific
adjustment factor
Other
•
10
3
1
Chemical-specific
adjustment factor
Other
•
10
3
1
Chemical-specific
adjustment factor
Other
Abbreviations: POD, point of departure; LOAEL, lowest-observed-adverse-effect level; NOAEL, no-observed-adverse-effect level; ED, effective dose;
BMD, benchmark dose; BB, body burden; ADD, average daily dose; AUC, area under curve; DRD, develop reference dose; U.F., uncertainty factor.
■ -o
O
o
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54
HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
nization of the United Nations [FAO]/World Health Organization [WHO],
and the Joint Expert Committee on Food Additives [JEFCA]). EPA’s deci
sion not to specify an RfD in the Reassessment may have depended on the
set of specific assumptions it selected, such as use of the 1% effective dose
(ED01) as the point of departure (POD) for this calculation and the magni
tude of the applicable uncertainty factors.
The Reassessment provides a thorough statement of the potential
sources of uncertainty for consideration in noncancer risk assessment, many
of which also apply in the context of cancer risk assessment:
Consideration should be given to a number of difficulties and uncertainties
associated with comparing the same or different endpoints across species,
such as differences in sensitivity of endpoints, times of exposure, exposure
routes, and species and strains; the use of multiple or single doses; and
variability between studies even for the same response. The estimated ED01s
may be influenced by experimental design, suggesting caution should be
used when comparing values from different designs. Caution should also be
used when comparing studies that extrapolate ED01s outside the experi
mental range. Furthermore, it may be difficult to compare values across
endpoints. For example, the human health risk for a 1% change of body
weight may not be equivalent to a 1% change in enzyme activity. Similarly,
a 1% change in response in a population for a dichotomous endpoint is
different from a 1% change in a continuous endpoint, where the upper
bound of possible values may be very large, leading to a proportional in
crease in what constitutes the 1% effect level. Finally, background expo
sures are often not considered in these calculations simply because they
were not known. (Reassessment, Part III, p. 5-24)
The Reassessment used empirical, full dose-response modeling to esti
mate PODs, specifically an ED for cancer and noncancer. Historically, a
POD for a noncancer end point was based on a no-observed-adverse-effect
level (NOAEL) or a lowest-observed-adverse-effect-level (LOAEL), a prac
tice inconsistent with cancer risk assessment. EPA now recommends the use
of a benchmark dose (BMD) approach to derive a POD for noncancer end
points. Although a lower confidence bound on an ED was cited in the
literature to define a BMD, EPA’s BMD guidance document (EPA 2000b)
defines the ED, BMD, and the lower one-sided confidence limit on the
BMD (BMDL).3 This definition unified the determination of PODs for
3“ BM D is used generically to refer to the benchmark dose approach; in the more specific
cases, BM D ... refer[s] to the central estimates, for example the ED x ... for dichotomous
endpoints (with x referring to some level of response above background, e.g., 5 % or 1 0 % ).
BM DL ... refers to the corresponding lower limit of a one-sided 95% confidence interval on
the B M D . . ” (EPA 2000b, Executive Summary)
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GENERAL CONSIDERATIONS
55
cancer and noncancer end points. The modeling process generally involves
two steps:
The first step is an analysis of dose and response in the range of observa
tion of the experimental or epidemiologic studies. The modeling yields a
POD near the lower end of the observed range, without significant extrap
olation to lower doses. The second step is extrapolation to lower doses.
The extrapolation approach considers what is known about the agent’s
mode of action. Both linear and nonlinear approaches are available. (EPA
2005a)
This analysis requires making several key decisions, including primarily
(1) determining appropriate types of studies (epidemiological, animal, both,
and other), (2) choosing specific studies and subsets of data (e.g., species
and gender), (3) choosing specific end points for dose-response modeling,
(4) choosing a specific dose metric, (5) choosing model type and form, (6)
selecting the benchmark response (BMR) and POD, and (7) characterizing
uncertainty.
Current EPA practice generally relies on choosing to model a single
data set, specifically the one that tends to show the most significant poten
tial adverse effect. This choice can introduce substantial uncertainty into
the risk estimation process, particularly in cases in which different data sets
yield very different results. One way to avoid the uncertainty introduced by
the selection of a single data set is to use multiple data sets. In particular,
EPA could place some weight on each of a number of data sets. Chapters 5
and 6 review EPA’s data set choices made in the Reassessment.
GENERAL ISSUES RELATED TO RISK CHARACTERIZATION
Critical issues related to risk characterization (see Chapter 7) include
the impact of decisions on the information communicated to risk managers
about the magnitude of uncertainties associated with the data used to gen
erate risk estimates. The impact of choices made in the risk assessment
process can be characterized by quantifying the impact of plausible alterna
tive assumptions at critical steps. The risk estimates can be most fully
characterized by performing probabilistic analyses when possible and by
presenting the range of possible risk estimates rather than by reporting the
single point estimates. Risk characterization should provide useful informa
tion to risk managers to help them understand the variability and uncer
tainty in the risk estimates. As further discussed in Chapter 5, the commit
tee understands that quantitatively addressing all sources of uncertainty in
a risk assessment can impose an analytical burden, which may result in
addressing some sources of uncertainty qualitatively. Quantifying the con
tribution of various assumptions to the overall uncertainty often proceeds
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
in an iterative manner. The process itself adds value by highlighting oppor
tunities to collect valuable information, and NRC (1994) provides some
guidance about at what point it makes sense to stop in the context of
characterizing risks to inform risk management decisions.
The rationale and scientific basis for important decisions should be
described in the Reassessment and the consequences of alternative assump
tions explored. For dioxin, these issues are best illustrated in relation to the
estimation of cancer risk. The choice of one possible approach, linear ex
trapolation from a POD, results in a CSF that could be used to estimate the
lifetime cancer risk for the U.S. population. Assessing the same epidemio
logical data with a M OE approach would describe the data available to
quantify the POD and exposure but would avoid the scientifically debat
able need to generate a slope factor with its inherent uncertainties (see
Chapter 5 for full discussion of these issues). For noncancer end points, the
hazard characterization data are tabulated, but EPA makes little attempt to
interpret or focus on critical effects or to define the strengths, weaknesses,
and uncertainties associated with effects relevant to critical life stages such
as in utero exposure (see Chapter 6 for full discussion of noncancer end
points).
The reality that the risk assessment process for TCDD, other dioxins,
and DLCs now extends over a period of 14 years, with multiple EPA
reports and iterations of these reports, leads the committee to suggest that
EPA should continue to treat the risk assessment as a process. In this
context, EPA should expect to continue to iterate and improve on the
assessment over time as new information becomes available. However,
instead of producing and continuing to add to massive reports, EPA should
consider a database structure that will allow it to focus its reports on
syntheses of new information that drive the quantitative estimates of risk
rather than on cataloging all information.
In addition, the committee expects that EPA could substantially im
prove its assessment process if it more rigorously evaluated the quality of
each study in the database. As an example, Table 2-3 summarizes one
approach used to describe the basic elements of conducting a systematic
review of scientific evidence. Although EPA performed many of these steps
in its evaluation of the epidemiological literature of carcinogenicity, it did
not outline eligibility requirements or otherwise provide the criteria used to
assess the methodological quality of other included studies. EPA could also
substantially improve the clarity and presentation of the risk assessment
process for TCDD, other dioxins, and DLCs by using a summary table or a
simple summary graphical representation of the key data sets and assump
tions (e.g., using trees like those shown by Evans et al. 1994a,b; Sangrujee
et al. 2003).
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GENERAL CONSIDERATIONS
57
TABLE 2-3 Components of a Systematic Review
•
•
•
•
•
•
•
•
State objectives and hypotheses
Outline eligibility criteria, stating types of study, types of participan ts, types of
interventions and outcom es to be exam ined
Perform a com prehensive search for potentially eligible studies
Decide eligibility and assess m ethodological quality o f included studies
T abulate study characteristics
E xtract data, w ith involvement o f investigators if necessary
Analyse results o f included studies, using statistical synthesis of data (m eta
analysis), if appropriate
Prepare a report o f review, stating aim s, m aterials and m ethods and describing
results and conclusions
SO URCE: Smyth 2000.
SELECTION OF DOSE METRIC
Section 1.3 of the Reassessment Part III considers various dose metrics
for understanding exposure and analyzing dose-response relationships,
which apply to both cancer and noncancer effects. EPA highlights the need
for a pragmatic approach that can be applied to issues of cross-species
scaling and to different end points detected under different exposure sce
narios. Risk assessments for most chemicals typically focus on the external
dose or exposure expressed as mass of substance per kilogram of body
weight per day, but many other options exist. The Reassessment discusses a
number of different dose metrics that represent the internal dose, including
estimates of area under the blood or plasma concentration-time curve
(AUC), plasma or tissue concentrations, body burden, and function-related
biomarkers of the internal dose such as aromatic hydrocarbon receptor
(AHR) occupancy or changes in cytochromes P450A1/2 protein (CYP1A1/
2) activity. The function-related biomarkers are intellectually appealing,
especially for extrapolating from animal to human, because they would
provide a means to address species differences in toxicokinetics and in the
initial events reflecting tissue sensitivity. However, EPA concluded that
insufficient data support the current use of function-related biomarkers in
risk assessment.
The Reassessment (Part III, p. 1-17) suggests that, at the present time,
body burden represents the most suitable dose metric for interspecies com
parisons (similar to the approaches used by other recent evaluations of
TCDD, other dioxins, and DLCs [SCF 2000, 2001; JECFA 2002]), while
lifetime AUC may also be suitable for comparisons of different human
exposures. EPA selected body burden for cross-species comparisons be
cause, “ assuming similar sensitivity between rats and humans at the tissue
level, effective doses should be a function of tissue concentration,” and
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
“tissue concentrations of TCDD and related chemicals are directly related
to the concentration of TCDD in the body” (Part III, p. 1-12).
Chapter 5 discusses the quantitative importance of this assumption in
terms of cancer risk assessment and provides additional discussion of alter
native dose metrics and the relative importance of the choice of dose metric
on ultimate cancer risk projections.
The Reassessment states, “ The steady-state concentration of TCDD in
the body, or steady-state body burden, can be estimated in rats and humans
using the following equation:
Dose (ng TEQ/kg) x t (days) x F
Steady-state body burden = _________________ X
Ln(2)
2-1
where Dose is the daily administered dose, F is the fraction absorbed, and tia
is the species-specific half-life of TCDD” (Reassessment, Part III, p. 1-12).
Body burdens after shorter periods of administration (non-steady state)
would require a different method of estimation.
The Reassessment does not quantitatively explore the impacts of this
choice or the choices of various inputs in the equation (see below) used to
estimate body burden at steady state. The summary table in the Reassess
ment (Part II, Table 1-6) gives limited data for the half-life estimates for
TCDD. Estimates of elimination half-lives for various tissues in rats range
from 11 to 53 days, with the best data coming from eight studies that used
a radiolabeled compound and that reported a range of 12 to 31 days. EPA
uses 25 days to calculate the body burden in rats at steady state. That
appears appropriate to the committee, but this estimate is clearly uncertain.
Similarly, the summary table in the Reassessment (Part II, Table 1-10) gives
limited data on half-life for TCDD in humans. The table provides an esti
mate of 5.8 years based on fecal excretion and 9.7 years based on changes
in adipose concentrations. Data from the Operation Ranch Hand Study
indicated TCDD half-lives of 7.1 (Michalek et al. 1992) and 11.3 years
(Wolfe et al. 1994), the most comprehensive recent analyses indicating a
half-life of 7.6 years (95% confidence interval of 7.0 to 8.2 years) (Michalek
and Tripathi 1999). The Reassessment (Part II, Table 1-13) reports a half
life of 7.2 years for the Flesch-Janys et al. (1996) study. An overall mean
serum TCDD half-life of 8.2 years was reported in 27 victims of the acci
dent in Seveso, Italy (Needham et al. 1994), although a recent study found
substantial interindividual variability and concentration-dependent differ
ences in TCDD half-life (Aylward et al. 2005). Overall the value of 2,593
days (or 7.1 years) used by EPA to calculate the body burden in adult
humans at steady state appears reasonable and realistic. The Reassessment
recognizes that TCDD half-life is shorter in neonates and infants. The
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GENERAL CONSIDERATIONS
59
Reassessment notes that TCDD half-life varies with percent body fat and
increases significantly with a high percent of body fat, suggesting that
people with more body fat tend to eliminate TCDD more slowly. The half
life of TCDD shows a significant correlation with body weight (IOM 2000).
These two pieces of data indicate that human variability in elimination is
related to differences in the apparent volume of distribution as well as
clearance (see below). The values for bioavailability used in the above
equation are also somewhat uncertain. The summary table in the Reassess
ment (Part II, Table 1-1) gives only limited data for TCDD in rats showing
a high bioavailability (70% and 84% in two studies using acetone, corn oil
gavage). The text describes the absorption of 88% of TCDD in male Fischer
344 rats after oral exposure in Emulphor/95% ethanol/water (1:1:3). EPA
assumed 50% absorption from the diet for rats, which appears reasonable
because a range of 50% to 60% absorbed has been reported. The summary
table in the Reassessment (Part II, Table 1-1) gives data from only one study
for TCDD in a human given a single oral dose and gives a bioavailability of
87% (Poiger and Schlatter 1986). Other studies have determined the extent
of absorption by mass balance (the amount ingested minus the amount
eliminated in feces), but such measurements are likely to be unreliable in
adults because elimination of unchanged TCDD in feces is an important
route of elimination of absorbed TCDD in humans. Overall the value pro
posed and used by EPA to calculate the body burden in humans at steady
state (80% absorption) appears reasonable, although the data are limited.
Equation 2-1 implicitly assumes that body burden represents a good
surrogate for tissue concentration and that adverse effects correlate with
steady-state body burden. This assumption represents a reasonable default
because the body burden generally appears to be proportional to tissue
concentration, with some caveats noted in Chapter 5, and the toxic effects
of TCDD, other dioxins, and DLCs increase with increased tissue concen
tration. However, the use of body burden as a dose metric (or a dose metric
based on tissue concentration) would not allow for species differences in
inherent target organ sensitivity to the presence of the chemical. Species
differences in target organ sensitivity could be taken into account by a full
biologically based kinetic-dynamic model, but EPA appropriately concluded
that the available models remain insufficiently well validated for risk assess
ment purposes. The committee did not discuss specific recommendations
for EPA related to collecting data for refining current BBDR models or the
regional induction models, but the committee encourages further develop
ment and use of these models as data become available to validate and
further develop them.
The use of Equation 2-1 implies comparable steady-state tissue concen
trations between species and between individuals simply on the basis of
body burden. Assuming dose linearity, a twofold increase in body burden in
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any individual will yield a twofold increase in the concentrations in all
tissues, but the actual concentrations in any tissue will depend on the
pattern and extent of tissue distribution of the total body burden.
Equation 2-1 implies that different half-life values between and within
species will result in different body burdens for the same daily intake.
However, the Reassessment does not explicitly characterize how different
half-life value choices influence risk estimates. The half-life depends on two
independent physiological variables: the clearance (CL), which reflects the
volume of blood cleared per unit time, and the apparent volume of distribu
tion (V), which reflects the apparent volume of blood that has to be cleared
of chemical and which is determined by the extent of distribution to tissues
(for the one-compartment model used by EPA, half-life = 0.693 x V/CL).
The half-life, and therefore the estimated body burden at steady state, could
differ between species or between individuals due to differences in clearance
or in the extent of tissue distribution (V)—for example, due to differences
in body fat content. Because half-life depends on both CL and V, and body
fat content represents the major determinant of V for TCDD and other
dioxins, a species with a proportionately higher body fat content would
have a proportionately higher value of V, a proportionately longer half-life,
and greater body burden at steady state for the same daily intake. For this
reason, the blood concentration at steady state offers a better metric of the
concentration available within tissues to produce an effect:
Steady-state concentration =
Daily Dose x Bioavailability
CL
2-2
where concentration means the concentration per unit volume in blood or
plasma, and CL is expressed as the volume of blood or plasma cleared of
chemical per day.
This equation cannot be readily used because no data are available on
CL for humans. (CL would be the sum of all processes that remove the
compound from the body, which in the case of TCDD would largely relate
to diffusion into fecal lipids, whereas for lower chlorinated congeners, the
value of CL would also reflect metabolism.)
The Reassessment (Part III, section 1.3.2) considers the possibility of
using AUC as a dose metric, especially for the purpose of estimating cancer
risk. However, EPA questions the use of AUC because animal studies show
more altered hepatic foci after a single high dose than after repeated lowdose exposures giving the same AUC and because of challenges in determin
ing the appropriate averaging time (e.g., the whole lifetime or some discrete
window of susceptibility). The Reassessment notes that species life-span
differences imply a time-based correction to AUC across species, the correc
tion making AUC equivalent to average steady-state concentration. EPA
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could convert the AUC over any period to an average concentration by
dividing by the time period. The AUC for a dose interval at steady state is
directly proportional to the daily dose and bioavailability divided by CL
because AUCdose interval at steady state = (dose X bioavailability)/CL. The appar
ent volume of distribution does not influence blood or plasma AUC for a
dose interval at steady state, unlike body burden. The blood or plasma
concentrations would not vary greatly during a dose interval (day) because
of the long half-life of TCDD in both rodents and humans, and therefore
the average blood or plasma concentration could be used. The criticism of
using AUC in the Reassessment (whether it should be the peak AUC or the
average AUC related to the toxic effect) is inappropriate because it applies
equally well to the body burden metric used by EPA.
The Reassessment (Part III, section 1.3.3) considers the use of plasma
or tissue concentrations as a dose metric and states that few such data exist
for the chronic and subchronic animal studies, whereas human exposure
data depend predominantly on such measurements. The human data ex
pressed on a lipid-adjusted basis complicate interspecies comparisons with
rodent plasma data, and few data are available to quantify tissue concentra
tions during toxicity studies in animals. If possible, direct comparisons of
the concentrations in the lipid fraction of human blood and rodent blood
would provide the most secure comparison of internal dose if such data
became available in the future. Tissue concentration data for animals and
humans could be developed with physiologically based pharmacokinetic
(PBPK) models, based on the proportion of body fat and data on organ
blood flows and partition coefficients. Differentiation of free compound
from lipid-bound compound within a PBPK model could provide the most
relevant dose metric for dose-response assessment.
The approximately 100-fold difference between rats and humans in
TCDD half-life combined with Equation 2-1 suggests that a 100-fold lower
daily intake in humans yields a total body burden equal to that in rats
(assuming the same bioavailability). This observation raises a key question
not considered adequately in the Reassessment: Would similar total body
burdens in rats and humans result in similar target organ concentrations?
Similar tissue concentrations in both species would occur if the pattern of
distribution of the body burden were the same in both species. However,
the extent of hepatic sequestration (higher in rats, see Reassessment, Part II,
Tables 1-4 and 1-5) and the proportion of body fat (10% of body weight in
rats according to Geyer et al. [1990] and about 25% in humans—see
Reassessment, Part III, p. 17) both show important differences between rats
and humans. The significance of the different body composition can be
illustrated by considering the TCDD concentrations in rats and humans
that would be associated with a total body burden of 200 ng/kg of body
weight (calculated from the intake and half-life), assuming a body fat/blood
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concentration ratio of 100:1 at equilibrium for both rats and humans, and
that body fat is 10% of body mass in rats and 25% of body mass in
humans. For rats, the fraction of the body burden of TCDD in fat would be
proportional to 100 x 0.1 (10), and the fraction of the body burden of
TCDD in nonfat tissues would be proportional to 1 x 0.9 (0.9). Hence, a
total of 183.5 ng of TCDD would be in fat, and 16.5 ng would be in nonfat.
The total concentrations are 1,835 ng/kg in fat and 18.3 ng/kg in nonfat
tissues. In humans, TCDD in fat would be proportional to 100 x 0.25 (25),
and the amount of TCDD in nonfat tissue would be proportional to 1 x
0.75 (0.75). Therefore, for a body burden of 200 ng/kg of body weight, the
total TCDD in fat would be 194.2 ng, giving a TCDD concentration of
776.7 ng/kg, and the total in nonfat tissue would be 5.8 ng, giving a
concentration of 7.8 ng/kg. Consequently, for the same total body burden,
the TCDD and other dioxins concentrations in the tissues of humans are
about two to three times lower than those in rats.
The higher hepatic uptake in rats compared with humans means that,
for the same total body burden, there would be a greater proportion of
TCDD in the livers of rats. The Reassessment applies the same body burden
correction factor between rats and humans for liver cancer and for
nonhepatic effects. The proportionately higher concentrations in the livers
of rats compared with humans means that a proportionately higher daily
intake would be necessary in humans to produce a comparable hepatic
concentration. The difference in hepatic concentration based on the use of
body burden as a dose metric for extrapolation of data on liver cancer in
rodent bioassays to humans would represent an assumption that makes the
resulting risk estimate conservative, although the implications of this as
sumption are not described in the Reassessment. In addition, the Reassess
ment does not consider alternative assumptions. Because of the difference
in the percent of body fat, the same overall TCDD body burden generally
corresponds to lower tissue concentrations in humans, a factor that makes
extrapolation of data for all effects (including hepatic effects) more conser
vative. The Reassessment does not address this factor.
The tissue distribution of the body burden in studies that used single
doses or short periods of treatment will not correspond to the steady-state
pattern. Before completion of the distribution phase, there will be higher
concentrations in well-perfused tissues and lower concentrations in adipose
tissue. JECFA (2002) allowed for such nonequilibrium distribution in its
recent evaluation of the in utero effects produced in rats shortly after a
single dose of TCDD. The EPA Reassessment did not consider this ap
proach in the body burden calculations for the same studies.
The Reassessment does not adequately consider the use of a PBPK
model to define species differences in tissue distribution in relation to total
body burden for either cancer or noncancer end points. Kim et al. (2002)
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compared the body burdens associated with different levels of biochemical
responses calculated using a simple kinetic approach and using the body
burden derived from a PBPK model. The results indicated that the simple
kinetic method, which was similar to that used by EPA, and the PBPK model
gave quantitatively different results. The differences were not consistent across
the biochemical end points studied, suggesting that the response model used
was influencing the magnitude of the difference. Nevertheless, this study
supports the conclusion by the committee that the Reassessment should use a
simple PBPK model to address some of the uncertainties inherent in the use of
species differences in body burden as a measure of species differences in
target organ exposure. Generic PBPK models and PBPK models developed
specifically for TCDD and its congeners incorporate about 7% of the body
weight present as adipose tissue in rats and about 15% in humans (Gerlowski
and Jain 1983; Wang et al. 1997; Maruyama et al. 2002, 2003; Emond et al.
2004). Simple PBPK models of TCDD biodisposition at steady state could be
used to convert the estimated body burden into an appropriate species-re
lated difference in steady-state tissue concentrations; the magnitude of the
resulting species difference could then be introduced as a correction factor in
the equation used by EPA to calculate body burden from intake, half-life, and
bioavailability. The same PBPK model might also be used to explore the
influence of human variability in body composition on the elimination half
life and therefore the body burden at steady state. The Reassessment did not
consider this approach or quantify its impact, despite its recognition of tissue
concentration as the best dose metric.
DOSE-RESPONSE MODELING
Background
A critical element to consider when assessing human variability in re
sponse to a toxic substance is the nature of the dose-response relationship,
and how it is modeled mathematically. As described in major textbooks in
toxicology (e.g., Eaton and Klaassen 2001), analysts model two fundamen
tal types of dose-response relationships. The graded (continuous), indi
vidual dose response characterizes the nature and magnitude of an
individual’s response to a toxic substance as the dose goes from a small,
ineffectual dose to a larger, toxic dose, potentially causing death. The na
ture of the response may differ qualitatively, depending on the dose and
duration of exposure. For any given individual and specific, defined effect,
a “threshold dose,” may exist, which is defined as the dose below which the
individual does not respond. The dose corresponding to that threshold may
differ across individuals. For the purposes of risk assessment and public
health protection, however, analysts typically use the second type of dose-
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response relationship, called the “ quantal dose-response relationship,” for
a population of exposed individuals. The quantal dose response describes
the relationship between exposure and the proportion of the population
that will exhibit a health effect (that is, a separate relationship for each
adverse end point).
In the case of TCDD, other dioxins, and DLCs, it is important to assess
the population-based dose-response relationship for cancer, birth defects,
immunotoxic effects, and so forth. For each end point of interest, individu
als in a population (e.g., rats and mice in laboratory studies and humans in
epidemiological studies) are identified as either responders or nonresponders
at defined doses (quantal responses). The cumulative quantal dose-response
relationship for the population is then determined from the distribution of
responses in the population across a defined range of doses. The term
threshold is often used to describe the dose below which no response occurs
for the graded (continuous) dose-response relationship or the dose below
which the probability of anyone in the population responding approaches
zero for the cumulative quantal dose-response relationship. A common but
scientifically unachievable goal in risk assessment is to identify a threshold
dose that protects everyone in the population. The term offers some value
in recognizing that for the vast majority of dose-response relationships
(either individual or population) some doses may exist below which no
measurable responses occur (in an individual or a population). However,
the term threshold remains subject to many vagaries of interpretation, and
the committee prefers to express ranges of dose in terms of MOEs. MOEs
are usually defined as the ratio of the highest dose (daily exposure) to an
agent presumably without adverse impact on the human population (the
so-called reference dose; Faustman and Omenn 2001) to the estimated daily
human dose that might occur, determined from analysis of actual exposure
scenarios.
Because of inherent biological differences between individuals, as well
as the probabilistic nature of many toxic responses, distributions in re
sponses in a population will always exist (that is, not everyone responds the
same way to the same dose). In human populations, differences arise from
genetic diversity, differences in age, gender, nutritional status, diseases, and
other concomitant exposures, which can modify the response of an indi
vidual to a toxic substance. However, such contributors to human variabil
ity are presumably represented in the data sets obtained in human popula
tion-based studies (epidemiological studies), although any one study
generally cannot capture the full range of possible individual variability in
response. A second major challenge in establishing population-based doseresponse relationships in epidemiological studies arises from the frequently
poor quality of exposure (dose) information. Although well-designed occu
pational and environmental epidemiological studies can yield useful infor-
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GENERAL CONSIDERATIONS
65
mation on human population variability, relatively little quantitative infor
mation is available about the potential impact on genetic polymorphisms in
the human population that might give rise to differences in susceptibility to
the toxic effects of TCDD, other dioxins, and DLCs. Chapters 5 and 7
provide more discussion about genetic, molecular, and biochemical mecha
nisms that might contribute to interindividual variation in response to
TCDD, other dioxins, and DLCs.
With these caveats noted, risk assessors commonly take existing data
sets (both animal and human) and attempt to develop mathematical models
to characterize the shape of the dose-response relationships from the ob
served data.
Dose-response modeling is a process to formally quantify dose-related
changes in the incidence or severity of an adverse effect. The scale of the
response can be quantal (e.g., cancer incidence) or continuous (e.g., AHRbinding immune response). Analysts use mathematical functions (prefer
ably with mechanistic parameters) to describe the dose-response relation
ship observed in the data. In the case of cancer or any quantal outcome, the
dose-response model, R(dose), is the same as the probabilistic risk of the
adverse outcome. With this dose-response model, or risk, R(dose), the EDa,
at which there is a prespecified, small amount (typically 1 ~ 10%) of risk
increase a above the background, can be estimated by the following equa
tion of excess risk:
R(ED ) - R(background exposure)
= a.
1 - R(background exposure)
The risk increase a is called the effective dose level. Because R(dose) is a
statistically estimated quantity (function), the resultant EDa is subject to
data variation.
In the case of a continuous response (or more generally, a nonquantal
response), EPA guidance documents discuss how the type of data and bio
logical knowledge will determine appropriate methods using general ap
proaches, but no single approach or model can be universally the “ best.”
Analysts first fit a dose-response model R(d) to the response data. They
then take additional steps to formulate a measure of risk based on the
model. Here, R(d) describes the mean response level of the toxicological
outcome (e.g., cognitive function as measured in terms of IQ test score in
the case of exposure to a neurotoxin). The Reassessment discusses several
proposed approaches (Part II, pp. 15-16), all of which identify a dose
associated with a specified level of response change relative to the control.
For continuous responses, this task is complicated by the ambiguous sepa
ration between a “normal response” and an “ adverse response.” In lieu of
an obvious dividing line, EPA used the “ dynamic range” approach (Murrell
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
et al. 1998), which defines EDa (EPA assigned an a value of 1%) as the dose
satisfying the relationship,
R(ED ) - R(background)
= a.
R max
where Rmax is the maximum range of total response, either theoretical or
estimated under the maximum exposure condition. The main drawback of
the approach used by EPA is that the response level associated with the EDa
may not be clinically or toxicologically important. The NRC (2000) de
scribed an alternative approach in the context of its review of methylmercury toxicity, based on work by Crump (1984) and Gaylor and Slikker
(1992). That approach first identifies an adverse response level, which
demarcates normal and abnormal (or adverse) responses. For example, in
the case of a neurotoxin, an IQ score of 70 points (two standard deviations
[SDs] below the population mean of 100 points) could be designated the
adverse response level because individuals with IQ scores below this level
often require community support to live (WHO 1992, as cited in EPA
2005c). The EDa is then defined to be the neurotoxin dose that increases
the background probability of an adverse response by a . Continuing the IQ
example, the ED05 is the level of neurotoxin exposure that increases the
background risk of having an IQ below 70 of 2.5% by an extra 5%
(5% *97.5% =4.875% ), to a total of 7.375%.
The Reassessment (Part II, p. 8-16) identifies difficulties with this ap
proach. Although such an adverse response level might not always identify
toxicologically meaningful events, it can identify unusual outcomes outside
the normal range. The committee recognizes this challenge and understands
that for some end points this may emerge as an insurmountable challenge.
Nonetheless, because the EDa definition used by EPA is difficult to interpret
toxicologically, EPA should strive to use the alternative approach described
here whenever possible.
Historically, risk assessment of noncancer effects used a NOAEL or a
LOAEL as the POD. The BMD approach (Crump 1984) eliminates some of
the limitations of the NOAEL and LOAEL approach and makes the analy
sis of noncancer effects more consistent with that of cancer.
The primary objective of dose-response modeling is to define an ED
toward the lower end of the experimental dose range where the model
remains supported by adequate data. The ED can then be used as a POD for
extrapolation toward an environmental background level or for safety as
sessment using the MOE approach.
The choice of model for dose-response assessment, choice of the POD,
and extrapolation below the POD thus represent other key areas of uncer
tainty. The Reassessment quantified the cancer dose-response relationship
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GENERAL CONSIDERATIONS
67
relying primarily on occupational cohort data. EPA also used selected ani
mal bioassay data to confirm the plausibility of the resulting estimates.
Specific issues related to choice of data set for cancer risk assessment are
discussed in more detail in Chapter 5
Data Set Selection
Full dose-response modeling requires adequate dose-response data, and
adequate selection criteria must be applied. EPA’s guidance document (EPA,
2000b, p. 14) states:
In general, studies with more dose groups and a graded monotonic re
sponse with dose will be more useful for BMD analysis.... Studies in
which responses are only at the same level as background or at or near the
maximal response level are not considered adequate for BMD analysis. It
is preferable to have studies with one or more doses near the level of the
BMR to give a better estimate of the BMD and, thus, a shorter confidence
interval. Studies in which all dose levels show changes compared with
control values (i.e., no NOAEL) are readily useable in BMD analyses,
unless the lowest response level is much higher than that at the BMR.
Depending on whether the scale of the selected end point is quantal (di
chotomous), continuous, or categorical, different statistical procedures and
models are required for dose-response modeling.
EPA’s Reassessment selected a large body of published data sets, using
the criteria of (1) a positive dose trend and (2) at least three dose groups in
addition to a control (more specifically for noncancer data). In dose-re
sponse modeling of human cancer data, EPA further used cancer death
incidence (time-to-event) data as the end point, which generally provides
more information than mortality data by considering when a death oc
curred. (These studies are discussed in more detail in Chapter 5.)
Statistical Power and Precision
Although meeting those minimal selection criteria (discussed above) is
critical, it does not guarantee adequate statistical power to ascertain the
shape of the dose-response curve, and it does not account for the associated
uncertainty. In the present context, statistical power refers to the general
ability of an experiment, and its associated data set, to provide information
needed to make a reliable inference, including testing positive dose effects
and ascertaining a fitted dose-response model.
The Reassessment did not discuss the issue of statistical power, al
though the cancer guidelines (EPA 2005a, see also Appendix B) recommend
assessing the statistical power of the studies used for dose-response assess
ment when possible. Even if a study possesses adequate statistical power to
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confirm a positive overall dose response within the observed data range, the
power might be inadequate to ascertain the shape of the dose-response
curve below the POD level. The lack of statistical power at the lower end
also represents a problem for both cancer and many of the noncancer data
sets, contributing additional uncertainty to the POD.
Choice of the Dose-Response Model
The goal of mathematical modeling in determining a POD is to fit a
model that describes the data set well, especially at the lower end of the
observable dose-response range. Fitting such a model involves first selecting
models for consideration, based on the characteristics of the data and ex
perimental design, and then fitting the models using one of a few estab
lished methods. Then, an ED, along with its upper and lower confidence
bounds, is calculated at the POD level. In the process, the analysis should
evaluate model fitting, determine goodness-of-fit, and compare models to
decide which one to use for obtaining the POD. For example, the BMD
guidance document (EPA 2000b) recommends use of a P value of 0.1 as the
reference critical value for goodness-of-fit (instead of the more conven
tional values of 0.05 and 0.01), examination of a graphical display of the
model fit, and use of Akaike’s information criterion for comparison of
models and selection of the model to use.
In the case of human cancer data, the Reassessment included fits of
linear and nonlinear models to the data (see Chapter 5). With the rodent
cancer data, EPA used a simple multistage model fitted with the BMD
software program. For noncancer data, EPA used the Hill model as the
default for continuous responses, with a power model as the alternative
when the Hill model failed to fit the data computationally. (See Chapter 6
for additional discussion about specific noncancer end-point modeling.)
EPA used the Weibull model as the default for quantal noncancer data. The
committee commends EPA for using flexible mathematical models (e.g., the
Hill and Weibull models) to account for both nonlinear and linear shapes of
the dose response for noncancer effects. However, the committee recom
mends that EPA apply similar efforts in dose-response modeling of human
cancer data (see Chapter 5).
The Reassessment did not conduct or report statistical tests of goodness-of-fit of the cancer risk models. Two reasons might explain the ab
sence of these test results. First, EPA relied on the models reported in the
original publications. For example, Steenland et al. (2001) fitted several
models to the risk ratio for cancer death incidence, including a power and a
piecewise linear model. The likelihood ratio test showed a statistically sig
nificant, positive dose response, but the graphical display clearly showed a
potential lack of fit. It is important to note that a higher statistical signifi-
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FIGURE 2-1 Vmax. As used in the BMD software for modeling dose-response
data, the term Vmax refers to the modeled maximum percent response seen in the
observed data set. SOURCE: N. Walker, NIEHS.
cance does not correspond to a higher degree of goodness-of-fit of the
model to the data. The Reassessment did not distinguish statistical tests of
significance from tests of goodness-of-fit. Second, EPA had access only to
summary data taken from the published literature for dose-response model
ing, not the raw data, and consequently may not have been able to conduct
statistical tests for goodness-of-fit. Nonetheless, the committee recognizes
that the critical choice of the dose-response model would benefit from as
much information as possible.
In contrast, EPA adopted an ad hoc method to assess goodness-of-fit in
dose-response modeling of noncancer end points. Specifically,
the model fits were evaluated with regard to the observed data. The good
ness of the model fit was determined as ‘good’ if the model curve included
nearly all of the data point means, ‘marginal’ if the model curve was
within one standard deviation of the data point means, or ‘poor’ if model
fit was not within one standard deviation of the means.
Furthermore,
for the Hill model fits, the Vmax [see Figure 2-1] estimates from ‘good’
and ‘marginal’ model fits were subjectively evaluated for stability and
biological plausibility with regard to the observed data. This evaluation
identified some potential problems with some of the Vmax estimates. In
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some cases the error associated with the Vmax could not be calculated by
the BMD software. In these cases if the Vmax model estimate was similar
to the ‘observed Vmax’ (i.e. the difference between the highest dose re
sponse level and the control response level) then the Vmax estimate was
considered biologically plausible and was used for the calculation of an
ED01. Otherwise the ‘observed Vmax’ was used for calculation of the
ED01. (Part II, p. 8-32)
This subjective approach to goodness-of-fit did not identify whether the
lack of fit occurs at the higher or lower end of the observed dose-response
range. Alternatively, the Reassessment could judge goodness-of-fit of an
empirical dose-response model on mechanistic grounds.
Finally, a statistically well-fit model alone does not guarantee that the
model approximates the true but unknown shape of the dose response,
especially below the observed dose-response range. With limited data (e.g.,
about three dose groups for noncancer data) and limited statistical power,
many of the data sets (including epidemiological studies) analyzed in the
Reassessment do not provide sufficient information to confirm the true
shape of the dose-response curve at the ED01 level. The committee empha
sizes that this critical uncertainty about low-dose extrapolation remains
one of the most significant uncertainties; at the same time, it represents an
uncertainty that EPA probably will not resolve in the short term. When
feasible, mechanistic and statistical information should be used to ascertain
the shape of the dose-response curve at lower doses. Minimally, EPA should
use rigorous statistical methods to assess model fitting to control and re
duce the uncertainty of the POD caused by a poorly fitted model.
Choice of the POD Value
Selection of the ED (BMR) level is critically important in the calcula
tion of an ED (BMD), and therefore, in the determination of a POD or
calculation of a MOE. The current cancer guidelines (EPA 2005 a, see also
Appendix B) and the draft BMD guidance document (EPA 2000b) give
detailed recommendations. For quantal data, an excess risk of 10% was
chosen as the default level because 10% response is at or near the limit of
sensitivity in most cancer bioassays and in some noncancer studies as well.
If a study offers greater than usual sensitivity, then a lower level (e.g., 1%)
can be used. EPA recommends the 1% BMR level for epidemiological
studies primarily because the 1% level is typically within the observed
range. In any case, according to the guidance document, the ED10 should be
reported along with any other possible POD options. EPA’s BMD guidance
document further recommends:
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For continuous data, if there is an accepted level of change in the endpoint
that is considered to be biologically significant then that amount of change
is the BMR. Otherwise, if individual data are available and a decision can
be made about what individual levels should be considered adverse, the
data can be ‘dichotomized’ based on that cutoff value, and the BMR set as
above for quantal data. Alternatively, in the absence of any other idea of
what level of response to consider adverse, a change in the mean equal to
one control standard deviation (SD) from the control mean can be used.
The control SD can be computed including historical control data, but the
control mean must be from data concurrent with the treatments being
considered. Regardless of which method of defining the BMR is used for a
continuous dataset, the effective dose corresponding to one control SD
from the control mean response, as would be calculated for the latter
definition, should always be presented for comparison purposes. (EPA
2000b, p. vii)
In EPA’s computation of ED01 for noncancer continuous end points,
the 1% BMR level is defined as the change of response from the back
ground level of the control group that was 1% of the maximum possible
total response range. The choice of a 1% BMR level ignored EPA’s own
guidance that “ if there is an accepted level of change in the end point that is
considered to be biologically significant then that amount of change is the
BM R” (EPA 2000b, vii). The Reassessment also did not consider an alter
native approach to dichotomize a continuous outcome into normal and
extreme outcomes below a lower or above an upper percentile (Gaylor and
Slikker 1990), an approach recommended in the BMD guidance document
(EPA 2000b) and implemented in EPA’s BMD software program.
Because the shape of the dose-response is less certain at the lower end
of the experimental range, the consequent uncertainty for the ED chosen in
this range is important. This uncertainty is likely to be greater for the lower
confidence bound of ED01 than on the central estimate of ED01 itself. The
Reassessment appears to have largely ignored this issue.
As the starting point of extrapolation of risk to environmental expo
sure levels, the POD directly influences the risk estimate. The lack of fit of
the model at the lower end of the dose-response curve leads to substantial
extrapolation of the model toward the POD, and that can bias the ED or
BMD estimates and widen their confidence intervals, adding substantially
to the uncertainty of the estimate.4
4The accuracy of any experimental measurement is limited by the ability to measure the
phenomenon, by any methodological errors introduced through sampling (e.g., limitations in
sample size or selection), and by assumptions made in fitting a model to the data. As such,
any result obtained provides an estimate of the “ true value” with some associated uncer
tainty. A confidence interval represents the likelihood that the “ true value” will occur within
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Despite the Reassessment’s consideration of multiple options and the
use of flexible model forms (such as the Hill model and the Weibull model)
to test for nonlinear dose response, mechanistic knowledge gaps, data gaps,
and model gaps remain. For example, many of the data sets of noncancer
effects yielded a Hill coefficient greater than 1.5, indicating a plausible
nonlinear dose response. However, those studies lacked adequate statistical
power to estimate the Hill coefficient reliably, rendering the estimate statis
tically nonsignificant (that is, the confidence interval includes unity). This
result represents a general data gap because the dose-response data required
to establish a nonlinear dose-response form do not exist, a problem that
becomes magnified in extending nonlinear models to the low-dose range.
At present, mechanistic knowledge of both cancer and noncancer effects
supports the plausibility of a nonlinear dose response at the lower range
(see also Chapter 5), but no adequate data or widely accepted dose-re
sponse models describe the shape below a chosen POD at or below the 1%
level. It is useful to differentiate the lack of data to confirm the shape of the
dose-response curve below the POD from the lack of qualitative evidence of
nonlinearity. On the whole, the committee concluded that the empirical
evidence supports a nonlinear dose response below the ED01, while ac
knowledging that the possibility of a linear response cannot be completely
ruled out. The Reassessment emphasizes the lack of such nonlinear models,
hence its adoption of the approach of linear extrapolation below the POD
level. Although this approach remains consistent with the cancer guidelines
(EPA 2005a, see also Appendix B), EPA should acknowledge the qualitative
evidence of a nonlinear dose response in a more balanced way, continue to fill
in the quantitative data gaps, and look for opportunities to incorporate
mechanistic information as it becomes available. The committee recommends
adopting both linear and nonlinear methods of risk characterization to ac
count for the uncertainty of dose-response relationship shape below ED01.
With respect to dose-response modeling, the committee recommends
that the Reassessment explicitly acknowledge the lack of statistical power
(precision) of the data to estimate the ED01 or test nonlinearity of the dose
response below the POD level of choice (e.g., ED01).
The committee notes that the choice of the 1% response level as the
POD substantially affects both the cancer and the noncancer analyses,
the range of the lower and upper confidence bound. For example, statisticians often choose to
report a 95% confidence interval, which implies a 95% chance that the true value will fall
within the stated range, but this represents a subjective choice and other choices (e.g., 90%
confidence interval) are equally valid. The confidence interval depends on the underlying
variability of the quantity being measured or modeled and the number of samples collected
and/or available to fit the data. For any given result, collecting more samples tends to narrow
the confidence interval.
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GENERAL CONSIDERATIONS
73
perhaps driving EPA’s decision not to develop an RfD. The committee
recommends that the Reassessment use levels of change that represent clini
cal adverse effects to define the BMR level for noncancer continuous end
points as the basis for an appropriate POD in the assessment of noncancer
effects. The Reassessment should also explicitly address the importance of
statistical assessment of model fit at the lower end and the difficulties in
such assessments, particularly when using summary data from the literature
instead of the raw data, although estimates of the impacts of different
choices of models would provide valuable information about the role of
this uncertainty in driving the risk estimates.
CONCLUSIONS AND RECOMENDATIONS
• Although EPA qualitatively addressed many sources of uncertainty
and variability, the Reassessment does not adequately address uncertainty
and variability that result from the numerous decisions EPA made in deriv
ing point estimates of cancer risk in the comprehensive risk assessment. In
contrast, EPA used concerns about uncertainties and uncertainty factors as
part of the justification for not setting an RfD for noncancer effects (see
Chapter 7 for further discussion).
• The Reassessment does not provide details about the magnitudes of
the various uncertainties surrounding the decisions EPA makes in relation
to dose metrics (e.g., the impact of species differences in percentage of body
fat on the steady-state concentrations present in nonadipose tissues). The
committee recommends that EPA use simple PBPK models to define the
magnitude of any differences between humans and rodents in the relation
ship between total body burden at steady-state concentrations (as calcu
lated from the intake, half-life, bioavailability) and tissue concentrations.
The same model could be used to explore human variability in kinetics in
relation to elimination half-life. EPA should modify the estimated human
equivalent intakes when necessary. Many opportunities exist to further
characterize sources of uncertainty and variability related to the dose metric
choices, and the committee recommends that EPA provide a clear evalua
tion of the impacts of possible choices on the risk estimates.
• The committee recommends that EPA make greater use of mechanis
tic information to assess the biological plausibility of different mathemati
cal models, use more rigorous criteria (e.g., goodness-of-fit tests) and fol
low its own guidance (EPA 2000b) in deriving a POD, and clearly identify
the BMR level of toxicological significance for noncancer end points. Many
opportunities exist to further characterize sources of uncertainty and vari
ability related to the POD and extrapolation choices, and the committee
recommends that EPA provide a clear evaluation of the impacts of possible
choices on the risk estimates.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
• The committee notes that EPA would substantially improve its
transparency and management of the complexity of the risk assessment of
TCDD, other dioxins, and DLCs by creating an ongoing process for clearly
identifying and updating the key assumptions that support the quantitative
risk assessment. This process would essentially require viewing the risk
assessment as an ongoing and iterative effort in which EPA continues to
create incentives to obtain and use better information when possible and
appropriate.
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T o x ic E q u iv alen cy F a c to rs
Assumptions, variability, and uncertainty are well delineated in Part II
of the Reassessment1 that addresses critical considerations in the applica
tion of the toxic equivalency factor (TEF) method (Part II, Chapter 9,
section 9.2.6, p. 9-10). In addition, conclusions in Part III appear to be
congruent with discussions in Part II, Chapter 9, and in the Reassessment
overall. No major omissions were identified in the Reassessment, but sev
eral aspects need to be addressed or updated.
DIOXIN-LIKE COMPOUNDS
The compounds that are the focus of the Reassessment include 7 of 75
polychlorinated dibenzo-p-dioxins (PCDDs), 10 of 135 polychlorinated
dibenzofurans (PCDFs), and of the total 209 polychlorinated biphenyls
(PCBs), only 4 of the 122 previously defined as 2,3,7,8-tetrachlorodibenzo-
1The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
2O f the 12 PCBs that received TEF values from the World Health Organization (WHO),
the U.S. Environmental Protection Agency (EPA) only considered four in the Reassessment.
The remaining eight mono-ortho-substituted PCBs were not considered at this time because
of concerns about the accuracy of previous in vivo and in vitro toxicological (relative p o
tency) results, given that a recent study found that many preparations of “ pure” mono-ortho
PCBs actually contained potent dioxin-like coplanar PCBs as minor contaminants.
75
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
p-dioxin (TCDD)-like by the World Health Organization (WHO) (van
den Berg et al. 1998). The toxic potency of each of these DLCs (their
TEFs) is expressed relative to that of TCDD (also referred to as dioxin),
the most potent member of this chemical class (Part III, Table 1.3, p. 1
20). These chemicals are classified as DLCs, given their similarity in chemi
cal structure and physiochemical properties, their ability to invoke a com
mon battery of toxic responses by a common aromatic hydrocarbon
receptor (AHR)-dependent mechanism in vivo, and their ability to be
persistent environmentally and to bioaccumulate. The lack of inclusion of
the eight mono-ortho PCBs previously assigned TEFs by WHO in the
Reassessment at the present time is due to concerns that the previously
reported activity of many of these chemicals might have been primarily or
partially due to a dioxin-like PCB contaminant (PCB126) present in these
mono-ortho PCB preparations (DeVito et al. 2003). Although some AHRdependent toxic effects have been observed with mono-ortho PCBs pre
pared by methods that should not produce the more toxic DLCs, it re
mains to be determined whether most of the reported toxicological effects
and resulting relative potency (REP) values of these chemicals are due to
contaminants, mono-ortho PCBs, or both. Given this uncertainty and the
fact that reanalysis of the mono-ortho PCBs as pure compounds is cur
rently being reexamined, they were not included in the list of relevant
DLCs for consideration in the Reassessment. Once these issues are re
solved, the mono-ortho PCBs should be considered in a follow-up to the
Reassessment if they are documented to produce AHR-dependent toxic
effects.
MAJOR ISSUES, ASSUMPTIONS, AND UNCERTAINTIES
The relative toxicological and biological potency of a complex mixture
is assessed by the TEF approach. Current TEFs are “ order-of-magnitude”
qualitative values for dioxins, other than TCDD, and DLCs that were
established by a WHO expert scientific panel that examined a large scien
tific database of REP estimates from in vivo and in vitro studies of the
biochemical and toxic effects. In the TEF approach, the concentration of
the individual compound present in the mixture (determined by instrumen
tal analysis) are multiplied by their specific TEF value, and the sum is
expressed as the TCDD toxic equivalent quotient (TEQ). Summation of the
calculated TEQs for all active TCDD-related compounds in a sample ex
tract yields the total TEQ for the specific sample extract. Numerous as
sumptions underlie the use of the TEF/TEQ approach; these have been well
delineated, and the major aspects are discussed in detail in the Reassess
ment (Part II, Chapter 9, and Part III, Chapter 1, section 1.2). These as
sumptions and uncertainties are described and discussed below.
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77
Role of AHR
Assumption: AHR mediates most toxicities produced by TCDD and
other PCDDs, PCDFs, and coplanar PCBs that are AHR agonists. Al
though AHR is necessary, the ability of TCDD, other dioxins, and DLCs to
produce their biochemical and toxicological effects results from down
stream events regulated by AHR and AHR-dependent gene expression. The
role of AHR in the toxic and biological effects of the TCDD, other dioxins,
and DLCs has been supported by a substantial number of quantitative
structure-activity relationship, biochemical, genetic, and targeted Ahr
knockout studies.
AHR-Independent Mechanisms Excluded
Assumption: Effects mediated by other mechanisms (AHR indepen
dent) and interactions with other chemicals are ignored. AHR-independent
effects of TCDD have been previously observed, including effects on intra
cellular calcium levels (Puga et al. 1997), changes in gene expression
(Oikawa et al. 2001), and selected toxicity in Ahr knockout mice
(Fernandez-Salguero et al. 1996; Lin et al. 2001). Whether all TCDDrelated compounds produce these effects is unknown. Although these
mechanisms may play a role in the biochemical effects of TCDD, other
dioxins, and DLCs, their significance and role in the overall toxic effects of
these compounds remain to be established. However, the Reassessment
should acknowledge that AHR-independent effects of TCDD occur and
that future studies might demonstrate a role for these effects in the overall
toxic and biological effects of TCDD, other dioxins, and DLCs.
Uncertainty of TEF Values
Considering the uncertainty in selection of the TEFs and the informa
tion presented on REPs and TEFs in the Reassessment, the 2000 EPA
Science Advisory Board (SAB) Panel “ questioned whether the uncertainty
in the TEFs and the application of this approach to predicting risks due to
current levels of exposure was adequately presented” (EPA SAB 2001, p.
29). They concluded that the Reassessment should acknowledge the need
for better uncertainty analysis of the TEF values, and although no current
method for doing so has been endorsed by the scientific community, several
approaches were suggested, such as the use of probabilistic distributions of
TEF values in TEQ evaluation (Finley et al. 2003). Available information
indicates a considerable amount of variability in the REP value data that
were used to derive the WHO TEF values. In addition, although the WHO
TEFs were derived based on a scientific consensus evaluation of the avail-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
able REP values using defined weighted criteria for individual studies, de
tails of the quantitative basis of this weighting scheme were not clearly
presented in the description publication (van den Berg et al. 1998). These
issues would contribute to variability and uncertainty in the application of
the WHO TEF values to health risk assessment. Application of a math
ematical value or percentage of the overall range of REP values, such as
those described by Finley et al. (2003), would be one way to make the
process of determining the specific TEFs more transparent and to provide a
standard method to develop TEFs for other TCDD-related compounds that
may be added at a later date. Some members of the 2000 EPA SAB Panel
also recommended “ that, as a follow up to the Reassessment, EPA should
establish a task force to build ‘consensus probability density functions’ for
the thirty chemicals for which TEFs have been established, or to examine
related approaches such as those based on fuzzy logic” (EPA SAB 2001, p.
29). The committee strongly recommends that the EPA consider inclusion
of uncertainty analysis of the TEF values as a follow-up to the current
Reassessment.
Consistency of DLC REP Values
Assumption: The REP of a chemical in this group is presumed to be
equivalent for all end points o f concern and for all exposure scenarios, and
all are full agonists. Although most in vitro and in vivo studies support this
assumption, the 2000 EPA SAB Panel noted in their review of the Reassess
ment (EPA SAB 2001) that there are reports of significant differences be
tween the potency of some dioxins, other than TCDD, and some DLCs and
specific “ toxic end points” is illustrated in Table 5-4 and Table 2-4 in the
Integrated Summary (SAB 2001, p. 31). For example, the panel indicated
that “ 1,2,3,7,8- PeCDF (pentachlorodibenzofuran) has the same tumorigenicity as TCDD but was ~38 times weaker for teratogenicity; the other
congener, 2,3,4,7,8-PeCDF had half the tumorigenic potency as TCDD, but
is ~8 times less potent for teratogenicity” (EPA SAB 2001, p. 31). However,
although it was noted that no other examples of that difference were pre
sented in the Reassessment, the observations did raise some concerns about
whether all toxic end points could be combined into a single TEF value. The
2000 EPA SAB Panel suggested that “ because TEFs vary among different
endpoints as well as congeners, it would also be helpful for the document to
note that, as data becomes available, it may be possible to derive TEQs [and
TEFs] for different endpoints” (EPA SAB 2001, p. 31). The committee
agrees that end-point-specific TEFs should be used in those situations in
which one is interested in assessing the effects of a sample on a specific end
point; however, for general monitoring or screening approaches (that is, for
TCDD-related compounds in food and environmental samples) in which all
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end points should be considered, TEF values that are based on all end
points should be used.
Use of TEFs for DLC Body Burdens
Perhaps the issue of greatest concern in this section of the Reassessment
is whether the current WHO TEFs, which were developed to assess the
relative toxic potency of a mixture to which an animal is directly exposed
by dietary intake, are appropriate for the assessment of internal TEQ con
centrations and potential toxic effects. Application of the equation relating
body burden, half-life, and bioavailability to congeners other than TCDD
to give TCDD equivalents based on intake TEF values assumes that the TEF
allows adequately for any difference between the congener and TCDD for
half-life and bioavailability aspects. In addition, if exposure and estimated
body burden of dioxins, other than TCDD, and DLCs are based on mea
sured tissue concentrations, then converting the tissue concentration to a
TEQ with TEFs derived from external doses might not be appropriate and
might introduce significant uncertainties into the total TEQ estimate. In
fact, previous studies have suggested that, because of toxicokinetic differ
ences, the REP values for three PCDFs (2,3,7,8-tetrachlorodibenzofuran
[TCDF], 1,2,3,7,8-PeCDF, and octachlorodibenzofuran [OCDF]) were
greater when estimated from tissue concentration than when estimated
from administered dose (DeVito et al. 1997). These data would support
development of body burden TEF values in which the level of toxicity is
directly related to body burden concentrations of a given DLC. Questions
have also been raised about including octachlorodibenzo-p-dioxin (OCDD)
and OCDF in the TEF scheme. Differences in the toxicokinetics of these
compounds from other chemicals complicated early studies. OCDF and
OCDD were originally assigned a TEF of zero because they failed to pro
duce effects in early toxicity studies. However, both OCDF and OCDD are
poorly absorbed in the gastrointestinal tract (Birnbaum and Couture 1988;
DeVito et al. 1998) and significant TCDD-like effects of each were ob
served only after repeated doses were given over an extended time to allow
accumulation in tissue (Couture et al. 1988; DeVito et al. 1997). Whether
toxicokinetic differences of dioxins, other than TCDD, and DLCs exist that
would similarly affect their REP and thus their TEFs need to be determined.
However, these results raise concerns about the use of intake TEFs for body
burden TEQ determinations and suggest that, if possible, it would be more
appropriate to generate an additional set of TEFs for body burden tissue
equivalents that could be used for DLC risk evaluation purposes. In addi
tion, the use of intake TEFs for body burden TEQ determinations questions
the overall conclusion that TCDD, other dioxins, and DLC body burden in
humans is currently close to levels that reportedly produce adverse effects in
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
animals. Would it be higher or lower depending on the specific TEFs ap
plied? A discussion of this point could not be found in the sections on toxic
equivalents and should be included.
Additivity of DLCs
Assumption: Mixtures exhibit additive toxicities based on TEFs of indi
vidual chemicals. Additivity is a particularly critical assumption for the TEF
approach. Considerable discussion of this issue is provided in the Assess
ment, Part II, Chapter 9, and from an overall perspective, this assumption
appears valid, at least in the context of risk assessment. Additivity in bio
chemical and toxic responses by the indicated compound has been sup
ported by numerous controlled mixture studies in vitro and in vivo and is
scientifically justifiable. That support is not the case with other non-DLC
PCDDs, PCDFs, and PCBs that are reported to be partial agonists or an
tagonists. The presence of partial agonists or antagonists in a complex
mixture or in vivo would likely reduce the overall toxic potency (TEQ) of a
mixture when tested in an animal when compared with the TEQ potency
calculated simply from application of TEF values to individual compounds
measured by instrumental analysis of the mixture. In fact, the ability of
some non-DLC PCBs and PCDFs to inhibit TCDD-induced cytochrome
4501A1 protein (CYP1A1) activity and immunotoxicity in C57BL/6J mice
has been reported (Bannister et al. 1987; Davis and Safe 1988; Biegel et al.
1989; Chen and Bunce 2004), as has the ability of a lower-affinity synthetic
PCDF, such as 6-methyl-1,3,8-trichlorodibenzofuran (6-MCDF), to inhibit
TCDD-induced CYP1A1, teratogenicity, immunotoxicity, and porphyria in
rodent models in vivo (Astroff et al. 1988; Harris et al. 1989; Bannister et
al. 1989; Yao and Safe 1989). These studies indicate that persistent nonDLCs can affect the magnitude of toxic and biological effects produced by
a defined amount of TEQ calculated for a given complex mixture. How
ever, given that the presence and concentration of these chemicals in a
particular extract can vary dramatically and that very few published studies
demonstrate significant alterations in the additive toxicities of dioxins, other
than TCDD, and DLCs by other persistent non-DLC AHR ligands in vivo,
the assumption of additivity of dioxins, other than TCDD, and DLCs should
be considered a valid approach at the present time. Several published pa
pers have demonstrated synergistic activation of AHR-dependent gene-ex
pression effects that involve cross-talk between signaling pathways even at
low concentrations. However, with respect to AHR-dependent toxic ef
fects, current data are consistent with ligand and agonist additivity, which
is a key assumption of the TEF/TEQ approach. However, EPA should
acknowledge the possibility that the presence of non-DLC AHR antago-
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81
nists in a complex mixture could affect the magnitude and overall toxic
effects produced by the calculated amount of TEQs present in a mixture
containing such compounds.
Rodent-to-Human Prediction
Assumption: REP of TCDD, other dioxins, and DLCs in rodent models
is predictive of REP in humans, given that the rank-order potency is similar
between species. Results from available in vivo, in vitro, and accidental and
occupational exposure studies are generally consistent with this assumption.
Numerous investigators have reported species-specific differences in AHR
ligand binding affinity of TCDD, other dioxins, and DLCs. Depending on the
system examined, the estimated affinity of binding of TCDD (and related
compounds) to the human AHR is about 10-fold lower than that observed to
the AHR from “responsive” rodent species and is comparable to that ob
served to the AHR from “nonresponsive” mouse strains (Roberts et al. 1990;
Ema et al. 1994; Poland et al. 1994; Ramadoss and Perdew 2004). This
reduced affinity appears to be at least in part due to a single amino acid
substitution within the ligand binding domain of the human and “nonre
sponsive” mouse AHRs (Ema et al. 1994; Poland et al. 1994; Ramadoss and
Perdew 2004). Although the affinity of binding of TCDD and related com
pounds to the human AHR is reduced compared with rodent AHRs, the
qualitative and quantitative rank-order potency of these chemicals is similar.
In addition to ligand binding, the REP of TCDD and related compounds to
induce AHR-dependent gene expression in human cells is also reduced by up
to 10-fold (Roberts et al. 1990; Harper et al. 1991; Xu et al. 2000; Zhang et
al. 2003; Peters et al. 2004; Silkworth et al. 2005). Because TEFs are ex
pressed relative to the toxicity of TCDD, the shift in TEF values of dioxins,
other than TCDD, and DLCs appears to be similar between species. Several
recent papers have reported that biological and toxicological responsiveness
of humans to TCDD, other dioxins, and DLCs can vary up to 10-fold in vivo
and in vitro and that these interindividual differences in responsiveness are
not due to specific polymorphisms in AHR (Anttila et al. 2000; Harper et al.
2002; Cauchi et al. 2003). Not only do the documented species differences in
AHR ligand binding and AHR responsiveness need to be addressed or taken
into consideration with regard to rodent-to-human extrapolation, but the
issue of interindividual variability among humans in their responses to
TCDDs, other dioxins, and DLCs also needs to be considered when assessing
human risk. The rank-order potency of other non-DLC AHR agonists is not
necessarily similar between species, and if these chemicals are to be included
in the TEF methodology in the future, species-specific TEFs would need to be
developed.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Other Persistent AHR Agonists
Assumptions: Although other classes of persistent halogenated environ
mental chemicals that are structurally related to TCDDs, other dioxins, and
DLCs have been identified, they are excluded because there are limited
toxicological data and no validated TEFs for these chemicals. Another
important source of uncertainty is the acknowledged likelihood that other
persistent halogenated chemicals, such as brominated and mixed chloro
and bromo coplanar chemicals, are present in environmental mixtures, the
identities of which are just now emerging and for which TEFs have not yet
been established (Reassessment, Part II, section 9.3.5; Part III, section 1.1).
Many of these chemicals have been examined and observed to produce
adverse AHR-dependent effects in vivo (Birnbaum et al. 1991, 2003). In
fact, one mixed polychlorinated and polybrominated dibenzo-p-dioxin (2,3dichloro-7,8-dibromo-dibenzo-p-dioxin) produced AHR-dependent toxic
ity in vivo (wasting and thymic involution) at concentrations up to 10 times
lower than that of 2,3,7,8-TCDD (IPCS 1998a, p. 879, Table 50). Al
though significant information on the polybrominated dibenzo-p-dioxins
and furans (PBDDs and PBDFs) is available and REP values for some of
these compounds have been developed, there still are few toxicological and
environmental distribution studies on these compounds. However, IPCS
(1998a) suggested that development of TEFs for selected PBDDs and PBDFs
is justified given their existing similarities in structure, mechanism, and
potency to PCDDs and PBDFs. There are also many other classes of polyhalogenated chemicals that are known to bind to and activate AHR (poly
chlorinated naphthalenes, benzenes, azobenzenes, azoxybenzenes, and oth
ers), and some of these have also been shown to produce TCDD-like effects.
However, the primary issue for the lack of consideration of these other
TCDD-related compounds in the current assessment is that insufficient
data are available on these chemicals, there are no currently determined or
validated REPs and TEFs, and questions remain about the presence and
persistence of these chemicals in the environment, food, and organisms.
EPA should include these chemicals in the TEQ calculations when validated
TEFs are developed.
Natural and Synthetic Non-DLCs AHR Agonists
Assumptions: Synthetic and natural non-DLC AHR agonists with a
short biological half-life and lower AHR binding affinity do not interfere
with PCDD-, PCDF-, and PCB-dependent TEQ predictions. It has been
recognized for several years that human and animal diets contain relatively
high concentrations of naturally occurring AHR agonists and antagonists
(Denison et al. 2002; Denison and Nagy 2003; Jeuken et al. 2003) and that
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there are non-dioxin-like halogenated aromatic hydrocarbons (HAHs)
(PCBs and PCDFs) that are relatively potent AHR antagonists (described
below). From a pharmacological and receptor binding kinetics point of
view, if one assumes that the binding of these non-DLC agonists or antago
nists to AHR is similar to that of TCDD (that is, binding is essentially
irreversible) (Farrell et al. 1987; Bradfield and Poland 1988; Henry and
Gasiewicz 1993; Brown et al. 1994; Petrulis and Bunce 2000), then the
presence of relatively constant and high concentrations of relatively weak
non-dioxin-like agonists or antagonists in blood and tissue (e.g., from
chronic consumption of relatively high levels of these chemicals) could be
expected to produce AHR-dependent effects or inhibit the overall toxic and
biological effects produced by a defined amount of TEQ calculated from
TCDD-related compounds present in a sample extract.
In most published studies, these metabolically labile non-DLC AHR
agonists do not produce AHR-dependent toxicity; however, a few studies
have reported the ability of some of these chemicals to produce TCDD-like
toxic effects. b-Naphthoflavone (a polycyclic aromatic hydrocarbon [PAH]
AHR agonist) was reported to produce thymic involution and splenom
egaly in “AHR-responsive” C57 but not “AHR-nonresponsive” DBA mice
(Silkworth et al. 1984) as well as wasting and brain developmental effects
in fish (Grady et al. 1992; Dong et al. 2002). Developmental exposure of
rats to indole-3-carbinol (I3C), a naturally occurring AHR ligand that can
be converted in acidic conditions in the stomach into potent AHR agonists,
including the high-affinity AHR agonist indolo-[3,2b]-carbazole (ICZ), was
reported to produce some AHR-dependent reproductive effects similar to
those of TCDD, although other distinct effects of ICZ were noted (Wilker
et al. 1996). In addition, inhibition of cytochrome P450-dependent metabo
lism of PAHs was reported to result in dioxin-like effects in developing fish
embryos exposed to PAHs that are AHR agonists (Wassenberg and Di
Giulio 2004a,b). Not only would inhibition of CYP-dependent metabolism
increase the persistence of the PAH in fish in vivo, but this scenario could
also occur in the environment where organisms are exposed to complex
chemical mixtures. In contrast to the above studies, the naturally occurring
AHR ligand I3C failed to produce adverse effects in rats not only in a 1year dietary chronic exposure study (Leibelt et al. 2003) but also in a high
dose, short-term study with subcutaneously administered ICZ for up to 10
days (Pohjanvirta et al. 2002).
The ability of metabolically labile phytochemicals to induce or inhibit
induction of CYP1A1-dependent activities by TCDD in cell culture model
systems has been reported by numerous laboratories (Williams et al. 2000;
Amakura et al. 2002; Jeuken et al. 2003; Zhang et al. 2003). Moreover,
while the naturally occurring AHR ligands I3C and diindolymethane have
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been reported to inhibit TCDD-dependent induction of CYP1A1 in B6C3F1
mice in vivo (Chen et al. 1995, 1996), ICZ failed to interfere with the
effects of TCDD in a high-dose 10-day study (Pohjanvirta et al. 2002).
Lower-affinity synthetic non-dioxin-like AHR agonists, such as 6-MCDF,
have been observed to inhibit TCDD-induced CYP1A1, teratogenicity,
immunotoxicity, and porphyria in rodent models in vivo (Astroff et al.
1988; Bannister et al. 1989; Harris et al. 1989; Yao and Safe 1989). The
ability of some non-dioxin-like PCBs and PCDFs to inhibit TCDD-induced
CYP1A1 activity and immunotoxicity in C57BL/6J mice has also been
reported (Bannister et al. 1987; Davis and Safe 1988; Biegel et al. 1989;
Chen and Bunce 2004). In addition, administration of a synthetic flavonoid
antagonist of the AHR (3'-methoxy-4' nitroflavone) to transgenic mice was
observed to inhibit TCDD-inducible CYP1A1 and an AHR-responsive pgalactosidase transgene (Nazarenko et al. 2001).
In EPA’s Reassessment, a strong case is made for the distinctiveness of
highly persistent AHR agonists, versus readily metabolized ones, in terms
of toxicological responses and risk assessment. However, the limitation
with regard to the lack of knowledge of the effects of the large number of
naturally occurring and synthetic AHR ligands on the overall toxic po
tency of TCDD-related compounds was acknowledged in the Reassess
ment (Part III, p. 9-40, lines 27 to 28). Although few studies have exam
ined the effects of non-DLC AHR agonists or antagonists on the overall
toxic and biological potency of TCDD-related compounds, a few in vivo
studies do provide supporting evidence that metabolically labile AHR
agonists or antagonists can actually reduce the overall toxic potency of
TCDD and presumably other dioxins and DLCs. On the other hand, an
excellent correlation between the predicted TEQ and the magnitude of the
observed response was observed in several studies examining the effects of
real-world samples (soot, incinerator fly ash, sediment leachate, and fish
or fish extracts) in animals exposed to these samples in vivo (DeCaprio et
al. 1986; Silkworth et al. 1989; Suter-Hofmann and Schlatter 1989; Tillitt
and Wright 1997; Powell et al. 1997). While the occurrence of AHRdependent antagonism by phytochemicals and other AHR antagonists in
humans has yet to be confirmed, given species similarities in the AHR and
AHR signaling pathway and the relatively high concentrations of many
naturally occurring dietary AHR antagonists, the possibility remains that
interactions or interferences between natural AHR agonists and TCDDrelated compounds might occur. Non-DLC AHR agonists could affect the
TCDD-related compounds dose-response relationships for short biologi
cal responses (that is, gene induction) and contribute to an additive re
sponse for the end points. However, the metabolic lability (that is, lack of
persistence) of these compounds prevent them from affecting longer-term
dose-response relationships (including threshold and nonlinear assump-
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tions) for toxic end points, such as cancer. That is one reason for the
Reassessment to focus only on TCDD, other dioxins, and DLCs that are
documented to produce AHR-dependent toxicity. Although these interac
tions would not affect individual TEF values or the calculation of an
overall TEQ determined in controlled laboratory experiments, they could
affect the magnitude and overall toxic effects produced by a defined
amount of total TEQs calculated from intake or present in the body.
Accordingly, EPA should acknowledge in the Reassessment the potential
for non-DLCs to affect the overall biological and toxic potency of a
defined amount of TEQs present in a complex mixture of chemicals and
propose considering these compounds in the overall calculations when
and if sufficient and appropriate in vivo data become available in the
published literature to support their modulatory effect on DLC- and AHRdependent toxicity.
KEY STUDIES AND PUBLICATIONS TO BE INCLUDED
Several relatively recent studies not included in the Reassessment sup
port using the TEF/TEQ approach for noncancer and cancer end points;
their inclusion would greatly strengthen the Reassessment.
• Studies in rats with TCDD or heptachlorodibenzo-p-dioxin (HpCDD)
revealed that the REP derived from acute toxicity studies were the same as
that obtained in a subchronic and chronic toxicity study; both had a TEF of
~0.007 for HpCDD, although no confidence bounds were provided (WHO
TEF = 0.01) (Viluksela et al. 1997a).
• A mixture of four PCDDs or individual PCDDs at equipotent
doses (based on TEFs) to rats produced comparable biochemical changes
after single as well as multiple doses. The authors concluded that TEFs
from acute toxicity studies can accurately predict the toxicity of dioxins,
other than TCDD, and DLC mixtures regardless of whether they are
administered as single compounds or as a mixture, the results support
ing additive toxicity for those compounds (Stahl et al. 1992; Viluksela et
al. 1998a,b).
• Rats given a mixture of two PCDDs, four PCDFs, and two PCBs (in
a ratio found in foodstuffs) at a concentration of 2.0 ^g TEQ/kg of body
weight produced adverse reproductive and developmental effects compa
rable to those at a TCDD concentration of 1 ^g/kg (Hamm et al. 2003). The
authors concluded that the TEQ approach was a reasonable predictor of
the reproductive effects studied.
• Application of TEFs adequately predicted the increased incidence of
liver tumors in rats (hepatocellular carcinoma and cholangiocarcinoma)
induced by exposure to a mixture of TCDD, 3,3',4,4',5-PCB, and 2,3,4,7,8-
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PeCDF compared with an equivalent concentration of TCDD (Walker et al.
2005).
CONCLUSION AND RECOMMENDATIONS
Overall Conclusion
Overall, even given the inherent uncertainties and limitations, the TEF
method, when applied correctly, is a reasonable, scientifically justifiable,
and widely accepted method to estimate the relative toxic potency of diox
ins, other than TCDD, and DLCs on human and animal health.
Specific Conclusions and Recommendations
• AHR-independent mechanisms excluded. AHR-independent effects
of TCDD have been reported, and although their significance and role in
the overall toxic effects remain to be established, the Reassessment should
acknowledge the existence of these AHR-independent effects because fu
ture studies may demonstrate that they play some role in the overall toxic
and biological effects of TCDD, other dioxins, and DLCs.
• Uncertainty of TEF values. A significant degree of uncertainty exists
in the current consensus TEFs, and the quantitative weighting consider
ations that have gone into their establishment are not clear. While the
Reassessment should acknowledge the need for better uncertainty analysis
of the TEF values, extensive and appropriate uncertainty analysis would
take considerable time and effort. Accordingly, the committee endorses the
recommendation of some members of the 2000 EPA SAB Panel “that, as a
follow up to the Reassessment, the EPA should establish a task force to
build ‘consensus probability density functions’ for the thirty chemicals for
which TEFs have been established, or to examine related approaches such
as those based on fuzzy logic” (EPA SAB 2001, p. 29).
• Consistency of REP values. Most in vitro and in vivo studies support
the assumption that the indicated dioxins, other than TCDD, and DLCs are
not only full agonists but that their REP is similar for all end points of
concern and exposure scenarios. However, significant end-point-specific
differences in the REP of some dioxins, other than TCDD, and DLCs have
been reported and whether other differences exist remains to be deter
mined. Consistent with the recommendations of the 2000 EPA SAB Panel,
this committee also suggests that it would be appropriate for the Reassess
ment to note that end-point-specific TEFs/TEQs might be derived as data
become available and that those specific values be used when that end point
is being considered. It should also be made clear that general monitoring or
screening approaches (that is, for TCDD-related compounds in food and
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environmental samples) should use TEF values that are based on REPs
values of all end points.
• Use o f TEFs for D LC body burdens. This is perhaps the greatest
issue of concern in this section of the Reassessment because it remains to be
determined whether the current WHO TEFs, which were developed to
assess the relative toxic potency of a mixture to which an animal is directly
exposed by dietary intake, are appropriate for the assessment of internal
TEQ concentrations and potential toxic effects. The issue was not well
described or well justified in the Reassessment and might be incorrect. It is
further complicated by an EPA paper (DeVito et al. 1997) suggesting that
use of TEFs for DLC body burdens might not be appropriate for some
PCDFs. The issue would be further complicated if toxicokinetic differences
of other DLCs similarly affect their REP. Overall, it remains to be deter
mined whether intake TEFs are appropriate for body burden TEQ determi
nations. If body burdens are going to be used as the dose metric, the
committee recommends that a separate set of body burden TEFs be devel
oped and applied for this evaluation or that the appropriateness of intake
TEFs for body burden TEQs be scientifically justified. Without these cor
rected values, the overall TEQs estimated by use of intake TEFs could be
inaccurate.
• Role o f AHR and additivity of DLCs. These aspects are well de
scribed and well supported by extensive numbers of scientific studies. How
ever, EPA should acknowledge the possibility that AHR antagonists present
in a complex mixture could affect the magnitude and overall toxic effects
produced by a calculated amount of total TEQs present in a given sample
even if they do not affect the TEQ calculations. This issue was not ad
dressed in the Reassessment.
• Rodent-to-human prediction. Although the REP of dioxins, other
than TCDD, and DLCs in rodent models is predictive of REP in humans
from a qualitative rank-order potency point of view, some species-specific
differences in AHR ligand binding affinity of TCDD, other dioxins, and
DLCs have been observed. However, because TEF values are expressed
relative to that of TCDD in the individual species, the TEF values for
dioxins, other than TCDD, and DLCs appear to be similar between species.
If significant differences in the REP of dioxins, other than TCDD, and
DLCs are found between humans and other species, then adjustments
should be made in the TEFs, and these should be acknowledged in the
Reassessment.
• Other AHR agonists.
— Related HAH DLCs. Lack of consideration of other persistent
halogenated chemicals, such as brominated, chlorinated, and mixed chloro
and bromo coplanar chemicals, which clearly exert their toxic and biologi
cal effects in an AHR-dependent manner could result in underestimation of
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
the overall TEQ for a given sample. Although REP values and TEFs have
been developed for some of these chemicals, few studies have been carried
out with most of them, and their relative toxic potency is unknown. Given
the structural similarities and mechanism of action of these chemicals in
vivo and in vitro with the established compounds, as validated REP values
become available, TEFs should be assigned, and these chemicals should be
included in the TEF/TEQ approach. This course of action should be noted
in the Reassessment.
— Synthetic and naturally occurring non-DLC AHR ligands. A
large number of synthetic and naturally occurring non-DLC AHR ligands
have been identified and are present in human diets and presumably in
blood and tissues. The assumption that non-DLC AHR agonists with a
short biological half-life do not interfere with DLC-dependent TEQ predic
tions for mixtures is controversial and remains to be confirmed. Although
receptor binding kinetic evaluations suggest that these chemicals could in
terfere with TCDD, other dioxins, and DLCs if at high concentrations in
blood and tissue, few of these metabolically labile non-DLC AHR agonists
have been observed to directly produce AHR-dependent toxicity. The Reas
sessment makes a strong case for the ability of only highly persistent AHR
agonists to produce toxicity, but the lack of knowledge of the effects ex
erted by the large number of naturally occurring dietary and synthetic AHR
ligands on the overall toxic potency of TCDD, other dioxins, and DLCs still
leaves the question open, particularly with regard to humans. Although
these AHR ligands would not affect TEQ calculations, they could affect the
magnitude of the toxic and biological effect of a defined amount of TEQ.
This point should at least be made clear in the Reassessment, and when a
sufficient number of published studies demonstrate the ability of non-DLC
AHR agonists or antagonists to modulate the overall effects of DLCs, then
EPA should consider how these chemicals would affect the current TEF/
TEQ approach for potency estimates.
• WHO’s plan to reexamine D LC TEFs in 2006. The major issues of
concern described above for the TEF approach will also be the focus of a
meeting of the International Programme on Chemical Safety (announce
ment in IPCS 2004). The issues include (1) considering methods and ap
proaches for deriving TEFs, including quantitative (statistical) methods,
such as establishing an uncertainty range of available REP data and appli
cation of a specified cut-off value to derive TEF values, application of
weighting factors to existing data, and related issues; (2) determining
whether to continue to include mono-ortho PCBs in the present TEF con
cept; (3) considering whether other compounds should be considered for
inclusion in the TEF concept, taking into account the prerequisites for
inclusion outlined by Van den Berg et al. (1998); and (4) determining the
applicability of the use of TEFs to estimate intake versus internal concentra-
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TOXIC EQUIVALENCY FACTORS
89
tions and to what extent could or should internal WHO TEF factors be
established in the future? EPA should consider the outcome of the IPCS
TEF update meeting and incorporate the issues and changes into the Reas
sessment.
• Updating the Reassessment. Although the Reassessment clearly states
that the WHO TEFs of 1998 will be used for assessment and calculation, if or
when TEF values are changed or new chemical TEFs are added by the current
or future WHO TEF panels (such as the 2005 panel), EPA should consider
incorporating the new TEF values and methods for TEQ determination.
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4
E x p o su re A sse ssm en t
The Reassessment1 addresses exposure in terms of sources, environ
mental fate, environmental media concentrations, food concentrations,
background exposures, and potentially highly exposed populations includ
ing important developmental stages. In this chapter, the committee dis
cusses the exposure characterization section provided in the Reassessment,
Part III. Part I of the Reassessment has a wealth of supporting information
and comprises an executive summary and three volumes: Sources o f Di
oxin-like Compounds in the United States2; Properties, Environmental
Levels, and Background Exposures; and Site-Specific Assessment Proce
dures.
ASSESSMENT PROCEDURES
The comments in this chapter are directed specifically at the use of
exposure assessment in the risk assessment provided in Part III of the Reas
sessment, but the committee consulted the more detailed companion docu
ments in Part I for supporting information.
1The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
2Although EPA gave this document the title Sources of Dioxin-like Compounds in the
United States, it provides information on sources of T C D D , other dioxins, and dioxin-like
compounds.
90
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EXPOSURE ASSESSMENT
91
Similar to the Reassessment, Part III, the chapter here is organized into
sections on sources, environmental fate, environmental media and food,
background exposures, and potentially highly exposed populations and
sensitive populations. This chapter has three major sections: an overview
and commentary on all aspects of the dioxin exposure assessment with an
effort to point out strengths, limitations, and omissions; the committee’s
findings; and specific recommendations.
OVERVIEW AND COMMENTARY ON
EPA’S EXPOSURE CHARACTERIZATION
In this section, the committee provides summary and commentary on
key issues related to exposure characterization for 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD, also referred to as dioxin), other dioxins, and
dioxin-like compounds (DLCs). This information includes sources, envi
ronmental fate, environmental media and food concentrations, background
exposures, and potentially highly exposed populations and particularly sen
sitive developmental stages.
For sources and environmental fate, EPA had a clearly articulated
stepwise approach that the committee primarily accepted with some com
mentary. The other steps in the exposure assessment are not as easy to
track, summarize, and critique. To comment on these steps, the committee
used a format that went beyond the simple narrative.
Sources
Summary of the EPA Approach
The type, geographic distribution, and time history of the sources and
associated emission magnitudes of TCDD, other dioxins, and DLCs are
essential inputs for risk characterization. In Part III of the Reassessment,
EPA discusses sources and emissions estimates for 1987 and 1995. More
recently, EPA issued a report that includes the year 2000 update on sources
and emissions estimates (EPA 2005b). These reports consider emissions of
polychlorinated dibenzo-p-dioxin (PCDD) and polychlorinated dibenzofuran (PCDF) compounds and dioxin-like polychlorinated biphenyl (PCB)
compounds. PCDDs and PCDFs have never been intentionally produced
outside research laboratories. They are released to the environment as unin
tended by-products from various combustion, industrial, and biological
processes. PCBs have been produced commercially in large quantities in the
United States and other industrialized countries but are no longer commer
cially produced in the United States and Europe.
Sources of TCDD, other dioxins, and DLCs considered in the Reassess-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
ment include combustion sources; metals smelting, refining, and processing
industries; and chemical manufacturing, biological and photochemical pro
cessing, and reservoir sources. PCDDs and PCDFs are formed in most
combustion systems—waste incineration and burning of coal, wood, and
petroleum products; other high-temperature sources (such as cement kilns);
and poorly or uncontrolled combustion sources (such as forest fires, build
ing fires, and open burning of wastes). PCDDs and PCDFs can be formed
during various types of primary and secondary metals operations, including
iron ore sintering, steel production, and scrap metal recovery. PCDDs and
PCDFs can be formed as by-products from the manufacture of chlorinebleached wood pulp, chlorinated phenols (e.g., pentachlorophenol [PCP]),
PCBs, phenoxy herbicides (e.g., 2,4,5-trichlorophenoxyacetic acid, or 2,4,5T), and chlorinated aliphatic compounds. Recent studies suggest that
PCDDs and PCDFs can be formed under certain environmental conditions
(e.g., composting) from the action of microorganisms on chlorinated phe
nolic compounds. EPA also reported that PCDDs and PCDFs have formed
during photolysis of highly chlorinated phenols.
Reservoir sources of TCDD, other dioxins, and DLCs are materials or
places that contain previously formed PCDDs and PCDFs or dioxin-like
PCBs and have the potential for redistributing and circulating these com
pounds into the environment. Potential reservoirs include soils, sediments,
biota, water, and some anthropogenic materials. Reservoirs become sources
when they release compounds to the surrounding environment.
Important Aspects of EPA’s Approach, Assumptions, and Findings
The key output of the Reassessment regarding sources is provided in
Table 4-2 of the Reassessment, Part III, which summarizes an “inventory”
of sources for the United States expressed as toxic equivalent quotients
(TEQ). In constructing this table, EPA developed a qualitative confidence
rating scheme in which they used qualitative criteria to assign high-, me
dium-, or low-confidence ratings to the inventory classes. This table and
comparisons of the years 1987, 1995, and 2000 are important inputs to
EPA’s conclusions about long-term trends in the emissions of TCDD, other
dioxins, and DLCs (furans and dioxin-like PCBs). In particular, the com
mittee notes that EPA relied more on emissions estimates than environmen
tal and biological media concentrations as a means of characterizing tem
poral trends in exposure to TCDD, other dioxins, and DLCs.
EPA’s use of the inventory table represents a “ bottom-up” approach.
EPA compiled a list of all potentially important source categories and pro
vided an estimate of the probable magnitude of emissions from each of
these categories. Summing these emissions by categories then provides an
overall estimate of current and historical emissions. As noted by EPA, this
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93
approach comes with large uncertainties in assigning emission values to
each category and may exclude an unknown major category or fail to
identify a number of minor categories that together provide large emissions.
An alternative “ top-down approach” would consider levels of PCDD and
PCDF compounds in various environmental media (soils, sediments, and so
forth) or biological media (vegetation, tree bark, fish tissues, and so forth)
and identify the level of emissions required to account for these PCDD and
PCDF levels. The top-down approach uses fate modeling and mass-balance
analyses applied to environmental samples along with a number of assump
tions to determine the extent to which deposition matches emissions (Rappe
1991; Harrad and Jones 1992; Brzuzy and Hites 1995; Eisenberg et al.
1998). Because of discrepancies among estimates of TCDD and related
compounds in reservoirs relative to known sources, several researchers
using a top-down approach concluded that EPA estimates of historical
national emissions might underestimate emissions (Rappe 1991; Harrad
and Jones 1992; Brzuzy and Hites 1995; Eisenberg et al. 1998). This sug
gests the possibility of unknown sources. Although the bottom-up and topdown approaches come with uncertainties, EPA could benefit substantially
from using both approaches simultaneously to set plausible bounds on the
historical and current trends in emissions. The committee recognizes that
each approach has significant limitations. For example, the identification of
ball clay as a potential source represents an interesting case, because it
represents an identified (and managed) new source. In the absence of any
other information, a bottom-up or a top-down approach is unlikely to find
a minor contributor, such as ball clay, to overall national-level TEQ.
One of the most important aspects of the EPA analysis emerges in the
discussion of the trends over time. With the most recent update to the
inventory (EPA 2005b), there were dramatic declines relative to 1995 and
1987 in the emissions of TCDD, other dioxins, and DLCs from identified
major sources. Unfortunately, the Reassessment and the background docu
ments do not provide sufficient information for the committee to review the
emission inventory table inputs, either the qualitative assessments or the
quantitative estimates. In the current organization of the Reassessment,
EPA does not clearly lay out the path for derivation of the emissions num
bers. The lack of clarity makes a task as basic as checking the calculations
and logic difficult.
Environmental Fate
Summary of the EPA Approach
Part III of the Reassessment provides a summary of key findings about
the transport and environmental fate of TCDD, other dioxins, and DLCs.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Another apparent purpose of section 4.2 in Part III of the Reassessment is
for EPA to make clear that assessment of environmental fate cannot be
based on TEQ but must be based on individual congeners, but in section
4.1, EPA presents estimates of environmental releases as TEQ. They elected
to present TEQ in place of mass quantities to better facilitate comparisons
across sources. For purposes of environmental fate modeling, however,
EPA notes that it is important to use the individual PCDD, PCDF, and PCB
congener quantities rather than TEQ, because the physical and chemical
properties of individual congeners vary and will behave differently in the
environment. This material on the need to address specific congeners ap
pears to have been added to the Reassessment in response to the Science
Advisory Board’s comment that the original dioxin reassessment report
(EPA 1994) implied that emissions expressed as TEQ could be used as
source terms for modeling transport, fate, and exposure in risk assessments.
Important Aspects of EPA’s Approach, Assumptions, and Findings
In its assessment of environmental fate in the Reassessment, EPA makes
the following key findings:
• TCDD, other dioxins, and DLCs are widely distributed in the envi
ronment as a result of a number of physical and biological processes.
• Because physical and chemical properties vary substantially among
individual congeners, the congeners will behave differently as they are trans
ported through and transformed in the environment. Thus, for purposes of
environmental fate modeling, it is important to use the individual PCDD,
PCDF, and PCB congener levels rather than TEQ.
• Atmospheric transport and deposition of TCDD, other dioxins, and
DLCs are the primary means of their dispersal throughout the environment.
• The two primary pathways for TCDD, other dioxins, and DLCs to
enter the ecological food chains and human diet are air-to-plant-to-animal
and water-and-sediment-to-fish pathways.
In reviewing these findings, the committee notes that they are sup
ported by the source and exposure information provided in the Reassess
ment. The committee further notes that EPA missed an opportunity to use
data on individual congeners to assess how TEQ changes in time and space.
Moreover, many EPA findings on sources, fate, and exposure tend to be
drawn from temporal and spatial trends in emissions. EPA did not make
full use of exposure media concentration data, particularly food concentra
tion data, to confirm that the space and time trends are reflected in expo
sure media. EPA missed the opportunity to use emissions data for indi
vidual congeners combined with fate modeling to assess the persistence of
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individual congeners to estimate the persistence of TEQ and the spatial
distribution of TEQ. Another issue of interest to the committee is how the
reliability of the TEQ estimate becomes more uncertain with time. Because
of uncertainty about toxic equivalency factors (TEFs) for the more persis
tent congeners, such as the hexa, hepta, and octa chlorinated congeners,
that tend to dominate the TEQ, the reliability of the TEQ characterization
degrades with the resulting accumulation of the more persistent congeners.
As a result of not considering this issue, EPA does not yet have the ability to
determine when reservoir sources will become significant relative to all
anthropogenic sources in characterizing the TEQ of TCDD, other dioxins,
and DLCs.
Environmental Media and Food Concentrations
EPA developed estimates of concentrations of TCDD, other dioxins,
and DLCs in various environmental media, including foods, using only
those studies from locations that they considered as representing back
ground levels of these compounds. The extent to which regions with high
exposures were either captured or excluded is not clear in the Reassess
ment. Moreover, because background has a continuum of low to high
concentrations, it is also not clear where the line was drawn to distinguish
background from “not background.”
Although TCDD, other dioxins, and DLCs in food and environmental
media have been declining over the last three decades, the presence of these
compounds in foods (primarily in animal fats and oils) now represent 90%
or more of human exposure (IOM 2003). However, there are significant
uncertainties inherent in calculating with accuracy or precision dietary ex
posure because of the limited analyses of individual foods; the method
ological improvements over time with corresponding lowering of the limits
of detection; the limited information on the congener composition of vari
ous foods; the values assigned to “nondetects” ; the alterations in concentra
tions of TCDD, other dioxins, and DLCs due to methods of preparation
and cooking; the wide diversity of human dietary composition and con
sumption patterns; and the inherent inaccuracies of the instruments used to
assess dietary intake in humans (IOM 2003). The Reassessment extensively
details the information available at the time (Part I, Volume 2, Chapters 3
and 4) and briefly mentions the Institute of Medicine (IOM) report (Part III,
section 4.3). EPA acknowledges that, in general, the available food data
come from “ studies that were not designed to estimate national back
ground means” and that “it is not known whether these estimates ad
equately capture the full national variability” (Part III, section 4.3).
Since the Reassessment, additional studies have estimated human di
etary intake of TCDD, other dioxins, and DLCs. In the Netherlands, “ the
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estimated median life-long-averaged intake of the sum of PCDDs, PCDFs,
and dioxin-like PCBs in the population is 1.2 pg WHO [World Health
Organization] TEQ per kg of body weight per day” (Baars et al. 2004). The
estimated median is below the WHO tolerable daily intake of 2 pg TEQ/kg
of body weight, and the authors estimated that approximately 8% of the
Dutch population have life-long averaged intakes above the WHO tolerable
intake level. Charnley and Doull (2005) and the U.S. Food and Drug Ad
ministration (FDA) (CFSAN 2005a,b) estimated human food exposures to
TCDD, other dioxins, and DLCs between 1999 and 2003 with data derived
from the FDA total diet study. These studies provided intake estimates since
2001 in which the average daily intake for all age groups fall below the
WHO tolerable daily intake level of 2 pg TEQ/kg of body weight. However
the estimates do not include breast-fed infants.
Charnley and Doull (2005) noted that when assessors represent expo
sure media concentrations of TCDD, other dioxins, and DLCs—primarily
in food—below the limit of detection (LOD) by one-half the detection limit,
“ approximately 5% of the intake estimates for 2-year-olds and 1% of the
intake estimates for 6-year-olds exceed the tolerable daily intake by about
10% .” When these media concentration measurements below the LOD are
set to zero (when only concentration values actually measured are used),
“ only 1% of intake estimates exceed the tolerable daily intake for 2-yearolds.” The committee notes that this reveals the problem of interpreting a
“mean” concentration. The arithmetic mean among individuals in these
cases is quite sensitive to the treatment of samples below the LOD. One
alternative is to avoid the use of sample means and instead consider com
parisons based only on percentile concentrations (e.g., median values and
90th percentile individual values). These percentile values only require in
formation about the rank of a sample and thus avoid the impact on central
value estimates introduced by LOD assumptions.
In both American (Charnley and Doull 2005) and Dutch (Baars et al.
2004) populations, meat and dairy products account for approximately
50% of the TCDD TEQ consumed in food, but the Dutch consume more
TCDD TEQ in fish than do Americans—16% and 5.8%, respectively. Ad
ditional data from the U.S. Department of Agriculture Food Safety and
Inspection Service confirmed that the contents of TCDD, other dioxins, and
DLCs measured in 2002 and 2003 in meat products sold in the United
States, including hogs, steer, heifers, young chickens, and young turkeys,
have declined significantly from the contents measured in 1994 through
1996, although methodological differences preclude a precise calculation of
the decrement (FSIS 2005).
Recognizing that some data gaps will remain in the source inventory, in
the environmental media concentrations describing the distribution and
environmental fate of these chemicals, and in various parts of the food
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chain and human tissue concentrations (e.g., breast-milk and serum con
centrations), the committee notes that it would be helpful if EPA could set
up a congener-specific database of typical concentrations in foods for the
whole range of PCDDs, PCDFs, and dioxin-like PCBs (those included in the
WHO TEF list). Such a database would need to fulfill clear requirements of
data quality and traceability (e.g., chemical analysis, representative and
targeted sampling, data representative consumer exposure, presentation of
data, and handling and presentation of values below the LOD). Making
such a database available could improve the transparency of how EPA
came to some of the conclusions in the Reassessment. Moreover, if TEF
values change, TEQ values can be easily recalculated. Such a database
could be updated on a regular basis to evaluate temporal trends. Here, it is
important to consider methodological aspects (e.g., reproducibility, sensi
tivity, specificity of the analytical determinations, inclusion of reference
samples, and comparable sampling strategy) to ensure that such a time
trend analysis is useful.
Background Exposures
The section of the Reassessment that addresses background exposures
provides a summary of information on human tissue levels, intake esti
mates, and variability in intake levels.
Tissue Levels
The section of the Reassessment addressing tissue levels evaluates
data on concentrations of TCDD, other dioxins, and DLCs in human
tissues expressed per gram of lipid and the changes in these concentra
tions that have occurred in recent decades. The Reassessment acknowl
edges the difficulty of comparing different data sets because some do not
include coplanar PCBs in the estimation of TEQ values. It is clear from
the data in Part III, Table 4-5, that TCDD per se is not the main source of
TEQs in human lipid. The Reassessment uses the calculation of body
burden at steady state along with EPA’s associated assumptions given in
section 1.3 to calculate the TEQ concentration in human lipid based on
the best estimate of current adult intake and the assumption of 25% body
fat. The result is about one-half of that actually measured in human lipid.
EPA assumes that the discrepancy arises from the presence of an historical
body burden and lipid concentration. Given the various assumptions in
the estimation of body burden at steady state, especially in relation to
application of the TCDD model to congener TEFs, it is reassuring that the
TEQ in human lipid predicted by the model are somewhat consistent with
the estimated values.
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Intake Estimates and Variability in Intake Levels
These sections describe intake estimates and the variability and agerelated changes in intake—in particular, by nursing infants.
Potentially Highly Exposed Populations or Developmental Stages
In compiling and evaluating available data on highly exposed popula
tions, EPA considered contamination of food, exposures to workers, and
exposures to nursing infants.
In the Reassessment, EPA assumes that contamination incidents in food
probably have not and will not lead to disproportionate exposures to popu
lations living near where they occurred. The basis for this assumption is
that meat and dairy products in the United States are widely distributed on
a national scale. As a result of this assumption, the Reassessment does not
comment on any disproportionate exposures due to interaction with con
taminated sites.
In considering the distribution of exposures to TCDD, other dioxins,
and DLCs in the U.S. population, EPA suggested that variability in expo
sure probably regresses toward the mean because Americans consume var
ied diets from multiple sources, meaning that EPA assumed that variations
in diet would prevent either very high or very low extremes of exposure.
EPA reported that this pooling of the food supply reduces the potential high
exposure that could result from high consumption of certain food products.
This assumption may be valid, but EPA should provide additional analyses
to support it and should also explicitly consider the possibility of popula
tions who violate the assumptions with respect to varied diets and multiple
sources (e.g., those who rely on home-produced foods or sustenance fish
ing). It is of interest that only in the last paragraph of this section is there
discussion of measurements reflecting potentially highly exposed groups.
Here it is mentioned without further discussion that several European stud
ies showed increased TCDD, other dioxins, and DLC levels in milk and
other animal products near combustion sources. EPA did not consider the
implications of this finding for the U.S. population. It thus seems that EPA
is implicitly assuming that this problem does not exist in the United States.
The Reassessment suggests that no clear evidence demonstrates that
increased exposures to TCDD, other dioxins, and DLCs are currently oc
curring among U.S. workers, but the Reassessment does not document the
level of ongoing monitoring and assessment to support this conclusion.
Low levels of occupational exposure are not congruent with their reported
inventory of sources.
To evaluate the impact of nursing on infants, EPA estimated changes in
body burden with a model developed by Lorber and Phillips (2002). This
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model includes a number of assumptions, including that the fraction of
TCDD, other dioxins, and DLCs absorbed by an infant after ingestion is
0.80 and that the dissipation rate of the ingested TEQ is rapid. The devel
opers evaluated the model with the data (from Germany) of Abraham et al.
(1998). The EPA evaluation does not necessarily confirm the numerous
assumptions (e.g., half-life and uptake). Moreover, the evaluation does not
capture variability or uncertainty in the model because of the assumptions.
The conclusions at the end of the Reassessment, Part III (p. 4-23, lines 7 to
15), include the presentation of model predictions that are implied to be
very precise. Yet, in view of the various assumptions, these results might or
might not reflect reality. In light of the amount of supporting information
available from other sources, it is unclear why EPA relied primarily on a
relatively detailed model with all its inherent uncertainties to report that the
annual infant TCDD-TEQ intake from nursing significantly exceeds the
currently estimated adult intake of 1 pg TEQ/kg/day. This observation can
be easily demonstrated from qualitative findings and simple assessments
based on TCDD half-life and lipophilicity, infant body size, breast-milk
composition, and breast-milk intake. The committee recommends that EPA
consider the value and availability of any data to confirm this modeling
result.
COMMITTEE FINDINGS
Is EPA’s Exposure Assessment Scientifically Robust?
In preparing its findings, the committee notes that those who will make
use of the Reassessment are likely to be interested in issues beyond risk
characterization and risk assessment methodology. For example, some us
ers will want to use the Reassessment to decide whether U.S. exposures to
TCDD, other dioxins, and DLCs pose an undue health risk, whereas others
will want to use the Reassessment to consider alternatives for reducing
exposures to these compounds and identifying strategies for achieving re
ductions of TCDD-TEQ burdens in the U.S. population. In preparing its
findings, the committee considered a range of potential uses for the Reas
sessment—including the following alternatives.
Source Characterization
Clearly, an important opportunity that EPA overlooks is checking the
observed decline in overall environmental concentrations against body bur
den changes over time. For example, the emissions estimates for PCBs and
mass-balance evaluation provided recently by Breivik et al. (2002a,b) pro
vide a better opportunity to consider global-scale chemical PCB fate by
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comparing model results with measured concentrations of PCBs at moni
toring stations located in regions of the Northern Hemisphere over the 70year period from 1930 to 2000. Calculations based on this 70-year estimate
of emissions will introduce uncertainties, but such an analysis could build
confidence about trends and better inform future investigation.
EPA did not fully address the issue of reservoir sources or explore their
potential impacts on the long-term distribution of TCDD, other dioxins,
and DLCs as well as the distribution of TCDD TEQ. It also did not fully
consider how reservoir effects vary among different congeners and thus
cause the TEQ from reservoir sources in soil and sediments to evolve and
change in time. Finally, EPA did not address the issue of when reservoir
sources are likely to become dominant relative to anthropogenic sources.
For example, some studies provide experimental evidence for how TCDD,
other dioxins, and DLCs are incorporated in soil and then reemitted (Brzuzy
and Hites 1995, 1996; Cousins et al. 1999a,b; Cousins and Mackay 2001;
McKone and Bennett 2003).
One of the most important aspects of the analysis emerges in the discus
sion of the trends over time. Given the importance of properly estimating
TEQ and the need for risk analysts to consider the impacts of exposure
timing for some potential dose metrics, the EPA inventory should yield
estimated TEQs associated with each identified source more transparently.
Part III of the Reassessment and the background documents do not provide
sufficient information for the committee to review the emissions inventory
table inputs, either the qualitative assessments or the quantitative estimates.
Although inventories shifted over time with the identification of new
sources, EPA did not examine the extent of that shift.
Environmental Fate Assessment
EPA’s finding regarding the wide distribution of TCDD, other dioxins,
and DLCs is supported by environmental sampling. There have been suffi
cient measurements to conclude that, as a chemical class, these compounds
are widely dispersed in the environment. With regard to individual conge
ners of PCDDs, PCDFs, and dioxin-like PCBs, a sufficient number of
samples are not available to conclude that each individual congener is
widely dispersed in the environment.
Although consideration of individual PCDD, PCDF, and PCB congeners
would be informative and useful, doing that for more than 200 congeners
would be excessive; summing up mass quantities instead of TEQ contributions
would be equally bad, and most other inventories (e.g., in Europe and Japan)
were also done in TEQs. According to the Reassessment (Part III, p. 1-8), five
congeners contribute approximately 80% of the total TEQ in humans: 2,3,7,8TCDD, 1,2,3,7,8-PCDD, 1,2,3,6,7,8-hexachlorodibenzo-p-dioxin, 2,3,4,7,8-
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PCDF, and PCB126. Thus, it would be informative to provide congener-spe
cific emissions estimates for these congeners in place of the TEQ estimates.
Environmental Media and Food Concentrations
In reviewing the EPA assessment of environmental media and food
concentrations, the committee had the following concerns:
• In using food concentration data to estimate intake, the choice of
LOD has significant impact on calculated mean values. EPA was not clear
about (1) how it made use of values below the LOD in making intake
estimates based on food concentration data, and (2) how its treatment of
the LOD had an impact on results. Because the committee found no basis
for making recommendations on other aspects of the food intake calcula
tion and because food supply issues are covered in the IOM (2003) report,
the committee elected to focus on the LOD issue.
• EPA did not make clear its criteria for distinguishing background
from non-background concentrations.
• Relative to dioxin and furan congeners, data on environmental me
dia and food concentrations of dioxin-like PCBs were generally lacking.
• TCDD-TEQ intake estimates from fish consumption did not include
direct consumption of fish oils.
The committee finds value in EPA’s establishing a congener-specific
database of typical concentrations for the range of PCDDs, PCDFs, and
dioxin-like PCBs (those included in the WHO TEF list). The details of such
a database are described above in the overview and commentary section in
the subsection on environmental media and food concentrations.
Estimates of Background Exposures
The committee found the text in this section noncontroversial and the
conclusions valid. The committee did not find any important errors in this
text, but issues arose concerning the interpretation of the background expo
sure data. It is not clear that the existing database as used by EPA covers all
foods consumed by the U.S. population (e.g., data were missing on fish
oils). It would be helpful to include or reference in the exposure estimates
the most recent data on food intake as produced by FDA. The committee
believes that EPA can make more efficient use of the existing data sets on
occurrence in foods and on food consumption to assess the distribution of
intakes of TEQ for the general U.S. population (different age groups, ex
pressed in picogram per kilogram of body weight per day) as well as intraand inter-person variability.
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Exposures in Highly Exposed Populations or at Key
Developmental Stages
In compiling and evaluating available data on highly exposed popula
tions, EPA failed to draw informative conclusions from the numerous stud
ies described in the full document (Part I). Part III of the Reassessment is
missing a summary that integrates the information compiled in Part I.
The Reassessment makes statements discounting the potential for hav
ing highly exposed groups without clearly documenting the basis for these
statements. First, it suggests that, with regard to the commercial food sup
ply, the incidents of contamination by TCDD, other dioxins, and DLCs are
likely to be low. Yet the Reassessment provides no formal assessment and
almost no data to support that determination. It also states that there is no
clear evidence that increased exposures are occurring among U.S. workers.
Finally, it reports that no or few studies show evidence of groups in the
United States being exposed to highly increased levels of TCDD, other
dioxins, and DLCs in situations in which people consume large quantities
of foods with high levels of these compounds. In spite of giving substantial
attention to nursing infants as a highly exposed group, EPA provides no
comment on the potential level of increased exposure that may have arisen
during recent contamination episodes involving the commercial food sup
ply (e.g., the ball clay incident and high levels in beef and dairy animals due
to PCP-treated wood).
Is There a Clear Delineation of All
Substantial Uncertainties and Variabilities?
Overall, the committee finds that EPA has qualitatively identified a
number of important uncertainties and variabilities. However, there are
some areas for which even the qualitative information provided by EPA
was unclear or incomplete. What is more important is that the Reassess
ment does not quantitatively characterize either variability or uncertainty in
exposure except in the limited sense of demonstrating increased average
daily dose estimates for children (on a body-weight basis) and analyzing
potentially increased exposures for nursing infants during their first few
years of childhood.
Source Characterization
The magnitude, type, geographic distribution, and time history of
TCDD, other dioxins, and DLC sources are essential components for risk
characterization. The interpretation of these factors is an important input
to decisions about managing both new and historical (reservoir) sources.
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Any errors in interpretation could lead to policies and regulatory actions
that are inefficient or ineffective in reducing human exposures to TCDD,
other dioxins, and DLCs. EPA exposure characterization excludes basic
data quality checks that could provide an opportunity to evaluate key
assumptions. The committee notes that EPA did not explore an alternative
top-down approach in an effort to evaluate the results from its bottom-up
approach for source-to-intake characterization. The Reassessment clearly
notes significant uncertainties in estimates of emissions and communicates
these uncertainties using qualitative confidence scores (A, B, and C). How
ever, given this clear acknowledgment of significant uncertainties in the
emission estimates, the committee questions the reliability of the
Reassessment’s trend analysis of emissions from 1987 to 2000. EPA does
not communicate these uncertainties in the Reassessment’s summary and
other sections where the trend analysis of emissions is discussed
Environmental Fate Assessment
EPA’s finding that atmospheric transport and deposition of TCDD,
other dioxins, and DLCs are a primary means of their dispersal throughout
the environment is strongly supported by theoretical models in combina
tion with observations of these compounds globally that are more uniform
than emissions sources and far from regions of release. However, there is
considerable uncertainty about the nature and magnitude of the re-emission
process that takes place after deposition.
The EPA finding that the two primary pathways for TCDD, other
dioxins, and DLCs to enter the food chain and human diet are air to plant
to animal and water and sediment to fish is supported by environmental
sampling, but significant uncertainty remains about mechanisms and rates
of transfer through food webs. There have been sufficient measurements to
conclude that, as a chemical class, TCDD, other dioxins, and DLCs, par
ticularly the more persistent ones, enter humans primarily through animal
products and fish. With regard to individual congeners of PCDDs, PCDFs,
and dioxin-like PCBs, samples are insufficient to conclude that each indi
vidual congener enters humans by these two primary pathways.
The committee concurs with EPA that, although it is appropriate to use
TEQ as a metric of release, it must clearly emphasize the uncertainty and
limitation of using the TEQ approach.
Environmental Media and Food Concentrations
• It is uncertain whether the existing information on background lev
els in environmental media and food adequately captures the full national
variability.
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• The effect of cooking and processing on concentrations of TCDD,
other dioxins, and DLCs in foods was considered too limited to draw firm
conclusions.
• EPA had only limited access to data that support any conclusions
about temporal trends in the occurrence of TCDD, other dioxins, and
DLCs in environmental media and foods.
Estimates of Background Exposures
When EPA assumes that less-than-LOD samples equal zero, there are
often significant differences between values of TEQ-based estimates of back
ground intake compared with estimates obtained when EPA assumes that
less-than-LOD samples equal half or the whole detection limit. This illus
trates the importance of analytical method sensitivity in limiting the ability
to determine the full range of population variation of PCDDs, PCDFs, and
dioxin-like PCBs in human and other tissues. EPA missed the opportunity
to quantify the effect of these differences in an uncertainty analysis of the
current exposure estimates.
Exposures in Highly Exposed Populations or at Key
Developmental Stages
As noted above, it is not clear to the committee why EPA relied entirely
on a model with all its inherent uncertainties to conclude that the annual
infant intake of TCDD, other dioxins, and DLCs from nursing significantly
exceeds the currently estimated adult intake of 1 pg TEQ/kg/day. EPA
failed to provide any measurements or environmental samples to support
the conclusions drawn from the model. Providing this information would
increase the confidence in its conclusions on this issue.
Major Assumptions
With regard to sources and emissions, the most appropriate way to
characterize historical sources of TCDD, other dioxins, and DLCs is to
compile a list of all known sources, make emissions estimates for each class
from the available literature, and then combine these emissions to establish
historical trends. The committee finds this assumption reasonable and suf
ficiently documented but finds that it would be valuable for EPA to con
sider alternative approaches (e.g., the top-down approach) for confirming
or revising this approach.
In its consideration of highly exposed subpopulations, EPA found in
formation indicating that breast-feeding might result in higher TCDD-TEQ
body burdens of the nursing infant compared with those of non-nursing
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infants. The issue that exposure of the developing infant is already starting
during pregnancy (in utero exposure) is not addressed in this section or not
clearly mentioned in the full Reassessment. EPA did not consider this infor
mation in their overall conclusions about exposure. Moreover, because of
the potential for causing anxiety among nursing mothers, EPA should ex
pand its discussion about the multiple known benefits of breast-feeding as a
footnote to the section describing exposures to nursing infants.
Modeling Assumptions
In characterizing exposures, EPA relied primarily on measurements
combined with assumptions for emissions and relied almost completely on
measurements of environmental and tissue levels for estimating exposure
and body burdens. With the exception of their toxicokinetic model for
nursing mothers, they did not rely on models for assessing transport and
distribution from sources to environmental (such as air, water, and soil)
and exposure (food products) media.
EPA’s finding that, for purposes of environmental fate modeling, it is
important to use the individual PCDD, PCDF, and PCB congener values rather
than TEQ is self-evident and robust. The committee concurs with the EPA
finding that TEQ should not be used in place of individual congener concentra
tion as the variable in fate models for TCDD, other dioxins, and DLCs.
Were the Most Appropriate Studies Relied Upon?
For characterizing emissions, EPA developed a comprehensive inven
tory of all known emissions of TCDD, other dioxins, and DLCs but did not
fully characterize the work of those researchers who looked at a top-down
approach for characterizing historical emissions of PCDD and PCDF com
pounds. Rappe (1991), Harrad and Jones (1992), Brzuzy and Hites (1995),
and Eisenberg et al. (1998) used fate modeling and mass-balance analyses
applied to environmental samples and a number of assumptions to deter
mine the extent to which deposition matches emissions.
CONCLUSIONS AND RECOMMENDATIONS
• To assess the total magnitude of emissions of TCDD, other dioxins,
and DLCs, EPA used a bottom-up approach in which they attempt to
identify all source categories and estimate the magnitude of emissions for
that category. EPA also should use a top-down approach that attempts to
account for observed levels and consider what emissions would be required
to account for these levels. These alternative approaches give rise to signifi
cantly different estimates of the historical levels of emissions. Both ap-
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proaches come with uncertainties. Thus, the readers of the Reassessment
could benefit substantially from EPA using both approaches simultaneously
to set plausible bounds on the historical and current trends in emissions.
• EPA needs to be explicit about how they dealt with measurements
below the LOD in environmental and exposure media samples. Whether
the less-than-LOD samples are assumed to be zero, assumed to be one-half
LOD, imputed by fitting a censored regression model, or dealt with by
using some other assumption could have significant impacts on estimates of
TCDD, other dioxins, and DLC intakes. EPA should explicitly address how
its assumption affects the magnitude and range of estimated intakes relative
to alternative approaches. Moreover, EPA should describe how the chang
ing LOD affects its estimate of the time trend of TEQ intake.
• Because many users of the Reassessment will be interested in reduc
ing exposures to TCDD TEQ and identifying strategies for achieving reduc
tions in body burden, EPA should add some discussion in the exposure
chapter about what factors (such as diet, activities, and location) tend to
increase or decrease TEQ intake.
• EPA should construct their reports so that information in the sum
mary emissions inventory table of Part III can be more clearly and more easily
traced back to the source chapters that provide background information.
• EPA should evaluate the impact on early emission-inventory esti
mates (1987, 1995) of sources added in more recent assessments (2005) so
that the overall percentage declines reflect all sources. Such an evaluation
would help to confirm dramatic decreases in TEQs that appear to have
occurred over time.
• EPA should define a strategy for collection of samples and reanalysis
of archived samples to answer a number of remaining questions about
exposure trends and to fill in some important data gaps. (The committee
does not consider it particularly useful or cost-effective for EPA to obtain
and analyze more environmental media samples for the full range of TCDD,
other dioxins, and DLCs.)
• EPA should create a congener-specific and active database of typical
concentrations for the whole range of PCDDs, PCDFs, and dioxin-like
PCBs (included in the WHO TEF list). This recommendation applies to
work separate from the Reassessment. The database should be based on a
compendium of all available data and be updated on a regular basis with
new data as they are published in the peer-reviewed literature. Maintaining
the database would not require EPA to conduct its own sampling program.
Such a database would need to fulfill clear requirements of data quality and
traceability, including chemical analysis, representative and targeted sam
pling, data representative of consumer exposure, presentation of data, han
dling, and presentation of less-than-LOD samples.
• In view of the number of sites with increased levels of PCBs in the
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EXPOSURE ASSESSMENT
107
environment and anticipating that those levels could result in higher contri
butions of the dioxin-like PCB fraction to total TEQ exposure (e.g., through
local fish consumption), EPA should explicitly characterize the variability
of population exposures to PCBs. EPA should estimate the magnitude of
the ratio of high-end to median and mean exposure, the factors (e.g., prox
imity to sources, geographic region, and eating habits) that give rise to high
end exposure, and the relative uncertainty with which high-end exposures
can be estimated.
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5
C an ce r
This chapter reviews the U.S. Environmental Protection Agency (EPA)
assessment of the carcinogenicity of 2,3,7,8-tetrachlorodibenzo-p-dioxin
(TCDD), commonly referred to as dioxin, other dioxins, and dioxin-like
compounds (DLCs), including EPA’s qualitative characterization of their
carcinogenicity, the assumption that the dose-response relationship is lin
ear, and the use of animal bioassay and epidemiological data to quantify
the dose response. The final section summarizes the committee’s conclusions.1
QUALITATIVE EVALUATION OF CARCINOGENICITY
EPA concludes that dioxin is “carcinogenic to humans” based on the
following evidence (Reassessment, Part III, pp. 6-7 to 6-8): evidence from
the occupational cohort studies that dioxin exposure increases mortality
from cancer aggregated over all sites and from lung cancer “ and, perhaps,
other sites” ; evidence from bioassays of cancer in both sexes of multiple
species at multiple sites; and evidence regarding dioxin’s mode of action,
including mechanistic evidence that dioxin acts as a tumor promoter via
receptor-mediated pathway(s) and the finding that the receptor-mediated
pathways that may give rise to cancer in laboratory animals appear to be
present and functional in human tissues.
^-The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-Dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
108
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In this chapter, the committee reviews the epidemiological, bioassay,
and mode of action evidence and then presents conclusions regarding both
qualitative and quantitative measures of carcinogenicity of TCDD, other
dioxins, and DLCs.
Epidemiological Evidence
The epidemiological evidence that provided the basis for EPA’s assess
ment consists primarily of studies following four cohorts. Of these, the
Reassessment reviewed in detail those related to the three cohorts that
provided quantitative dose-response estimates linking serum dioxin to can
cer mortality (Ott and Zober 1996; Becher et al. 1998; Steenland et al.
2001). The cohorts were quite variable in size and exposure ranges. Ott and
Zober (1996) studied a relatively small number of men exposed to an
accidental release of dioxin in 1953 (N = 243, 13 cancer deaths). Becher et
al. (1998) examined a cohort of 1,189 men employed in pesticide and
herbicide production, from which 124 cancer deaths were identified. The
third cohort represents a large occupational population originally studied
by Fingerhut et al. (1990, 1991), who examined 5,172 male employees in
12 manufacturing facilities. An update on this cohort was provided by
Steenland et al. (1999), who applied “job-exposure matrix”2 estimates to
5,132 workers in the original cohort who were followed for 6 more years.
The total number of cancer deaths in this cohort was 377. In 2001,
Steenland et al. updated this study again on a subcohort of 3,538 workers
(with 256 cancer deaths) and used data from 170 members of this cohort
for which estimated external exposures and known serum dioxin levels
were available to establish a quantitative dose-response assessment.
Each study identified a cohort of workers who had been employed in
industrial settings in which dioxin was a by-product. These settings in
cluded pesticide production (Ott and Zober 1996; Becher et al. 1998) or
chemical plants more broadly (Steenland et al. 2001). In each instance,
current serum dioxin measurements were available for a subset of workers.
Development of exposure estimates for the entire cohort required two ex
trapolations: from current serum dioxin measurements to historical expo
sure levels using estimates of serum dioxin half-life, and from workers with
current serum dioxin measurements to those without by linking available
serum dioxin measurements to job characteristics based on knowledge of
the industrial processes. Although these extrapolations decrease the accu-
2A “ job-exposure m atrix” refers to an algorithm by which experience in particular jobs are
assigned estimated exposure levels. Each job (a row of the matrix) has a corresponding series
of exposure levels assigned (columns).
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
racy of the assessment, they were necessary to provide historical exposure
estimates so that there would be a sufficient number of cohort members
who could be included in the analysis.
In addition to these three cohorts, Part II, section 7.5.4 of the Reassess
ment describes studies that reported on an occupational cohort of 2,310
workers in two plants that prepared and manufactured phenoxy herbicides
in the Netherlands (see Reassessment, Part II, Table 7-21 for a summary of
all four studies). Bueno de Mesquita et al. (1993) found no statistically
significant increases in cancer mortality among all workers (31 deaths,
standardized mortality ratio [SMR] = 107, 95% confidence interval [CI] =
73 to 152) and among a subset of 139 workers involved in a 1963 indus
trial accident (10 deaths, SMR = 137, 95% CI = 66 to 252). Comparing
exposed workers (N = 963) to unexposed workers (N = 1,111), both total
cancer mortality (rate ratio, [RR] = 1.7, 95% CI = 0.9 to 3.4) and respira
tory cancer mortality (RR = 1.7, 95% CI = 0.5 to 6.3) were nonsignificantly
increased.
A follow-up study by Hooiveld et al. (1996) reported a statistically
significant increase in cancer mortality among workers in one of the two
plants (SMR = 146, 95% CI = 109 to 192). No such increase was observed
in the other factory. Follow-up analysis by Hooiveld et al. (1998) reported
a statistically increased incidence of malignant neoplasms among 140 work
ers involved in the 1963 industrial accident (SMR = 1.7, 95% CI = 1.1 to
2.7). The incidence of malignant neoplasms was also increased in a larger
group of 549 workers (SMR = 1.5, 95% CI = 1.1 to 1.9). A comparison of
this group of 549 exposed workers to 482 unexposed workers, also from
this cohort, yielded an increased total cancer mortality risk (RR = 4.1, 95%
CI = 1.8 to 9.0) and an increased respiratory cancer mortality risk (SMR =
7.5, 95% CI = 1.0 to 56.1).
There are three major issues to consider regarding EPA’s review of the
epidemiological studies investigating the relationship between dioxin expo
sure and cancer. First, although EPA identified the cohort studies capable of
generating quantitative dose-response information for the dose-response
modeling and considered the broader epidemiological literature in the back
ground documents, Part III of the Reassessment did not provide a thorough
and systematic analysis of the body of epidemiological evidence from which
these three studies were chosen. In particular, although Part II described the
complete array of studies, including those by Kogevinas et al. (1997) and
Bertazzi et al. (1998), the Reassessment did not analyze site-specific tumors
consistently across all studies but rather emphasized the positive findings in
each paper without a full discussion of consistency, or lack thereof, across
studies.
A second issue is EPA’s decision to focus on total cancers instead of
specific types of cancer. EPA argues that because dioxin is not genotoxic
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and is instead presumed to act primarily as a promoter rather than an
initiator of cancer, the lack of specificity in tumor type is to be expected. If
dioxin promotes cancer through the Ah receptor mechanism, however, then
an increased tumor incidence would require expression of the receptor in
that tissue. The Ah receptor is expressed in most tissues but to varying
degrees. It is uncertain whether the level of expression is an important
determinant of tumor promotion. There are also many downstream events
from ligand-receptor interaction that are tissue specific and essential for
tumor promotion to occur via a receptor-mediated response, and these
downstream events differ from tissue to tissue (see also discussion on mode
of action later in this chapter). In any case, EPA reasons that, in the face of
limited power, increased risk of total cancers (which would reflect the
increased incidence across the multiple sites affected by dioxin) is easier to
detect than an increased risk of individual cancer types (see Part III, pp. 2
9 to 2-10). This rationale would be valid for a given relative risk (e.g., a
doubling of the incidence or mortality). However, a given absolute incre
mental risk (e.g., an additional 10 cancers due to exposure) would be more
readily identified for a specific cancer site than for cancers in the aggregate.
The more compelling argument for aggregating across cancer types is
the practical one that the results for specific cancers are extremely imprecise
in these cohorts of modest size. If, in fact, multiple cancer types all showed
a small increment in risk of equal magnitude, there would be greater preci
sion and statistical power for the aggregation. For example, in the case of
ionizing radiation, the aggregation across a series of radiosensitive cancers,
each with small increases in risk, yields a more statistically precise indica
tion of an increase in cancers related to radiation exposure than do any of
the individual cancers. In the case of dioxin, it is not clear that a specific set
of cancers is affected that can then be aggregated to enhance statistical
power.
To evaluate the patterns across cancer sites, the committee examined
selected papers from the three cohorts (Ott and Zober 1996; Flesch-Janys et
al. 1998; Steenland et al. 1999). This evaluation revealed that only limited
information is available regarding numbers of cases at specific sites, hence
limiting the opportunity to examine consistency across studies. As noted by
others, there is some consistency across studies for respiratory cancers, but
there is a general lack of concordance for the other cancer sites reported in
more than one study. The degree of replication or lack thereof should not
be overstated given the small number of studies and imprecise information
on specific cancer sites from all but the Steenland et al. (1999) report.
Overall, the committee concurs with the value of conducting analyses
of total cancers, given the potential for dioxin to affect multiple types of
cancer and the limited precision of risk estimates for individual cancer
types. Nonetheless, the potential for effects limited to specific types of
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
cancer, as has been found for other causes, also warrants an analysis of
major cancer types (e.g., respiratory cancers), the imprecision notwith
standing.
Another concern is the potential role of confounding by lifestyle factors
such as smoking and by occupational exposures that co-occur with dioxin.
Although smoking is a powerful lung carcinogen, quite capable of generat
ing spurious relative risks on the order of those reported in the epidemio
logical studies for dioxin of around 1.5, the design of those studies makes
its potential role as a confounder unlikely in this case. The key comparisons
were not between industrial workers and the general population, which is
quite susceptible to confounding by lifestyle factors, but among subsets of
workers with different levels of estimated dioxin exposure. It is not likely
that smoking histories would differ markedly among men located at differ
ent jobs within the industrial plant or in relation to duration of employ
ment. In contrast, there is greater potential for confounding by other work
place agents given that the industrial cohorts had exposure to pesticides and
potentially carcinogenic chemicals in addition to dioxin. Although these ac
companying workplace hazards likely differed for the three cohorts that
contributed to the quantitative risk assessment, confounding could have oc
curred in each to yield a similar falsely elevated measure of association. The
difficulties in isolating the health effects of single agents from the complex
mixtures encountered in chemical manufacturing must be recognized.
Epidemiological evidence for an association between cancer and expo
sure to DLCs has been characterized as “inadequate but suggestive” (EPA
1987) and “ limited” (IARC 1997). ATSDR (2000) concluded that the epi
demiological evidence “taken in totality, indicates a potential cancer caus
ing effect for PCBs.”
On the whole, it was the committee’s impression that EPA’s narra
tive in discussing epidemiological studies in Part III of the Reassessment
tended to focus on positive findings without fully considering the
strengths and limitations of both positive and negative findings. Part III
of the Reassessment would be strengthened if EPA clearly identified
specific inclusion criteria for those studies for which quantitative risk
estimates were determined.
Bioassay Data
Several large and well-conducted dioxin-related cancer bioassays
(Kociba et al. 1978; NTP 1982a,b; NTP 2004) have reported induction of
several types of cancer in both rats and mice. The study in hamsters was
confounded by use of dioxane, which is a potential carcinogen, as the
delivery vehicle. Table 5-1 summarizes these studies. In all studies in which
dioxin elicited an increase in tumors, the increase was site specific. With
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oral administration, the organ most frequently affected was the liver, re
flecting the mode of action of carcinogenicity, as discussed below.
Of the 21 DLCs of concern, EPA (Part II, p. 6-30) reported that carci
nogenicity bioassays have been conducted on only two pure polychlori
nated dibenzo-p-dioxin (PCDD) and 1,2,3,7,8-pentachlorodibenzo-p-dioxin
(PeCDD), and a mixture of two congeners (1,2,3,6,7,8- and 1,2,3,7,8,9hexachlorodibenzo-p-dioxin [HxCDD]). Carcinogenicity bioassays have
also been conducted on one polychlorinated dibenzofuran (PCDF)
(2,3,4,7,8-pentachlorodibenzofuran [PeCDF]) and one PCB (126; 3,3',4,4',
5-pentachlorobiphenyl (PeCB) (Table 5-2).
However, the ability of a variety of dioxins other than TCDD and
DLCs to enhance the carcinogenicity of known carcinogens (promoter as
says) has also been reported for PeCDD, HpCDD, 2,3,7,8-tetrachlorodibenzofuran (TeCDF), PeCDF, and 1,2,3,4,7,8-hexachlorodibenzofuran
(HxCDF) (summarized by IARC 1997). Bioassays have also been con
ducted on mixtures of PCBs, and although they provide some information
on the carcinogenicity of components, they do not identify the specific
responsible chemical(s).
Mode of Action
Dioxin does not have structural features that would lead to a reactive
electrophile, and it is clearly not DNA reactive, as no DNA binding or
adducts were found in rodent tissues (Poland and Glover 1979; Randerath
et al. 1988; Turteltaub et al. 1990). Absence of DNA reactivity is supported
by negative findings in genetic toxicological assays (IARC 1997).
Nevertheless, EPA notes (Part II, p. 6-1) the hypothesis that dioxin
might be indirectly genotoxic, either through induction of oxidative stress
or by altering the DNA damaging potential of some endogenous com
pounds, including estrogens. No evidence is available for estrogen-medi
ated DNA damage resulting from dioxin exposure, but oxidative DNA
damage has been documented after 30 weeks administration of dioxin
(Tritscher et al. 1996; Wyde et al. 2001). Indirect genotoxicity has been
postulated to initiate carcinogenicity, but there is insufficient evidence that
dioxin has initiating activity.
Dioxin was reported to have weak initiating activity in one study
(DiGiovanni et al. 1977) in which it was applied to mouse skin prior to a
promoting agent. This finding has not been corroborated, and in contrast
to what would be expected from an initiating agent, application of dioxin
to mouse skin at a dosage greater than that required for a promoting effect
did not induce skin tumors (Poland et al. 1982).
Moreover, dioxin has not been specifically tested as an initiator in
standard models in rat or mouse liver in which chemicals can be evaluated
Copyright © National Academy of Sciences. All rights reserved.
4^
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TABLE 5-1 Dioxin Cancer Bioassays
Species/Strain
Route and Dose
Sex
Sites o f Tumor Increases
Reference
Rat/SpragueDawley
Oral in feed 1, 10,
100 pg/kg/day
Male
Oral cavity
Kociba et al. 1978
Female
Lung, oral cavity, liver
NTP 1982a
Gastric instillation 10,
50, 500 pg/kg/week
for 104 weeks
Male
Thyroid
Female
Liver
Rat/SpragueDawley
Gastric instillation 3, 10,
22, 46, or 100 mg/kg, 5
days/week for 104 weeks
Female
Liver, lung, oral cavity, uterus
NTP 2005
M ouse/B6C3Fl
Gastric instillation 0.01,
0.05, 0.5 mg/kg/wk for
104 weeks (males)
Male
Liver
NTP 1982a
0.04, 0.2, 2.0 mg/kg/wk
for 104 weeks (females)
Female
Liver, thyroid
Topical application 0.005
pg 3 days/week for 104
weeks
Female
Skin
Rat/Osborne
Mendel
Mouse/Swiss
Webster
NTP 1982b
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h-A
h-A
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Intraperitoneal injection
1, 30, 60 pg/kg/week for
5 weeks
Male
Thymus (both), liver (B6C3 only)
Female
Thymus (both), liver (B6C3)
Gastric instillation 2.5,
5.9 pg/kg/week for 52
weeks
Male
Liver
Female
Liver
Mouse/Swiss
Gastric instillation 0.007,
0.7, 7.0 pg/kg/week for
52 weeks
Male
Liver
Toth et al. 1979
Mouse/TG.AC
Topical application for
24 weeks
Male
Skin papillomas
Eastin et al. 1998
Female
Skin papillomas
Gastric instillation 250
pg/kg, 1,000 pg/kg twice
weekly for 24 weeks
Male
None
Female
None
Intraperitoneal or
subcutaneous injection
50 or 100 pg/kg every
4 weeks
Male
Skin
Mouse/B6C3
Mouse/TP53+/~
Hamster/Syrian
Golden
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Mouse/B6C3
and B6C
DellaPorta et al. 1987
DellaPorta et al. 1987
Eastin et al. 1998
Rao et al. 1988
h-A
h-A
■ -o
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
TABLE 5-2 TCDD, Other Dioxins, and DLC Cancer Bioassays
Congener
B ioassay
D ioxins
2 .3 .7 .8 - TC D D
1 .2 .3 .7 .8 - PeCDD
1 .2 .3 .4 .7 .8 H xC D D
1 .2 .3 .6 .7 .8 H xC D D
1 .2 .3 .7 .8 .9 H xC D D
1 .2 .3 .4 .6 .7 .8 H pC D D
1 .2 .3 .6 .7 .8 - and 1,2 ,3 ,7 ,8 ,9 -H xC D D m ix
O CD D
R at (M ,F)/m ouse (M,F)
R at (M ,F)/m ouse (M ,F)/prom oter rat (F)
N o b ioassay conducted
Com bination study
Com bination study
Prom oter rat (F)
R at (M ,F)/m ouse (M,F)
N o b ioassay conducted
Furans
2 .3 .7 .8 - TC D F
1 .2 .3 .7 .8 - PeCDF
2 .3 .4 .7 .8 PeCDF
1 .2 .3 .4 .7 .8 H xC D F
1 .2 .3 .6 .7 .8 H xC D F
1 .2 .3 .7 .8 .9 H xC D F
2 .3 .4 .6 .7 .8 H xC D F
1 .2 .3 .4 .6 .7 .8 H pC D F
1 .2 .3 .4 .7 .8 .9 - H pC D F
OCDF
Prom oter m ouse (F)
N o b ioassay conducted
R at (F)/prom oter m ouse (F) and rat (M)
Prom oter m ouse (F)/rat (M)
N o b ioassay conducted
N o b ioassay conducted
N o b ioassay conducted
N o b ioassay conducted
N o b ioassay conducted
N o b ioassay conducted
N on-ortho PCBs
3,3 ',4 ,4 '-T C B (77)a
3 ,4,4',5-T C B (81)
3,3',4,4',5-P eC B (126)
3 ,3 ',4 ,4 ',5 ,5 '-H x C B (169)
N o b ioassay conducted
N o b ioassay conducted
R at (F)
N o B ioassay Conducted
M ono-ortho PCBs
2,3,3',4,4'-P eC B (105)
2,3,4,4',5-P eC B (114)
2,3',4,4',5-P eC B (118)
2',3,4,4',5-P eC B (123)
2 ,3 ,3 ',4 ,4 ',5 -H x C B (156)
2 ,3 ,3 ',4 ,4 ',5 '-H xC B (157)
2 ,3 ',4 ,4 ',5 ,5 '-H x C B (167)
2 ,3 ,3 ',4 ,4 ',5 ,5 '-H p C B (189)
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
N o B ioassay Conducted
^International Union of Pure and Applied Chemistry numbers in parentheses.
Abbreviations: O CD D , octachlorodibenzo-p-dioxin; O CD F, octachlorodibenzofuran; TCB,
-tetrachlorobiphenyl.
as initiators followed by administration of a promoting substance (Enzmann
et al. 1998). Also, in several chronic bioassay studies in which dioxin was
administered to female Sprague-Dawley rats for 30 weeks at dosages asso
ciated with an increased incidence of liver tumors in carcinogenicity studies,
no increase in hepatic preneoplastic lesions indicative of initiation was
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found (Lucier et al. 1991; Maronpot et al. 1993). Thus, at present, there is
no direct experimental evidence that dioxin acts as an initiator in rat liver.
A lack of initiating activity would be consistent with an absence of
direct genotoxicity (Williams 1992). Nevertheless, some dose-response
modeling of data that show a promoting effect of dioxin on rat liver
preneoplastic lesions suggested that dioxin also had “ a weak” (Moolgavkar
and Luebeck, 1995) or “ a slight” (Portier et al. 1996) initiating effect. In
contrast, analysis of a two-cell clonal growth model reproduced such data
without presuming an effect on mutation rates (that is, initiation) (Conolly
and Andersen 1997).
Resolution of the question of initiating activity of dioxin awaits experi
mental evidence. Also, the postulated linkage between potential initiating
activity and oxidative DNA damage is not established. In an investigation
of the mode of action of hepatocarcinogenicity of pentachlorophenol, oxi
dative DNA damage was not found to produce liver initiation (Umemura et
al. 1999).
The committee agrees with EPA that TCDD, other dioxins, and DLCs
appear to enhance tumor development in female rat liver via tumor promo
tion. The promoting activity and liver tumor-enhancing activity of dioxin
seem to be mediated through activation of the Ah receptor (aromatic hy
drocarbon receptor [AHR]), which in turn leads to a variety of changes in
gene expression, including notably induction of cytochromes P450 (CYPs)
(Whitlock 1989) and genes related to cell proliferation (Puga et al. 1992)
(see Figure 5-1). Whether those gene changes mediate the reported oxida
tive stress is not known. Nevertheless, both CYP induction and oxidative
stress could be involved in liver cytotoxicity, which was found in studies
that examined this parameter (Maronpot et al. 1993; Viluksela et al. 2000).
Cytotoxicity, in turn, elicits regenerative cell proliferation (Williams and
Iatropoulos 2002), as reported in several dioxin studies (Lucier et al. 1991).
Dioxin-induced changes in gene expression, however, can occur without
enhancement of hepatocelluar proliferation (Fox et al. 1993). In fact, in
creases in cell proliferation have been documented only after 30 weeks of
dioxin administration (Lucier et al. 1991). The enhanced cell proliferation
arising from either altered gene expression or cytotoxicity or both could be
the principal factor leading to promotion of hepatocellular tumors (Busser
and Lutz 1987; Whysner and Williams 1996). The sensitivity of female rat
liver to dioxin, which apparently does not extend to the mouse, clearly
depends on ovarian hormones (Lucier et al. 1991; Wyde et al. 2001). This
sensitivity has been ascribed to induction of estradiol metabolizing enzymes
(Graham et al. 1988) and is hypothesized to lead either to generation of
reactive metabolites of endogenous estrogen or to active oxygen species of
estrogens. Oxidative DNA damage has been implicated in liver tumor pro
motion (Umemura et al. 1999). In contrast to the extensive work on hepa-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
FIGURE 5-1 Possible mechanism for TCDD hepatocarcinogenicity.
tocellular neoplasia, little is known about the pathogenesis of the bile-duct
tumors (Table 5-3).
Mechanistic issues are discussed in greater detail below in the context
of evaluating whether the dose-response relationship is likely to be linear.
In any case, the committee agrees with EPA’s general conclusion that there
is sufficient evidence from epidemiological studies, animal bioassays, and
mode of action studies to support the qualitative conclusion that TCDD,
other dioxins, and DLCs are likely to cause cancer in humans with ad
equate conditions of dose and duration of exposure.
Committee’s Perspective on Whether the Scientific Evidence Supports
Classification of Dioxin As a Known Human Carcinogen
After extensive discussion of EPA’s revised definition of “ carcinogenic
to humans” and “likely to be carcinogenic to humans” provided in EPA’s
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TABLE 5-3 Dioxin Rat Bioassays
Study End Point
Kociba et al. (1978)
(1, 10, 100 ng/kg/day)
NTP (1982a)
(3, 10, 100 ng/kg/wk)
NTP (2005)
(10, 22, 46, 100 ng/kg)
Survival
Decreased at 100 ng/kg
No effect
NOEL for tumors
1 ng/kg
Liver
Adenoma/carcinoma (58% );
bile-duct adenoma, some in
low dose
Cholangiocarcinoma, adenoma (30% ),
cholangioma, hepatocholangioma
Cholangiocarcinoma (46, 100 ng/kg),
adenoma (100 ng/kg)
Lung
Keratinizing squamous cell
carcinoma (100 ng/kb)
Cystic keratinizing epithelioma
(100 ng/kg)
Cystic keratinizing epithelioma
(100 ng/kg)
Oral cavity
Squamous cell carcinoma,
hard palate
Squamous cell carcinoma, gingival
Squamous cell hyperplasia (all)
10.22 ng/kg
Abbreviation: NOEL, no-observed-effect level.
h-A
h-A
VO
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
2005 Guidelines for Carcinogen Risk Assessment (EPA 2005a, see also Ap
pendix B) and consideration of the points above, the committee agrees that
there is strong and convincing evidence that dioxin is likely to be a human
carcinogen. Although the committee does not reject outright the somewhat
higher classification of dioxin as “carcinogenic to humans,” there was not
unanimous agreement that the available scientific information on human
dioxin carcinogenicity met condition (a) of EPA’s cancer guidelines, which
states “there is strong evidence of an association between human exposure
and either cancer or the key precursor events of the agent’s mode of action
but not enough for a causal association” (EPA 2005a, p. 2-54).
The committee was in general agreement that the epidemiological evi
dence, although not “ strong,” was generally consistent with a positive
association between occupational dioxin exposure and mortality from all
cancers, but the magnitude of the effect was modest, and the limited evi
dence for any specific tumor type being significantly associated was of
some concern. This conclusion is in fact quite similar to EPA’s assessment
of the relative strength of the epidemiological evidence (Reassessment, Part
III, p. 2-21). In its discussion, the committee remained uncertain about the
intent of the language in the 2005 Guidelines for Carcinogen Risk Assess
ment stating that condition (a) could be satisfied if there is “ strong evi
dence of an association between human exposure and either cancer or the
key precursor events of the agent’s mode of action but not enough for a
causal association” (EPA 2005a, p. 2-54). The committee agreed that there
is convincing evidence supporting the interaction of dioxin with the human
Ah receptor and that the interaction with the receptor was necessary, but
not sufficient, to cause cancer in animals. However, the committee was not
in complete agreement about whether these conditions met the stated crite
rion of a “key precursor event of the agent’s mode of action” (EPA 2005a,
p. 2-54). For example, it was noted that, even though TCDD binds to the
human Ah receptor, several endogenous and exogeous substances, includ
ing bilirubin, biliverdin, and p-naphthoflavone, also bind to the Ah recep
tor but are not carcinogenic in rodent models (Seidel et al. 2000); hence,
some other key precursor event(s) may need to be identified to meet that
criterion. However, it was also recognized that persistence of the Ah recep
tor activation may be a key determinant for carcinogenicity because ge
netic modification of the AhR gene, causing activation in the absence of
any ligand, results in a tumorigenic response in mice (Andersson et al.
2002).
Furthermore, there is evidence that prolonged stimulation of AHR by
the nonpersistent ligand indole-3-carbinol (or its acid condensation prod
ucts; derived from broccoli and other cruciferous vegetables) can promote a
variety of tumor types after initiation with different genotoxic compounds
(Pence et al. 1986; Bailey et al. 1987; Dashwood et al. 1991; Kim et al.
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1997; Dashwood 1998; Yoshida et al. 2004). The committee recommends
that EPA use the 2005 Guidelines for Carcinogen Risk Assessment (EPA
2005a, see also Appendix B) specifically in its final assessment and carefully
delineate its interpretation of what constitutes “ strong” evidence and a
“key precursor event” under condition (a) of the definition of “carcino
genic to humans.”
The committee noted that classification artificially places an apparent
bright line distinguishing a substance as “carcinogenic to humans” and
“likely to be carcinogenic to humans,” whereas the actual scientific evi
dence lies on a continuum. In the context of a weight-of-evidence con
tinuum, the committee found that the scientific evidence favored the high
end of the “ likely to be a human carcinogen” classification or the lower end
of the “carcinogenic to humans” classification and emphasized that dioxin
remains unique with respect to the International Agency for Research on
Cancer (IARC) Group 1 designation based to a large extent on total cancers
instead of a specific cancer type.
The committee recognizes that the 2003 Reassessment used a different
definition of “carcinogenic to humans” based on EPA’s 2003 draft carcino
gen risk assessment guidelines. The committee found that the argument
provided by EPA in the 2003 Reassessment to support its position that the
epidemiological data met the criterion of “ strong evidence of an associa
tion” between dioxin exposure and cancer risk was unconvincing. How
ever, the committee questioned whether it is worth EPA’s investment of
significant efforts to further qualitatively classify the carcinogenicity of
dioxin. The committee considers that quantitative risk estimates for dioxin
should not depend on which side of the artificial bright line between “ likely
to be a human carcinogen” and “carcinogenic to humans” EPA ultimately
places dioxin. The committee urges EPA to focus on improved quantitative
characterization of risks and to reduce the emphasis on qualitative charac
terization of hazard in this case.
QUANTITATIVE CONSIDERATIONS IN ASSESSING TCDD,
OTHER DIOXINS, AND DLC CARCINOGENICITY
EPA’s Assumption That the Dose-Response Relationship Is Linear
To estimate a cancer slope factor (CSF) for dioxin using either animal
bioassay data or epidemiological data, EPA estimated a point of departure
(POD) dose as the dose yielding an excess cancer risk of 1% and then
extrapolated back to zero incremental dose using a straight line. The dose
(mg/kg-day) corresponding to a 1% excess cancer risk is referred to as the
ED01 (effective dose). Dividing the ED01 dose into 0.01 yields the CSF,
expressed in units of (mg/kg-day)_1. A more conservative, but widely used,
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estimate of the CSF can be calculated by estimating the lower confidence
limit on the ED01 (designated the LED01 [lower confidence bound on the
effective dose]) and then dividing that value into 0.01.
EPA describes its approach as follows (Part III, p. 5-15):
Extrapolation from the POD to lower doses is conducted using a straight
line drawn from the POD to the origin—zero incremental dose, zero in
cremental response—to give a probability of extra risk. The linear default
is selected on the basis of the agent’s mode of action when the linear
model cannot be rejected and there is insufficient evidence to support an
assumption of non-linearity.
Because EPA’s assumption of linearity at doses below the 1% excess
risk level for carcinogenic effects of TCDD, other dioxins, and DLCs is
central to the ultimate determination of regulatory values, it is important to
critically address the available scientific evidence on the most plausible
shape of the dose-response relationship at doses below the POD (LED01).
On the basis of a review of the literature, including the detailed review
prepared by EPA and presented in Part II of EPA’s Dioxin Risk Assessment
and new literature available since the last EPA review, the committee con
cludes that, although it is not possible to scientifically prove the absence of
linearity at low doses, the scientific evidence, based largely on mode of
action, is adequate to favor the use of a nonlinear model that would include
a threshold response over the use of the default linear assumption. The
committee concludes that four major considerations of the scientific evi
dence support the use of a nonlinear model for low-dose extrapolation.
TCDD, Other Dioxins, and DLCs Are Not Directly Genotoxic
As noted earlier, available evidence suggests that TCDD, other dioxins,
and DLCs are not directly genotoxic. There is general consensus in the
scientific community that nongenotoxic carcinogens that act as tumor pro
moters exhibit nonlinear dose-response relationships, and that thresholds
(doses below which the expected response would be zero) are likely to be
present. In addition, even among compounds that covalently react with
DNA, the dose response may be nonlinear (Williams et al. 2005). For
example, the ED01 study (Staffa and Mehlman 1979) used more than
24,000 mice to evaluate the shape of the dose-response relationship over a
5-fold range of administered dose (30 to 150 parts per million) of the
potent carcinogen 2-acetylaminofluorene, which is metabolized to a highly
genotoxic metabolite that forms DNA adducts. The results of the lifetime
feeding study showed a dose-related increase in bladder and liver tumors.
The dose-response relationship for the liver tumors appeared to be linear,
whereas the bladder tumor dose-response was markedly sublinear at the
lower end of the curve.
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Receptor-Mediated Agents Have Sublinear Dose-Response Relationships
Some studies suggest that TCDD (and, presumably, other dioxins and
DLCs) may cause DNA damage indirectly via generation of reactive oxygen
species that may result in oxidative DNA damage and intrachromosomal
recombination, although, as noted above, initiating activity has not been
demonstrated. However, these effects are secondary to a series of down
stream events that are secondary to Ah receptor activation, a phenomenon
that would be likely to cause the dose-response relationship to be sublinear
at low doses. It is recognized that a roughly linear increase in response with
increasing dose will occur at doses above a minimal response level (e.g., 1%
or 5% excess risk), as would be expected for any receptor-mediated re
sponse. These comments are focused on extrapolation of the dose-response
relationship to doses well below those associated with a minimum response
level (POD).
The observation that adverse effects caused by TCDD, other dioxins,
and DLCs depend on AHR activation underlies mechanistic considerations
for these compounds. Part III, p. 3-1 (lines 5 to 14), of the Reassessment
states that
much evidence indicates that TCDD acts via an intracellular protein (the
AhR) which functions as a ligand-dependent transcription factor in part
nership with a second protein (ARNT [AHR nuclear translocator pro
tein]). Therefore, from a mechanistic standpoint, TCDD’s adverse effects
appear likely to reflect alterations in gene expression that occur at an
inappropriate time and/or for an inappropriate long time. Mechanistic
studies also indicate that several other proteins contribute to TCDD’s
gene regulatory effects and that the response to TCDD probably involves
a relatively complex interplay between multiple genetic and environmen
tal factors. If TCDD operates through such a mechanism, as all evidence
indicates, then there are certain constraints on the possible models than
can plausibly account for TCDD’s biological effects, and, therefore, on
the assumptions used during the risk assessment process.
EPA cites further mechanistic studies describing interactions between
the AHR and other critical regulatory proteins and transcription factors
(Rb, SIM, HIF1-a, REL-A, among others) as evidence of the complex inter
play between dioxin and other genetic and environmental factors. Table
3-1 in the Reassessment appropriately describes early molecular events.
There is widespread agreement in the scientific community that all or
nearly all the adverse effects of TCDD, other dioxins, and DLCs depend on
a receptor-mediated mechanism. Both IARC and EPA (see above) conclude
that these compounds act through a mechanism involving the AHR. As
noted in the Reassessment (Part III, p. 2-19, lines 28 to 33):
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Despite this lack of a defined mechanism at the molecular level, there is a
consensus that 2,3,7,8-TCDD and related compounds are receptor-medi
ated carcinogens in that (1) interaction with the AhR is a necessary early
event, (2) 2,3,7,8-TCDD modifies a number of receptor and hormone
systems involved in cell growth and differentiation, such as the EGFR
[epidermal growth factor receptor] and estrogen receptor, (3) sex hor
mones exert a profound influence on the carcinogenic action of 2,3,7,8TCDD.
Mechanistic considerations for DLCs and dioxins other than TCDD
are less well established, although it is widely held that most of the toxic
and carcinogenic effects of other dioxins and DLCs are mediated via the
same receptor signaling pathways as those for TCDD. The Reassessment
cites “ comparative binding studies and other data” (Part III, p. 2-3, lines 25
and 26) to suggest that DLCs and dioxins other than TCDD exhibit TCDDlike responses in proportion to their receptor binding affinity (generally
reflected in their toxic equivalency factors [TEFs]). Although this associa
tion may hold for most toxic and biochemical responses, there are few, if
any, biochemical or mechanistic studies describing interactions when DLCs
and dioxins other than TCDD are the ligands/inducers. It is not clear if
those interactions play a role in the adverse health effects of TCDD, other
dioxins, and DLCs, nor have such interactions been characterized.
There is a large body of scientific data on receptor-mediated responses.
However, while the relationship of receptor binding and effects on tumor
development in rodents remains uncertain from a mechanistic point of view
(Whysner and Williams 1996), the recent National Toxicology Program
(NTP) bioassay results using the TEF/TEQ (toxic equivalent quotient) ap
proach (which is dictated by AHR binding) strongly supports the role of
AHR in hepatocarcinogenicity of DLCs. Receptor binding appears neces
sary, but insufficient, because many tissues with receptors are not sites of
TCDD-induced (or, by inference, induced by other TCDD, other dioxins,
and DLCs) preneoplastic changes or tumors.
A fundamental concept in pharmacology is that receptor-mediated re
sponses show sigmoidicity in the shape of the log dose-response relation
ship, although ligand-receptor interactions and subsequent “ down stream”
events that ultimately produce drug efficacy or toxicity are complex (Ross
and Kenalkin 2001). Response is a function of the number of occupied and
activated receptors, which typically exhibit steep dose-response relation
ships. For example, Kohn and Melnick (2002) modeled the shape of the
dose-response relationship for receptor-mediated responses, using the es
trogen receptor and various xenoestrogens as a model receptor and ligands,
respectively. The model included a variety of assumptions with regard to
receptor number, ligand binding affinity, and partial agonist activities, yet
in every instance clear sublinear responses were observed at low doses. In
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all instances modeled, a predicted response indistinguishable from the back
ground response was seen at doses less than one order of magnitude lower
than the dose providing the lowest detectable response (conceptually simi
lar to a POD). The model parameters were based on ligands with relatively
short half-lives and reversible binding to the receptor and thus may not be
directly applicable to TCDD, other dioxins, and DLC binding to AHR.
Carcinogenicity of DLCs is not solely and quantitatively related to
receptor binding. There are numerous synthetic and naturally occurring
AHR ligands, to which humans are exposed through diet and the environ
ment, that bind to and activate the receptor (and induce a transcriptional
response as measured by cytochrome P4501A protein [CYP1A] mRNA and
enzyme activity) and yet do not seem to act as tumor promoters or directly
produce AHR-dependent toxic responses commonly seen with TCDD, other
dioxins, and DLCs. Thus, although binding to and activation of AHR
appears to be required for tumor promotion, it is not sufficient. On the
other hand, others (Carney et al. 2004) have shown that morpholinos to
AHR block cardiovascular toxicity in zebrafish, but morpholinos to CYP1A
do not. This conclusion strongly suggests that additional downstream events
are critical to the promotional effects of these chemicals. Because multiple
additional steps are necessary, each probably with homeostatic mechanisms
functional at low doses but perhaps overwhelmed at high doses, sublinearity
with a response approaching zero at low doses would be expected.
EPA determined in previous evaluations of receptor-mediated carcino
gens that a nonlinear, low-dose model, that may accommodate a threshold
is appropriate. For example, numerous pesticides found to cause thyroid
cancer secondary to modulation of thyroid hormone levels have been evalu
ated as threshold-type carcinogens (EPA 1998). In the recent NTP studies
with dioxin, the observed thyroid tumors are undoubtedly due to perturba
tion of thyroid homeostasis (NTP 2005). Similarly, the induction of liver
tumors from peroxisome proliferators was also deemed to occur via a
threshold-type response but was further deemed largely irrelevant to hu
mans because of species differences in peroxisome proliferator activated
receptor (PPAR) function (EPA 2003d).
The final cancer guidelines (EPA 2005a, see also Appendix B) provide
the following guidance on choosing between linear and nonlinear risk ex
trapolation approaches: “A nonlinear approach should be selected when
there are sufficient data to ascertain the mode of action and conclude that it
is not linear at low doses and the agent does not demonstrate mutagenic or
other activity consistent with linearity at low doses” (p. 3-22). This is an
important decision, as it will influence the methodology adopted in subse
quent risk assessments. The final EPA cancer guidelines also make the
following statement about risk assessment for carcinogens with a nonlinear
mode of action (EPA 2005a, p. 3-20).
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
TABLE 5-4 Hepatic Toxicity in TCDD Rat Bioassays
Kociba et al. (1978)
NTP (1982a)
NTP (2005)
0 ng/kg, severity = 0.6 (57% )
1 ng/kg, severity = 1.2 (88% )
0 ng/kg, 0 incidence
0 ng/kg
10 ng/kg, severity = 2.1 (95% )
3 ng/kg, severity = 1.0 (4% )
10 ng/kg, severity = 1.3 (15% )
100 ng/kg, severity = 3.6
(100% )
100 ng/kg, severity = 3.5
(100% )
10 ng/kg severity +
22 ng/kg severity
2 + 4 6 ng/kg severity
3+
100 ng/kg severity 4+
At this time, safety assessment is the default approach for tumors that
arise through a nonlinear mode of action; however, EPA continues to
explore methods for quantifying dose-response relationships over a range
of environmental exposure levels for tumors that arise through a nonlin
ear mode of action. (EPA 2002)
Evidence That Liver Tumors Are Secondary to Hepatotoxicity
In the Reassessment, EPA used the female rat liver tumor data from the
Kociba et al. (1978) study to develop a dose-response relationship. In that
study, the liver was the main site of carcinogenic activity (see Table 5-3).
Dioxin is retained preferentially in the liver in rats (Fries and Marrow
1975; Kociba et al. 1978), in addition to adipose tissue, which may underlie
the liver susceptibility. In the rat liver, hepatic toxicity was accompanied by
increases in liver tumors (Table 5-4), and numerous studies have shown
that hepatotoxicity results in increased cell proliferation (Williams and
Iatropoulos 2002). In the most recent dioxin bioassay (NTP 2004), toxic
hepatopathy was found at 31 weeks at 100 mg/kg and at 53 weeks at 46
mg/kg and 100 mg/kg, but not at low dosages. Hepatocellular labeling
indices were consistently elevated at these dosages at 31 and 53 weeks. In
other studies, hepatotoxicity was less pronounced in male rats, for which
no increase in tumors was seen. The hepatocarcinogenicity in female rats is
related to estrogens and may be due to elevation of estrogen catechol levels
resulting from AHR-dependent induction of cytochromes P450 in the CYP1
family responsible for generating catechols from estradiol. Accordingly,
toxicity and cell proliferation may have been key events for hepatocarcinogenicity in these studies, as has been delineated for a variety of other rodent
hepatocarcinogens (Williams 1997).
The cancer guidelines (EPA 2005 a, see also Appendix B) caution against
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using tumor data for quantitative, low-dose extrapolation when clear evi
dence of cytotoxicity is present:
Studies that show tumor effects only at excessive doses may be compro
mised and may or may not carry weight, depending on the interpretation
in the context of other study results and other lines of evidence. Results of
such studies, however, are generally not considered suitable for dose-re
sponse extrapolation if it is determined that the mode(s) of action under
lying the tumorigenic responses at high doses is not operative at lower
doses. . . . Studies that show tumors at lower doses, even though the high
dose is excessive and may be discounted, should be evaluated on their
own merits. (EPA 2005a, p. 2-18)
Earlier in the document, EPA states,
In addition, overt toxicity or altered toxicokinetics due to excessively high
doses may result in tumor effects that are secondary to the toxicity rather
than directly attributable to the agent. (EPA 2005a, p. 2-17)
Thus, based on these criteria, evidence of substantial hepatoxicity in
tumor-bearing animals would raise questions about the use of hepatic tu
mors in female rats for quantitative, low-dose extrapolation.
Although there is evidence that the liver tumors observed may be due to
hepatotoxicity and have a sublinear dose-response relationship, the com
mittee notes that two other types of epithelial tumors (keratinizing epithe
lioma of the lung and squamous cell tumors of the oral mucosal epithelium)
were increased in a dose-dependent manner with no apparent indication of
cytotoxicity in these tissues. However, the shape of the dose-response rela
tionship for these tumors suggests that they may be nonlinear, as described
below.
Bioassay Evidence of Nonlinearity
The recent NTP bioassay data (NTP 2004; Walker et al. 2005) show a
consistent sigmoidicity to the tumor dose response. Walker et al. (2005)
reported a Hill coefficient3 of 2.81 (standard error [SE] = 0.68) for
cholangiocarcinoma, 3.74 (SE = 1.5) for hepatocellular adenoma, 23.4 (SE
insufficiently stable to report) for keratinizing epithelioma of the lung, and
2.14 (SE insufficiently stable to report) for squamous cell tumors of the oral
mucosal epithelium. The central estimates for these coefficients all exceed
3The Hill function is defined as f(dose) = dosen/(kn + dosen) and can be used as a model
component for dose effects. The coefficient n > 1 signifies a departure from linearity at lower
doses.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
2, hence suggesting nonlinearity, although the 95% CIs do not exclude a
Hill coefficient of 1, which corresponds approximately to a linear dose
response at low doses. Nonetheless, although the data alone do not rule out
a linear tumor response at doses below a 5% response level (because of
small sample size and limited statistical power), the observed data are more
consistent with a sublinear response that approaches zero at low doses
rather than a linear dose response. On the other hand, the tumor data
would also probably fit a linear, low-dose model because of the small
number of data points in the low-dose region.
EPA Evaluation of Bioassay Data to Estimate the CSF
For the purpose of estimating a CSF for dioxin based on animal data,
EPA considered the assays conducted by Kociba et al. (1978) and NTP
(1982a). In each case, EPA restricted attention to those tumor types for
which incidence increased with dioxin exposure (five types in the Kociba et
al. study and eight types in the NTP study). Based on an analysis by Portier
et al. (1984) using a simple multistage model (order up to 3), the ED01 body
burdens for these 13 dose-response relationships ranged from 14 to 1,190
ng/kg. The corresponding LED01 values ranged from 10 to 224 ng/kg.
EPA also considered two alternative ED01 estimates developed using
the Kociba et al. (1978) female rat liver tumor data. First, EPA described an
ED01 estimate calculated from a model developed by Portier and Kohn
(1996). That model combined a pharmacokinetic model characterizing gene
expression induced by dioxin with a two-stage carcinogenesis model to
analyze the female rat liver tumors from the Kociba et al. study. Using that
model, EPA calculated ED01 = 2.7 ng/kg. Second, EPA reported an estimate
for ED01 equal to 31.9 ng/kg (LED01 = 22.2 ng/kg). EPA developed this
estimate using its benchmark dose software and a reevaluation of the Kociba
et al. (1978) pathology results by Goodman and Sauer (1992). The revised
estimate also reflected other changes in the procedure for fitting a function
to the data.
In 2003, when EPA’s Reassessment was issued, the most recent NTP
bioassay results (NTP 2004) were not yet published. Because this study
represents an extensive data set developed using state-of-the-art methodol
ogy, EPA should integrate this information into its analysis.
In addition, EPA should specify criteria used to identify those data sets
to be included in its analysis. The EPA Reassessment does not explain why
EPA chose to rely on a single site (liver) from one sex (female) in one species
(rat), as measured in a single study (Kociba et al. 1978). Consideration of
other data sets that were available to EPA would have yielded a substan
tially wider range of potency estimates. Whereas the LED01 for liver tumors
in female rats from the Kociba et al. study is 22.2 ng/kg, the ED01 values
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calculated using all the animal bioassay data sets considered by EPA (data
sets from the NTP and Kociba et al. studies that suggested tumor incidence
increases with dose) range as high as 1,190 ng/kg. In addition, use of the
mechanistic model developed by Portier and Kohn (1996) to analyze the
Kociba et al. female rat liver tumor data yields a substantially lower ED01
value (2.7 ng/kg). The range of values would be even broader if EPA had also
estimated upper ED01 (UED01 [upper confidence bound on the effective dose])
values. Like the LED01 values, these values are indicative of the range of
estimates that are consistent with the data and hence are indicative of inher
ent uncertainty. Calculations of a slope factor that considers the effects of
dioxin on ALL tumor sites, as was done for the human epidemiological data,
would probably further broaden the range of plausible ED01 values. Because
dioxin is presumed to promote tumor growth at a wide range of sites, EPA
should explain why it chose not to evaluate the dose-response relationship for
“ all tumors combined” in the animal studies if it considers this approach to
be appropriate for use in human epidemiological studies.
The committee notes that extrapolation of results across species is
highly uncertain, even when dose is scaled to account for body burden.
Although data from animal and human cells and tissues suggest a qualita
tive similarity across species in the response to DLCs (Reassessment, Part
III, p. 2-3, lines 28 and 29, and p. 3-10, lines 30 to 33), they do not support
the hypothesis that the responses across species are quantitatively similar.
For example, there is no explanation for the observation that the LD50
(50% lethal dose) for dioxin in guinea pigs and hamsters differs by more
than a factor of 8,000 (Part II, p. 3-1) even though their respective receptors
do not differ substantially in terms of dioxin binding and other responses
(e.g., CYP1A1 induction differs by only a factor of 4). Similarly, whereas
the LD50 for dioxin in two strains of rats differs by a factor of 300 to 1,000
(Part II, pp. 3-1 to 3-3), AHR ligand binding in these two strains has similar
affinities, and CYP1A1 inducibility does not differ. These observations
complicate interspecies comparisons. Recent studies comparing the response
of human hepatocytes to dioxin with that of rat and mouse hepatocytes
further illustrate that quantitative extrapolation of rodent data to humans
is highly uncertain (Silkworth et al. 2005).
TCDD, other dioxins, and DLCs act as potent inducers of CYP, a
property that can affect both the hepatic sequestration of these compounds
and their half-lives. Hepatic sequestration of dioxin may influence the quan
titative extrapolation of the rodent liver tumor results because the bodyburden distribution pattern in highly dosed rats would differ from the
corresponding distribution in humans subject to background levels of expo
sure. EPA should consider the possible quantitative influence of dose-de
pendent toxicokinetics on the interpretation of animal toxicological data.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
EPA’s Characterization of Uncertainty for CSF Estimates
As part of their quantification of risk, it is important for risk assessors
to provide a full characterization of the uncertainty inherent in their esti
mates. Although risk managers may choose to focus on conservative esti
mates of risk, the risk assessment must be kept distinct (NRC, 1983) and
should describe the lower and upper ranges of plausibility for risk esti
mates. A complete characterization of a risk’s uncertainty facilitates (1)
comparison of that risk estimate with other risk estimates that may have the
same point value but a different degree of uncertainty, (2) comparison of
risks with the costs and countervailing risks associated with interventions
to address the primary risk, and (3) evaluation of research needs. A number
of reports have addressed the need for a comprehensive treatment of uncer
tainty in risk assessment, including a National Research Council (NRC
1994) report.
Part III, Chapter 5, of the Reassessment describes EPA’s development
of a CSF for TCDD, other dioxins, and DLCs. EPA identifies 1 x 10_3 pg
TEQ/kg of body weight per day (pg/kg-day)_1 “ as an estimator of upper
bound cancer risk for both background intakes and intakes above back
ground” (Part III, pp. 5-28 to 5-29). While EPA qualitatively notes many of
the factors contributing to this estimate’s uncertainty, the Reassessment
does not adequately discuss how these factors contribute quantitatively to
the underlying uncertainty. By omitting the quantitative implications of
these factors, the Reassessment understates the uncertainty inherent in these
estimates and overstates the consistency of the data and risk estimates
across all studies.
EPA should have addressed quantitatively the following sources of
uncertainty:
• Basis for risk quantification: (1) bioassay data, (2) occupational
cohort data.
• Epidemiology data to use: (1) risk estimate developed with data
aggregated from all suitable studies, (2) risk estimate or estimates devel
oped using each study individually.
• Factors affecting extrapolation from occupational to general popu
lation cohorts, including differences in baseline health status, age distribu
tion, the healthy worker survivor effect, and background exposures.
• Bioassay data to use: (1) risk estimate developed with the single data
set implying the greatest risk (that is, single study, tumor site, gender), (2)
risk estimate developed with multiple data sets satisfying an a priori set of
selection criteria.
• Dose-response model: (1) linear dose response, (2) nonlinear dose.
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• Dose metric: (1) average daily intake, (2) area under the blood con
centration-time curve, (3) lifetime average body burden, (4) peak body
burden, (5) other.
• Dose metric—biological measure: (1) free dioxin, (2) bound dioxin.
• POD: (1) ED 1 0 , (2) ED05, _(3) ED0i.
• Value from ED distribution to use: (1) ED, (2) lower confidence
bound value for the ED (LED), (3) upper confidence bound for the ED
(UED).
Where alternative assumptions or methodologies could not be ruled
out as implausible or unreasonable, EPA could have estimated the corre
sponding risks and reported the impact of these alternatives on the risk
assessment results. The potential impacts of four sources of uncertainty are
discussed below.
• The full range of plausible parameter values for the dose-response
functions used to characterize the dose-response relationship for the three
occupational cohort studies selected by EPA (Ott and Zober 1996; Becher
et al. 1998; Steenland et al. 2001).
• Use of other points of departure, not just the ED01 (or LED01), to
develop a CSF.
• Alternative dose-response functional forms as well as goodness of fit
of all models, especially at low doses.
• Uncertainty introduced by estimation of historical occupational ex
posures.
If these factors are considered, the range of plausible CSF values be
comes much larger, with more extreme upper and lower bound estimates,
as shown below.
EPA’s development of a CSF value emphasizes the analysis of three
occupational cohort studies (Ott and Zober 1996; Becher et al. 1998;
Steenland et al. 2001). In all cases, the studies estimated SMRs or rate
ratios (RRs) as a function of cumulative dioxin lipid burden (CLB, nano
gram of dioxin per kilogram of lipid weight x years) (see Reassessment,
Part III, Table 5-2). Using these dose-response relationships, EPA sum
marizes its ED01 and CSF calculations (Part III, Table 5-4). The ED01
values are reported as lifetime average body burdens for dioxin (LABB,
ng/kg). Although the relationship EPA used to convert from CLB to
LABB was not transparent in the Reassessment, the committee assumes
it can be described as CLB = 4 x 75 x LABB. Here, the factor of 4
accounts for conversion from nanogram per kilogram of lipid to nano
gram per kilogram of body weight (see Reassessment note (a) of Table 5-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
4 in Part III for this assumption), and 75 corresponds to the “ average”
lifetime in years.
We note that EPA identified the dose corresponding to 1% excess risk
(the ED01) from the relationship.
Risk (ED01) - Risk (Background)
^
Risk (Infinity) - Risk (Background)
EPA estimated the risk function in the above equation from the occupa
tional cohort studies by converting the hazard function to a probability of
death by age 75 years. This risk estimate satisfies the requirement that risk
(infinity) = 1—that is, as dose increases, the risk approaches 100%. Critical
findings are reproduced in Table 5-5.
The committee also notes that EPA’s analyses of the Hamburg and
BASF cohorts considered all cancer deaths with no latency, but the analysis
of the National Institute of Occupational Safety and Health (NIOSH) co
hort considered a 15-year lag. The committee is aware of the problem with
cancer mortality studies in which the subjects are without cancer at baseline
(first exposure) so that the cancer mortality at the start of follow-up (in the
few years after first exposure) will be artificially low. There is thus good
reason to consider deaths only after some fixed time, and in dose-response
calculations, one has to estimate cumulative dose appropriate to the date of
occurrence of the cancer. These considerations were not part of the basis
for determining the latency used in the NIOSH analysis, which was based
on the assumption that effects should not be seen for many years after first
exposure and the dose calculations ignored all doses for the immediately
preceding 15 years. The committee is unconvinced of the validity of such
assumptions in the context of dioxin as a promoter and furthermore sees no
justification for considering the NIOSH results any differently than the
other two cohorts.
Full Range of Plausible Parameter Values
In Part III of the Reassessment, EPA makes use of only the ED01 and
LED01 for the purpose of estimating a CSF. Of course, a more complete
range of plausible CSF values can be developed by considering parameter
estimates corresponding to dose-response relationships that are less than
the central estimate relationship (used to identify the ED01). EPA’s recently
released cancer guidelines (EPA 2005a) recommends use of both lower- and
upper-bound values. In section 3.2.4 of the document, which is entitled
Point of Departure (POD), EPA states, “ risk assessors should calculate, to
the extent practicable, and present the central estimate and the correspond-
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TABLE 5-5 EPA Inputs to CSF Estimates Using Epidemiological Dataa
Central
P
Estimate
Value
Study
Function RR(x)b
for bb
for bb ED01C LED01C
Becher et al.
Power: (1 + kx)b
b = 0.326 for
0.026
6
NAd
1998
k = 1.7 x 10-4
Additive: 1 + bx
b = 1.6 x 10-5
0.031
18.2
NAd
NAd
Multiplicative: ebx b = 8.69 x 10-6 0.043
32.2
Steenland
et al. 2001
Power:
(x/background)b
Piecewise linear:
ebx
b = 0.097
0.003
1.38e
0.71e
b = 1.5 x 10-5
NAf
18.6
11.5
Ott and Zober
1996
ebx
b = 5.03 x 10-6
0.05
50.9
25
aEDoi values here represent estimates only for males (as is the case in EPA’s Part III, Table 5
4). Female ED0 1 values are modestly larger because the background cancer rate for females is
less than it is for males. For example, the Ott and Zober (1996) ED0 1 value for females is 62
ng/kg and the corresponding LED0 1 is 30.5 ng/kg.
bSee Part III, Table 5-2. Note that x is exposure expressed in terms of cumulative lipid burden
(CLB), ng of dioxin/kg of fat x years.
¿See Part III, Table 5-4. The ED0 1 values are expressed in terms of lifetime average body
burden (LABB), ng of dioxin/kg of body weight.
^Not available. EPA did not estimate LED0 1 values (or the corresponding upper bound for
ED0 1 ), although these values can be calculated, as described below.
¿EPA reported these values in Part III, Table 5-3. Note that EPA omitted further consideration
of the power function for this dataset, stating that “this formula predicts unreasonably high
attributable risks at background dioxin levels in the community due to the steep slope of the
power curve formula at very low levels” (Part III, p. 5-37).
/Not available. Steenland et al. (2001) did not report the P value for this parameter, although
EPA reported a value for LED0 1 .
ing upper and lower statistical bounds (such as confidence limits) to inform
decision makers” (EPA 2005a, p. 3-17).
To illustrate the quantitative impact on the range of uncertainty from this
one assumption, the committee considered the upper ED01 (UED01) values that
correspond to the lower 95% confidence interval on the dose-response rela
tionship. EPA provides the UED01 values for the Steenland et al. (2001) and Ott
and Zober (1996) studies (see Table 5-3 in Part III of the Reassessment). As
explained below, the committee has calculated the UED01 values for the Becher
et al. (1998) study. Together with the ED01 and LED01 values, the UED01
values help to describe the range of plausible ED01 values and hence the uncer
tainty that attends the CSF estimates due to finite sampling.
To estimate the range of plausible ED01 values, the committee assumes
that the set of plausible values for the dose-response relationship parameter
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
(b—see column 3 of Table 5-5 in this report) is normally distributed with a
mean equal to the parameter’s central estimate (b—see column 3 of Table
5-5) and a standard deviation equal to the estimate’s standard error (bSE).4
When possible, the committee estimated the value of bSE using the P value
for the dose-response relationship parameter, as reported in the Reassess
ment, Part III, Tables 5-2 and 5-3 (shown in column 4 of Table 5-5 of this
report). In particular, bm, bSE, and P satisfy the relationship
b m - N - 1 (l - 2 ) X b SE =
0,
where N -1 is the inverse cumulative normal function. For example, if bm =
0.1 and P = 0.05, then bSE = 0.051. Designating bUED = bm - 1.96bSE, the
value of b yielding the UED01, and bLED = bm + 1.96bSE, the value of b
yielding the LED01, the UED01 satisfies the relationship RR(bUED, UED01) =
RR(bLED, LE D 01), where the function RR is the dose-response relationship
(rate ratio) taking two arguments (the parameter b and a dose).
When the P value is not available but both LED01 and ED01 are speci
fied, the committee assumed that bLED - bm = bm - bUED. If necessary, the
value of bLED was estimated from the relationship RR(bLED, LE D 01) =
RR(bm, ED ) and UED01 from the relationship RR(bUED, UED01) =
RR(bLED, LE D 01). In the case of the Becher et al. (1998) study, inserting bm
into any of the RR formulas along with the ED01 value for that doseresponse function yields an RR of approximately 1.09. It was assumed that
the UED01 is the dose that yields an RR of 1.09 when inserted into the doseresponse function along with bUED. For example, the Becher et al. power
function yields an RR of 1.09 if a LABB of 6 ng/kg (CLB = 1,800 ng/kgyear) is used along with the exponent parameter bm= 0.326. In particular,
(1 + 0.00017 x 1,800)0326 = 1.09. The value of bUED is 0.039 and (1 +
0.00017 x 45,300)°.°39 = 1.09. That is, CLB = 45,300 ng/kg-year produces
the same RR when used with b = bUED. Dividing CLB by 4 x 75 = 300 yields
a LABB of 151 ng/kg. The committee assumed that because the LABB of
151 ng/kg also yields an RR of 1.09, this dose is the UED01.
Table 5-6 summarizes the ED01, LED01, and UED01 values for the doseresponse relationships listed in Table 5-2 of Part III of the Reassessment.
The results in Table 5-6 indicate that the set of plausible ED01 values spans
at least one or two orders of magnitude for the Becher et al. (1998) study
and the Ott and Zober (1996) study.
0 1
4When estimated using a large number of observations, statistical parameters typically
have normal error distributions. Of course, it is possible that the error distributions for the
bmparameters are not normal and hence not symmetric.
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TABLE 5-6 E P 01, LEP01, and UED01 Values
LABB (ng/kg)
Study
Function RR =
ED01 LED01
Becher et al. 1998
Power: (1 + kx)
6
3
Additive: 1 + bx
18.2
9.5
Multiplicative: ebx
32.2
16
UED01
150
200
1,000
Steenland et al. 2001
Power: (x/background)
Piecewise linear: ebx
1.38
18.6
0.71
11.5
8.95
49
Ott and Zober 1996
ebx
50.9
25
Infinite
NOTES: Bolded values represent the committee’s estimates of UED0 1 for the Becher
et al. (1998) study. These values were estimated by assuming a normal error distribu
tion for b (see column 3 in Table 5-5 of this report). All other values were as reported
by EPA in Table 5-3 of Part III of the Reassessment.
Consideration of Alternative Points of Departure
EPA explains that while a 10% level is generally used as a POD (that is,
an ED10 is generally used to estimate the CSF), “where more sensitive data
are available, a lower point for linear extrapolation can be used to improve
the assessment (e.g., 1% response for dioxin, ED01)” (Part III, p. 5-15).
EPA’s cancer guidelines (EPA 2005 a, see also Appendix B) state, “ Conven
tional cancer bioassays, with approximately 50 animals per group, gener
ally can support modeling down to an increased incidence of 1-10%; epide
miologic studies, with larger sample sizes, below 1% ” (p. 3-17).
However, these generalities do not imply that extrapolation down to
low levels is justified in all circumstances. EPA’s carcinogen risk assessment
guidelines document explains, “Various models commonly used for car
cinogens yield similar estimates of the POD at response levels as low as
1% . . . . Consequently, response levels at or below 10% can often be used
as the POD” (EPA 2005a, p. 3-17). The key point here is that a lower
response level is justified only if the estimated dose corresponding to this
response is insensitive to the functional form (provided the other functional
forms fit the data to a comparable degree). The dose-response functions for
the epidemiological data identified by EPA suggest this criterion is not
satisfied. For example, as detailed in Table 5-3 of Part III of the Reassess
ment, the ED01 for males in the Steenland et al. (2001) study is 1.38 ng/kg
of body burden if the power function is used, more than an order of
magnitude less than the ED01 of 18.6 ng/kg calculated using the piecewise
linear function. In the Becher et al. (1998) study, the ED01 spans a factor of
five, depending on which dose-response function is used.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Although EPA states that a 1% response above background (corre
sponding to RR « 1.09) is within the range of observed response for the
three occupational cohort studies considered, it is clearly at the low end of
the observed range. For example, among the five exposure groups defined
in the Becher et al. (1998) study (excluding the comparison group, for
which SMR is fixed at 100), the lowest RR is 1.12. Of the six exposure
groups in the Steenland et al. (2001) study (excluding the comparison
group), one has an RR value below 1.09 (RR = 1.02 for the second lowest
exposure group). RR values for the other five groups were 1.26 or greater.
For the Ott and Zober (1996) study, RR values for the three comparison
groups were 1.2, 1.4, and 2.0.
The use of alternative points of departure for the power dose-response
relationships would greatly increase the range of plausible CSF values.
Table 8-2 (Reassessment, Part II) demonstrates this point. For the Steenland
et al. power function, the 95% confidence interval for ED01 spans approxi
mately one order of magnitude. As a result, the CSF calculated with this set
of ED01 estimates likewise spans approximately an order of magnitude. In
contrast, the 95% confidence interval for ED05 spans approximately three
orders of magnitude. Similarly, the ED01 confidence interval derived from
the Becher et al. power function spans a factor of approximately 50. The
corresponding range for ED05 spans nearly four orders of magnitude.
Thus, it is evident that the choice of POD can have a substantial impact
on the uncertainty of the final risk estimate, especially if both upper and
lower confidence limits are provided. The importance of this assumption is
not readily evident in the Reassessment. The transparency of the uncer
tainty of CSF calculations, and thus risk estimates, would be substantially
improved if the document presented CSF ranges and risk estimates calcu
lated from both ED01 and ED05 values to illustrate the importance of this
assumption.
Consideration of Alternative Dose-Response Functional Forms
Because there are so many functional forms from which to choose for
the purpose of modeling dose response, EPA should establish criteria for
selecting acceptable solutions. For example, there are formal goodness-offit tests that can help to identify the best candidates. Note that a higher
statistical significance for a positive dose response does not necessarily
imply that, using standard statistical criteria, the model adequately fits the
data. Evaluating the goodness of fit for the occupational cohort analyses
was complicated by EPA’s lack of ready access to the original data. Despite
these complications, it is important that EPA provide a cogent set of criteria
for determining which functional forms were used. This section identifies
four instances in which EPA eliminated from consideration alternative dose-
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137
response functional forms without providing adequate justification. EPA
should describe the range of ED01 and ED05 values implied by dose-re
sponse functions that are statistically consistent with the occupational co
hort data and the inclusion criteria established for this assessment.
First, EPA eliminated from consideration the power function doseresponse relationship calculated from the Steenland et al. (2001) study,
explaining only that this relationship “predicts an unrealistic risk for the
background exposure” (Reassessment, Part II, p. 8-67) and that it “leads to
unreasonably high risks at low exposure levels, based on calculations of the
attributable risk that this model would predict from background dioxin
levels in the general population” (Reassessment, Part III, p. 5-13). EPA
provided no criteria by which it judged the reasonableness of the Steenland
et al. power function, nor does EPA provide any further explanation on this
point. The Reassessment should provide further scientific rationale for ex
cluding the Steenland et al. power function, or it should be considered as
valid as any of the other dose-response relationships.
Second, EPA considered only dose-response relationships based on the
assumption of no background incremental risk (that is, SMR = 100 at
background exposure levels). This assumption is inconsistent with the find
ings of two analyses identified by EPA (Starr 2001, 2003; Crump et al.
2003) (Part III, p. 5-14) that rejected the assumption on statistical grounds
that SMR = 100 at baseline exposure levels. If relaxing this assumption
yields an estimate of SMR > 100 at background exposures, the resulting
dose-response relationship would tend to be shallower, yielding smaller
CSF values. For example, EPA (Part III, p. 5-15) noted that in a pooled
analysis of Ott and Zober (1996), Flesch-Janys et al. (1998), and Steenland
et al. (2001), fixing SMR = 100 at background exposure levels yielded ED01
= 51 ng/kg-day, whereas dropping this assumption resulted in ED01 = 91
ng/kg. EPA should provide an explanation for assuming SMR = 100 at
background exposure levels. Short of doing so, EPA should consider the
impact of relaxing this assumption on the estimated value of the ED01.
Third, for the piecewise linear dose-response function developed for the
Steenland et al. data set, EPA considered only one cut point (40,000 ng/kg
x years) (the cut point, or changing point, is the dose at which the slope of
the piecewise linear dose-response relationship changes). Although this is
the best-fit cut-point estimate and the only relationship of this form re
ported by the authors, other cut-point values are plausible. (Other cut
points would yield dose-response relationships that could not be statisti
cally rejected.)
Finally, EPA considered only a subset of the plausible dose-response
relationships that could be fit to the data in the Becher et al. (1998) study.
Becher et al. considered a family of dose-response relationships of the form
RR = (kx + 1)^, where the value of k is chosen arbitrarily. The best-fit value
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
of 13 depends on the value of k selected. The relative plausibility of different
values of k can be determined by comparing likelihood function values.
Finally, holding ft = 1 yields a linear function where the value of k is
uncertain. Becher et al. reported that k = 0.00017 maximizes the likelihood
function and that, for this value of k, the corresponding value of ft = 0.326.
Holding p = 1 yields k = 0.000016. However, Figure 1 of Becher et al.
indicates that the likelihood function is relatively insensitive to the value of
k selected. Hence, other dose-response relationships are plausible. Estimat
ing the ED01 values corresponding to these alternative dose-response rela
tionships would require further primary analysis of the data.
Uncertainty Associated with Estimation of Historical Exposures
The assumed half-life for dioxin in humans plays a major role in the back
extrapolation of dioxin lipid concentrations to the estimation of peak body
burdens in occupational cohorts. The Reassessment states, “Using published
first-order back-calculation procedures, the relatively small difference (<10
100-fold) in body burden between exposed and controls in the dioxin epidemi
ology studies makes exposure characterization in the studies a particularly
serious issue” (Part III, p. 5-7). The high exposures in the occupational cohorts
suggest a high likelihood of enzyme induction during the period of occupa
tional exposure that may have led to a reduction of the half-life to less than the
assumed value of 7.1 years. Aylward et al. (2005) discussed the issue of half
lives and the impact of this parameter on risk estimates.
EPA’s Reassessment compared the impact of using either a half-life of 4
years or the default of 7.1 years on the back-extrapolation estimate. EPA
reported that using a 4-year half-life increases the peak body burden and
the area under the curve (AUC) by 4.6-fold and 3.8-fold, respectively. This
difference would have increased the estimated ED01 values by the same
amount and hence decreased the CSF estimates, resulting in a lower risk
estimate. Given the potential importance of this issue, the committee finds
the following statement by EPA surprising: “This bounding exercise sug
gests that impacts on back-calculated peak and AUC values may become
significant if the models predict prolonged periods with half-lives of less
than 4 years” (Part III, p. 5-8).
Because the impact of the half-life used for back-extrapolation depends
on the back-extrapolation duration required in any particular study, EPA
should have estimated the impact of using the 4-year alternative value for
each of the main epidemiological studies separately. EPA should also con
sider the issues raised by Aylward et al. (2005). Overall, the Reassessment
does not provide sufficient quantification of the impacts of these choices,
and the committee believes these decisions influence the estimated doseresponse relationships.
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Overall Uncertainty
Table 5-4 (Reassessment, Part III) summarizes EPA’s all-site cancer
ED01 values. For the three occupational cohort studies, these values span
less than an order of magnitude (6 to 50.9 ng/kg). The corresponding CSF
values range from 5.7 x 10_4 to 5.1 x 10_3 (pg/kg-day)_1. On the basis of
this range, EPA concludes that “ A slope factor estimate of approximately 1
x 10_3 per pg of dioxin per kg of body weight per day represents EPA’s
most current upper bound slope factor for estimating human cancer risk
based on human data” (Part III, p. 5-28). The animal-based ED01 values
listed in EPA’s Table 5-4 range from 22 to 30.9 ng/kg, leading to the
conclusion that “ A slope factor of 1.4 x 10_3 per pg dioxin/kg body weight/
day represents EPA’s most current upper bound slope factor for estimating
human cancer risk based on animal data” (Part III, p. 5-28).
Whereas a CSF of approximately 1 x 10_3 per pg/kg-day (equivalently,
an ED01 of approximately 30 ng/kg LABB) lies within the range of plausible
values, this discussion has focused on the relative magnitude of the range of
plausible values. Consideration of the set of all plausible parameter values
(that is parameter values within the 95% confidence interval) for the doseresponse functions considered by EPA considerably widened the range of
values estimated from the Becher et al. and Ott and Zober studies (see
Table 5-6). The CSF values (risk per pg/kg-day) can be calculated by first
converting the ED01 expressed as ng/kg LABB to an ED01 expressed as a
daily intake (ng/kg-day) using EPA equation 5-1 (Part III, p. 5-18) and then
dividing this intake into a risk of 0.01. For the Becher et al. (1998) study,
the resulting CSF values range from 3.0 x 10_5 to 1.0 x 10_3per pg/kg-day,
more than two orders of magnitude. The 95% confidence interval for the
CSF calculated from the Ott and Zober study (1996) has a lower bound of
zero and an upper bound of 1.2 x 10_3. Only the Steenland et al. (2001)
study retains an ED01 range (CSF range) with a span confined to less than
two orders of magnitude (CSF 6.1 x 10_4 to 3.0 x 10_2). Figure 5-2 com
pares the range of plausible CSF values identified by EPA with the range of
plausible values consistent with the dose-response parameter 95% confi
dence intervals.
Consideration of alternative points of departure can greatly inflate the
confidence interval for the power function dose-response relationships. Us
ing an ED05 (rather than an ED01) broadens the confidence interval for the
function to more than three orders of magnitude. Consideration of alterna
tive dose-response relationship forms could further broaden the range of
plausible CSF values, although primary analysis of the data would be re
quired to quantify the impact. The Reassessment could develop a distribu
tion for the CSF by assigning some probability to different options for each
of the assumptions discussed here (McKone and Bogen 1992; Evans et al.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
FIGURE 5-2 Range of plausible CSF values: consideration of parameter confi
dence intervals only. Solid blocks are central estimate values. For EPA, this value
(1,000) corresponds to EPA’s stated central estimate of dioxin’s CSF (1 x 10“3 per
pg/kg-day). For the Becher et al. (1998) study, the central estimate in the figure
corresponds to the average of the three ED0 1 values that EPA reports in Part III,
Table 5-4. For the Ott and Zober (1996) and Steenland et al. (2001) studies, the
central estimate corresponds to the individual ED01 values listed in EPA, Part III,
Table 5-4.
1994a,b). In any case, a more thorough consideration of plausible alterna
tive values for key assumptions is needed to portray accurately to risk
managers the magnitude of uncertainty that underlies the quantitative risk
estimates derived from epidemiological studies.
CONCLUSIONS AND RECOMMENDATIONS
Qualitative Weight-of-Evidence Carcinogen Classification
• The committee concluded that the classification of dioxin as “carci
nogenic to humans” versus “ likely to be carcinogenic to humans” depends
greatly on the definition and interpretation of the specific criteria used for
classification, with the explicit recognition that the true weight of evidence
lies on a continuum with no bright line that easily distinguishes between
these two categories. The committee agreed that, although the weight of
epidemiological evidence that dioxin is a human carcinogen is not strong,
the human data available from occupational cohorts are consistent with a
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modest positive association between relatively high body burdens of dioxin
and increased mortality from all cancers. Positive animal studies and mecha
nistic data provide additional support for classification of dioxin as a hu
man carcinogen. However, the committee was split on whether the weight
of evidence met all the necessary criteria described in the cancer guidelines
(EPA 2005a, see also Appendix B) for classification of dioxin as “carcino
genic to humans.” EPA should summarize its rationale for concluding that
dioxin satisfies the criteria set out in the most recent cancer guidelines (EPA
2005a, see also Appendix B) for designation as either “carcinogenic to
humans” or “likely to be carcinogenic to humans.”
• The committee agreed that other DLCs are most appropriately clas
sified as “likely to be carcinogenic to humans.”
• Should EPA continue to classify dioxin as “carcinogenic to humans,”
more justification will be required to rationalize why a mixture containing
dioxin would not also meet the classification of “carcinogenic to humans.”
• If EPA continues to designate dioxin as “carcinogenic to humans,” it
should explain whether this conclusion reflects a finding that there is a
strong association between dioxin exposure and human cancer or between
dioxin exposure and a key precursor event of dioxin’s mode of action
(presumably AHR binding). If EPA’s finding reflects the latter association,
EPA should explain why that end point (e.g., AHR binding) represents a
“key precursor event.”
• The committee considers the distinction between these two catego
ries to be based more on semantics than on science and recommends that
EPA spend its energies and resources more carefully delineating the as
sumptions used in quantitative risk estimates for TCDD, other dioxins, and
DLCs derived from human and animal studies.
Quantitative Risk Estimation of Cancer Potency
• The committee concludes that there is an adequate scientific basis to
support the hypothesis that the shape of the relationship between dioxin dose
and cancer risk is sublinear at low doses, perhaps reflecting responses indis
tinguishable from background risk at doses below which dose-response data
are available, including evidence that (1) TCDD, other dioxins, and DLCs are
not genotoxic; (2) dioxin acts through receptor mediation, and receptormediated carcinogens tend to exhibit sublinear dose-response relationships;
(3) dioxin-induced liver tumors are secondary to hepatotoxicity and enhanced
rates of cell proliferation; (4) bioassay results suggest sublinearity (Hill coef
ficient central estimates substantially greater than 1); and (5) epidemiological
results do not help to distinguish between zero and nonzero responses at the
low-dose end of the dose-response curve. Accordingly, a risk assessment can
be conducted without resorting to default assumptions.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
• To the extent that EPA favors using default assumptions for regulat
ing dioxin as though it were a linear carcinogen, such a conclusion should
be supported with scientific evidence. (For example, EPA could explore
whether background exposures raise the population to the linear portion of
the dose-response relationship.) Alternatively, the decision to use the linear
dose-response relationship could be made as a part of risk management,
although the risk assessment should provide the scientific strengths and
weaknesses for both linear and nonlinear approaches. EPA should adhere
to the division between risk assessment, which is a scientific activity, and
risk management, which takes into account other considerations, as de
scribed by the National Academy of Sciences more than two decades ago
(NRC 1983).
• EPA has not adequately justified use of the 1% excess risk level as
the POD for the analysis of either the epidemiological or animal bioassay
data. Although demonstrating that the POD is within the range of the data
is necessary, it is not sufficient to justify its use. Other conditions, such as
demonstrating that the POD is relatively insensitive to functional form (as
noted in EPA’s cancer guidelines), must also be satisfied. EPA should ac
knowledge the larger extrapolation from justifiable POD values down to
environmentally relevant doses that would be necessitated by use of a
higher-response-level POD.
• Regarding EPA’s review of the animal bioassay data, the committee
recommends that EPA establish clear criteria for including different data
sets. The reliance on one site from one gender of one species, as reported by
a single study, does not adequately represent the full range of data avail
able. The committee recommends that EPA consider the full range of data,
including the new NTP animal bioassay study on TCDD for quantitative
dose-response assessment.
Characterization of Uncertainty Surrounding Cancer Risk Estimates
• EPA should characterize more completely the uncertainty associated
with risk estimates inferred from the epidemiological data by (1) taking into
account the full range of EDxx values statistically consistent with the data
(not just the central and lower estimates), (2) considering alternative PODs,
(3) considering alternative dose-response functional forms consistent with
the data, and (4) considering uncertainty associated with the half-life esti
mates of dioxin in humans for the purpose of back-extrapolating exposures
in occupational cohort studies.
• The committee recognizes that explicit characterization of uncer
tainty could result in an especially wide range of risk estimates. Narrowing
consideration to a subset of those estimates could be made as part of the
risk management task. For example, a “health protective” (conservative)
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estimate could be used to support an imperative to protect public health.
Alternatively, if the goal is to compare that risk with other risk manage
ment priorities, countervailing risks, or the economic costs of risk mitiga
tion, a central or arithmetic mean value could be used. Finally, to address
uncertainty associated with specification of the dose-response relationship
functional form below the POD (that is, linear vs. nonlinear), EPA could
choose to use a margin of exposure approach in place of estimating popu
lation risk. These options are the purview of risk management rather than
risk assessment.
• On the whole, it was the committee’s impression that EPA’s narra
tive in discussing epidemiological studies in Part III of the Reassessment
tended to focus on positive findings without fully considering the strengths
and limitations of both positive and negative findings. Part III of the
Reassessment would be strengthened if EPA clearly identified specific in
clusion criteria for those studies for which quantitative risk estimates were
determined.
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6
N o n c a n c e r E n d P oin ts
This chapter reviews the U.S. Environmental Protection Agency (EPA)
assessment of the noncancer end points, including immune function, repro
duction and development, diabetes, thyroid function, lipid levels, and other
effects related to exposure to 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD,
also referred to as dioxin), other dioxins, and dioxin-like compounds
(DLCs) in animals and humans. The purpose of this chapter is to critically
assess, to the extent possible, whether EPA has met the criteria set forth in
the “ Statement of Task” with respect to the noncancer effects of TCDD,
other dioxins, and DLCs. Toward this end, this chapter focuses on the
uncertainties and assumptions made by EPA in determining whether TCDD,
other dioxins, and DLCs have noncancer effects in humans; determining
the methods and models used for assessing these effects; determining the
breadth and robustness of the studies used and the balance with which the
studies are presented in the Reassessment,1 and finally, determining whether
the conclusions reached by EPA are consistent with the current scientific
peer-reviewed literature.
IMMUNE FUNCTION
EPA uses a sizeable immunotoxicology database derived largely from
laboratory animal studies and a smaller number of epidemiological studies in
1The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
144
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its assessment of immunotoxicity produced by TCDD, other dioxins, and
DLCs.
The assessment of changes in immune competence, regardless of the
cause, is complex, as the immune system is composed of a large and diverse
group of cellular and soluble components. In addition, compensatory and
overlapping mechanisms in host immunity can make it difficult to identify
subtle or modest changes within the immune system.
Because of the many different cell types and soluble factors, which
alone or cooperatively mediate a wide variety of evocable immunological
responses, there is no one test or assay that can measure all the different
elements. Therefore, the approaches used to identify changes in immune
status are multifaceted, including pathological examination of lymphoid
organs, enumeration of leukocyte subpopulations, quantification of
soluble mediators, and measurement of immune function responses, such
as susceptibility to infection or reduced immunological responses to vac
cines. Standard assays are available for all the above determinations;
however, because of the sheer enormity of the task to measure them all,
immune competence is typically assessed by using a small number of
immunological end points, often quantifying various aspects of innate,
humoral, and cell-mediated immunity. Therefore, the EPA review draws
on a large but diverse database of studies that are often difficult to com
pare because different assays, model systems, responses, and animal spe
cies were used.
EPA draws several important conclusions about the immunotoxicity
of TCDD, other dioxins, and DLCs that are summarized in the Reassess
ment, Part III, Integrated Summary and Risk Characterization. The first is
that “there appears to be too little information to suggest definitively that
2,3,7,8-TCDD at levels observed (in the reported studies) causes long
term adverse effects on the immune system in adult humans” (p. 2-34;
lines 19 to 21). The second is that “ cumulative evidence from a number of
studies indicates that the immune system of various animal species is a
target for toxicity of TCDD and structurally-related compounds, includ
ing PCDDs, PCDFs [polychlorinated dibenzofurans] and PCBs [polychlo
rinated biphenyls]” (p. 2-34; lines 24 to 26). Third, animal studies show
that TCDD suppresses both “ cell-mediated and humoral immune re
sponses, suggesting that there are multiple cellular targets within the im
mune system that are altered by TCDD” (p. 2-34; lines 26 to 28). EPA
goes on to state that “it can be inferred from the available data that
dioxin-like congeners are immunosuppressive” in animals. Fourth, the
weight of evidence from animal studies in vivo and in vitro “ supports a
role for Ah-mediated immune suppression” by DLCs (p. 2-35, lines 27 to
28); “ other in vivo and in vitro data, however, suggest that non-AHR
(aromatic hydrocarbon receptor)-mediated mechanisms may also play
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some role in immunotoxicity” (p. 2-35, lines 28 to 30). Finally, EPA
concludes that “ there are insufficient clinical data from these studies to
fully assess human sensitivity to TCDD exposure. Nevertheless, because
of extensive animal work, the database is sufficient to indicate that im
mune effects could occur in the human population from exposure to
TCDD and related compounds at some dose level. At present, it is EPA’s
scientific judgment that TCDD and related compounds should be re
garded as nonspecific immunosuppressants and immunotoxicants until
better data to inform judgment are available” (p. 2-37, lines 4 to 10). The
strengths and weaknesses of those conclusions are discussed below.
Uncertainties and Assumptions in Determining Whether TCDD Is
Immunotoxic in Humans
Is the Assumption Correct That the Immune System in Humans and in
Animal Models, Primarily Mice, Are Similar?
Historically, the mouse has been the animal model of choice for
immunologists; it has also been widely embraced as the model of choice
for immunotoxicological studies. Hence, most immunotoxicological
studies used by EPA in preparing its report are based on studies in
mouse models. The human and mouse immune systems are similar in
composition and function; therefore, from the standpoint of compari
sons based on the composition of this target organ, it is reasonable to
assume that studies in mice provide important qualitative insights into
the mechanism of action of TCDD, other dioxins, and DLCs on the
human immune system. Providing, insights into the mechanism of ac
tion is one of the primary strengths of the mouse model for which there
are genetically defined AHR high- and low-responding mouse strains,
congenic mice at the Ahr locus, and AHR null (Ahr-/-) mice. More
reagents, assays, and biological probes are available for the mouse
immune system than for any other species except the human immune
system. However, as is the case for other toxicological end points,
information-derived from animal studies is qualitative in that the phar
macodynamics, pharmacokinetics, half-lives of the compounds, affin
ity of AHR, linkage of the receptor to signal transduction pathways,
and numerous other factors can be significantly different, at least quan
titatively, between humans and other animal species. Therefore, direct
quantitative extrapolations from animal models to humans can result
in a significant underestimation or overestimation of risk. When strong
scientific evidence exists concerning specific species differences, these
factors should be incorporated into the risk characterization.
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Is the Toxic Equivalency Factor/Toxic Equivalent Quotient Approach for
Estimating the Immunotoxicity of Mixtures Scientifically Justified?
The toxic equivalency factor/toxic equivalent quotient (TEF/TEQ)
approach is based on a well-defined structure-activity relationship for
persistent dioxins, other than TCDD, and DLCs for which there is a
positive correlation between AHR affinity and toxic potency. There is
good general agreement in comparisons of results across studies, prima
rily in mice, where the acute immunotoxic effects for individual dioxin
like congeners were examined, suggesting that the immunotoxic potency
for various congeners and AHR activation exhibit the same qualitative
rank order. Few immunotoxicological studies investigated structure-ac
tivity relationships for immunotoxic potency. Of those studies, with only
a few exceptions, the rank order immunotoxic potency correlated posi
tively with AHR activation. Several exceptions to this relationship are
found in the current literature, and some are discussed in the Reassess
ment, Chapter 4, Part II. For example, 2,7-dichlorodibenzo-p-dioxin, a
congener that would be predicted to exhibit low binding affinity for AHR,
was found to be equipotent in suppressing the anti-sheep red-blood-cell
(anti-SRBC IgM) antibody-forming response to TCDD. For this example,
the TEF/TEQ approach would not provide a reasonable estimate of
immunotoxicity. Because in vivo immunotoxicity data for 2,7-dibenzo-pdioxin are available only in the mouse, it is unclear whether the unex
pected potency of this congener occurs in other animal species and hu
mans. A second example pertains to certain halogenated aromatic
hydrocarbons (HAHs), including several diortho PCB congeners, which
exhibit antagonist activity when administered in a mixture with AHR
agonists. The latter point may not be trivial, as several of the diortho PCB
congeners (e.g., PCB153) are abundant environmental cocontaminants
with dioxins, other than TCDD, and DLCs. The aforementioned examples
of exceptions for which the TEF/TEQ approach might not predict toxic
potency accurately are presented and discussed in a balanced manner
(Part II, pp. 4-6 to 4-7, lines 19 and 20).
In spite of the aforementioned caveats, based on what is known about
the cell biology of AHR and the mechanisms of immunotoxicity for TCDD
and related compouds, the TEF/TEQ approach for assessing the immunotoxic potency of mixtures of persistent dioxins, other than TCDD, and
DLCs is scientifically justified. Having said that, the effective application of
the TEF/TEQ approach for assessing immunotoxic potential ultimately
would critically depend on the TEF values assigned to individual congeners
for a given immunological response. What is unclear is which immune
responses should be used in risk management, as all are not equally sensi
tive to modulation by HAHs.
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What Is the Profile of Immune Toxicity in Humans Exposed to DLCs?
Well-documented human exposure to TCDD, other dioxins, and DLCs
has occurred in the occupational setting and within the general population
from industrial accidents and through consumption of contaminated food.
Even though immune function and status have been examined in exposed
individuals, only a small number of studies have been conducted with
appropriate controls and accurate measures of exposure. Likewise, a small
number of epidemiological investigations evaluating immunologically re
lated outcomes from chronic exposures have been reported. The results
from these human studies for the most part have yielded inconclusive re
sults. In several studies, modest changes in immune status were observed;
yet in other studies, the findings were not reproduced or were even contra
dicted. In animal studies, it is clear that TCDD, other dioxins, and DLCs
markedly suppress both humoral and cell-mediated immune responses. This
profile of activity has not been unequivocally demonstrated in humans. The
most obvious reason for the inconclusive findings in human studies is that,
in many cases, a very small number of subjects were evaluated, their pri
mary exposures were often long before measurement of immune end points
and concentrations of TCDD and related compounds, and the nature of
those exposures were often unclear. Furthermore, obtaining an accurate
estimate of the level of exposure through back-extrapolation from current
body levels may be more complex than simply using a single half-life
throughout.
Another contributing factor to the inconsistent results is that immune
responses to defined stimuli are highly variable among humans. This vari
ability can be attributed to genetic variability, age, environmental history,
and other still undefined causes. This variability in individuals substantially
limits the ability to identify subtle and even moderate alterations of immune
function after exposure to agents, especially in human populations. There
fore, in the absence of more comprehensive immunotoxicological human
data, it is reasonable to assume that TCDD, other dioxins, and DLCs will
exert a profile of immunotoxicity comparable to that observed in animal
models, such as mice. For risk analysis, the more critical issue is whether the
immunotoxic potency observed in certain animal models is significantly
greater (by orders of magnitude) than in humans.
What Is the Sensitivity of the Human Immune System to DLCs?
For many of the same reasons as discussed in the previous section, the
sensitivity of humans to immune suppression by TCDD, other dioxins, and
DLCs is also currently unclear. There are four separate reports of a longitu
dinal study in a cohort of Dutch children suggesting that the developing
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human immune system may be susceptible to immunotoxic alterations
from exposure to Western European environmental levels of TCDD, other
dioxins, and DLCs (Weisglas-Kuperus et al. 1995, 2000, 2004; ten
Tusscher et al. 2003). Exposures before 1990 in the Netherlands resulted
in breast milk concentrations of TEQ (at the start of the study) at 30 to 60
parts per trillion (ppt) (lipid), whereas concentrations in the United States
at approximately the same time period were in the 15- to 24-ppt range.
Three industrial areas were compared with a rural area with about 20%
less PCBs in maternal plasma (Koopman-Esseboom et al. 1995). The
Dutch study measured the major PCB congeners in plasma in the mother
and the newborn and all TCDD and related compounds in maternal breast
milk 10 days and 3 months postpartum (Feeley 1995). About half the
study subjects were fed a formula that had TCDD and related compounds
at less than 2 ppt, and the other half of the test subjects were breast-fed
and could be divided into groups breast-fed less than 4 months and those
breast-fed more than 4 months. Therefore, individual calculations of total
exposure could be correlated with plasma PCB concentrations in children
at 3 months, 18 months, 42 months, and 9 years. Three more recent
studies (Weisglas-Kuperus et al. 2000, 2004; ten Tusscher et al. 2003) are
important because some of the same findings observed in these later stud
ies were also reported in the 1995 studies, indicating persistence of alter
ations. Weisglas-Kuperus et al. (2000) reported on 207 healthy motherinfant pairs with increased prenatal exposure to TCDD, other dioxins,
and PCBs; the results showed an association between exposure and immu
nological changes, which included an increase in number of lymphocytes,
Y-S T cells, CD3+HLA-DR+ (activated) T cells, CD8+ cells, CD4+CD45RO+
(memory T cells), and lower antibody levels after mumps and measles
vaccination at preschool age. In addition, an association was found be
tween prenatal exposure and decreased shortness of breath with wheeze,
and current PCB burden was associated with a higher prevalence of recur
rent middle-ear infections and chicken pox and a lower prevalence of
allergic reactions. Although an association between TCDD and PCB ex
posure and changes in immune status was observed, all infants were found
to be in the normal range. In a second study, ten Tusscher et al. (2003)
reported modest but persistent changes in immune status in children with
perinatal exposure to dioxin as evidenced by a decrease in allergy, persis
tently decreased thrombocytes, increased thrombopoietin, increased CD4+
T cells, and increased CD45RA+ cell counts in a longitudinal subcohort of
27 healthy 8-year-old children with documented perinatal exposure to
TCDD. The original cohort at 42 months demonstrated an association
between reduced vaccine titers, increased incidence of chicken pox, and
increased incidence of otitis media with higher TEQ. However, by 8 years
of age, the more frequent recurrent ear infections were still apparent
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(overall), although the chicken pox frequency showed an inverse correla
tion with PCB and TCDD concentrations. The subcohort in the ten
Tusscher study was small, and therefore the results are not as robust as
those in the Weisglas-Kuperus et al. (2004) study, which included almost
91% of the original 2000 study cohort.
Animal studies also suggest that the developing immune system is sen
sitive to persistent changes in immune function or status, especially when
exposure occurs in the perinatal and neonatal stage and especially in T-cellmediated immunity. Less compelling studies exist from which to estimate
the sensitivity of the adult human immune system.
EPA concludes that
there is insufficient clinical data from these studies to fully assess human
sensitivity to TCDD exposure. Nevertheless, based on the results of the
extensive animal work, the database is sufficient to indicate that the im
mune effects could occur in the human population from exposure to
TCDD and related compounds at some dose level. At present, it is EPA’s
scientific judgment that TCDD and related compounds should be regard
ed as nonspecific immunosuppressants and immunotoxicants until better
data to inform judgment are available. (Reassessment, Part III, p. 2-37,
lines 4 to 10)
Indeed, based on the extensive animal data, it is reasonable and pru
dent for EPA to regard TCDD as an immunotoxicant. Furthermore, the
Dutch study provides some suggestive evidence for this conclusion. How
ever, it is unclear in EPA’s conclusion what is meant by TCDD and related
compounds being regarded as “ nonspecific immunosuppressants and
immunotoxicants” and this should be clarified.
How Persistent Are the Immunotoxic Effects of TCDD on the
Human Immune System (Reversible Versus Irreversible Changes)?
Because it has not yet been unequivocally established that TCDD in
duces immune suppression in humans, it is not possible at this time to
delineate the persistence of TCDD-mediated immunotoxicity in humans.
The lowered total white-blood-cell numbers reported in the studies of Dutch
children (Ilsen et al. 1996) were no longer evident 2 years after birth
(Weisglas-Kuperus et al. 2000). The elevated T-cell subpopulations in Dutch
children at 42 months of age did not appear to persist at 8 to 10 years of age
(ten Tusscher et al. 2003). However, Weisglas-Kuperus et al. (2004) also
reported a positive association in children (3 to 7 years of age) between
increased postnatal PCB exposure and increased prevalence in recurrent
middle ear infections. In addition, there was a positive association between
increased prenatal PCB exposure and decreased chicken pox frequency as
well as allergy and asthma. A second recent report (Van den Heuvel et al.
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2002) suggested that TCDD, other dioxins, and DLCs may produce subtle
but persistent changes in immune status as evidenced by a reduction in
allergy and asthma. In this study of 200 Flemish adolescents, a negative
correlation was observed between exposure to TCDD, other dioxins, and
DLCs with TCDD TEQ measured and allergic responses in airways. In
addition, serum immunoglobulin G levels were also negatively correlated
with PCB exposure.
Relevance of Rodent Models to Human Quantitative Risk
Assessment for Immunotoxicity
In the Reassessment, Part III, Appendix A, Table A-1, EPA centers its
risk characterization for adult immunological effects on four studies con
ducted in mice (Vecchi et al. 1983; Narasimhan et al. 1994; Smialowicz et
al. 1994; Burleson et al. 1996) and its developmental immunotoxicological
risk characterization on a single rat study (Gehrs and Smialowicz 1999) (see
Appendix A, Table A-1; for immune end points; see Figures 12 to 15, pp. A7 to A-9; same studies identified in Table 5-6, p. 5-39, in the Reassessment,
Part III). Based on these studies, lowest-observed-adverse-effect levels
(LOAELs) and no-observed-adverse-effect levels (NOAELs) are used to de
rive ED01 (1% effective dose) and human equivalent intake values. Because
of the importance that these studies have to the Reassessment and the
potential importance that the derived values may have for risk manage
ment, some additional comments are provided here.
The study of Burleson et al. (1996) showed the lowest LOAEL (6 ng/
kg), and NOAEL (3 ng/kg) values, which result in calculated “ human
equivalent intakes” of 1 pg/kg/day and 2 pg/kg/day, respectively. The study
showed that the sensitivity to alteration by TCDD of host resistance of mice
to H3N2 influenza A (Hong Kong/8/68) virus is strikingly sensitive com
pared with other LOAELs for immunotoxicological end points in adult
rodents given in Table A-1, the LOAEL ranging from 100 to 1,200 ng/kg.
In fact, the sensitivity to TCDD in the Burleson et al. study is striking even
when compared with similar and more recently published host-resistance
studies using influenza virus. For example, Nohara et al. (2002) showed
that TCDD doses up to 500 ng/kg did not increase mortality in a number of
different strains of mice, including B6C3F1, C57Bl/6, Balb/c, and DBA/2
mice infected with influenza A virus (A/PR/34/8, H1N1). More mice per
group were used in the Nohara study than in the Burleson study, thus
providing even greater statistical power. In a study using influenza A/
HKx31, Warren et al. (2000) reported that in certain experiments, TCDD
treatment (1 to 10 pg/kg) increased mortality, whereas in other experiments
no mortality was observed. Furthermore, Warren and coworkers stated
that in some experiments TCDD doses as high as 10 pg/kg produced no
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mortality, whereas 80% mortality was observed at the same dose in other
experiments. Such data emphasize the variability typically observed in hostresistance studies. The reason for the significantly greater sensitivity to
TCDD in the Burleson et al. (1996) study is unclear but strongly suggests
that further studies are needed before using results from this study for risk
characterization.
The LOAEL, NOAEL, ED01, and human equivalent intake values for
immunotoxicity are based on suppression by TCDD of the anti-SRBC IgM
antibody-forming cell response in studies by Vecchi et al. (1983),
Narasimhan et al. (1994), and Smialowicz et al. (1994) (the other three
studies identified in Table A-1 and used for risk characterization of the
adult immune system). Numerous laboratories have demonstrated suppres
sion of the antibody-forming cell response by TCDD, and in general, there
is good concordance in the ED50 doses (600 to 770 ng/kg) derived from
these studies (see Table 4-1, p. 4-38) (Vecchi et al. 1980; Davis and Safe
1988; Kerkvliet and Brauner 1990; Kerkvliet et al. 1990). In contrast, some
variability in the LOAEL values identified in Table A-1 were observed in
three other studies: 100 ng/kg (Narasimhan et al. 1994), 300 ng/kg
(Smialowicz et al. 1994), and 1,200 ng/kg (Vecchi et al. 1983). The varia
tion is due primarily to dose selection in each of the studies. It is clear how
the LOAEL and, in the case of the Narasimhan study, the NOAEL were
identified from the data presented in each of the published reports. It is not
clear, however, how the ED01 values were calculated.
Several concerns also exist about using the Gehrs and Smialowicz
(1999) study for characterizing developmental immunotoxicological risk
by TCDD. The Gehrs and Smialowicz study gives no indication as to the
number of rat offspring studied; therefore, it is unclear whether the results
are robust. In addition, both males and females were found to be more
sensitive to immune suppression by TCDD after 14 months of age than at 4
months of age, as measured by a delayed type hypersensitivity response,
which is somewhat puzzling. Although hypotheses could be advanced to
explain these unexpected findings, it would be valuable and prudent to
repeat that study before using those results for characterizing developmen
tal immunotoxicological risk by TCDD.
Congruence with Full Document
In Part II, Chapter 4 of the Reassessment, a comprehensive and bal
anced synthesis of the immunotoxicology literature on TCDD, other diox
ins, and DLCs is presented. Results from more than 200 published studies
are discussed in an organized and logical manner. Moreover, there is good
congruence between Chapter 4 and section 2.2.3 on immunotoxicity in the
Executive Summary.
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CONCLUSIONS AND RECOMMENDATIONS ON THE
IMM UNOTOXICITY OF TCDD, OTHER DIOXINS, AND DLCS
• Present clinical findings are inconclusive about whether or in what
way TCDD, other dioxins, and DLCs are immunotoxic in humans, a con
clusion that EPA acknowledges, and human data are also sparse. Perhaps
the most compelling data that these compounds are human immunotoxicants, at possibly relevant environmental levels, come from the studies of
the Dutch children’s cohort. These studies show an association between
prenatal exposure and changes in immune status. However, the effects are
modest and do not lie significantly outside the full range of normal. The
correlation of increased otitis media in the very young with perinatal TEQ
is the only statistically significant immunological clinical finding. Some of
the same findings were made in acutely exposed Taiwanese and Japanese
cohorts (Yu et al. 1995). Concordant with findings in Dutch children are a
number of animal studies that also suggest that the developing immune
system is especially sensitive to modulation by TCDD, other dioxins, and
DLCs. Collectively, in light of the large database showing that these com
pounds are immunotoxic in laboratory animal studies together with sparse
human data, EPA is being prudent in judging TCDD, other dioxins, and
DLCs to be potential human immunotoxicants in the absence of more
definitive human data.
• EPA’s conclusion that TCDD, other dioxins, and DLCs are immunotoxic at “ some dose level” by itself is inadequate. At a minimum, a section
or paragraph should be added that discusses the immunotoxicology of
these compounds in the context of current AHR biology. Specifically, there
is evidence showing that the affinity of TCDD for the human AHR is at
least an order of magnitude lower than that in high-responding Ahrb-1
mouse strains (Ramadoss and Perdew 2004), which has been the most
commonly used animal model for investigations of immunotoxicity of
TCDD, other dioxins, and DLCs. Other properties of AHR, in addition to
binding affinity, such as specificity for target genes and transactivation
potential, will contribute to the toxicity produced by AHR ligands. Never
theless, EPA supports a TEF/TEQ approach for estimating the immunotoxic
potency of mixtures of dioxins, other than TCDD, and DLCs. The Reas
sessment assumes that immunotoxicity is therefore primarily mediated
through an AHR-dependent mechanism, so some discussion should be
included acknowledging the possibility that rodents, especially certain
mouse strains expressing Ahrb-1 might be significantly more sensitive to the
immunotoxic effects of TCDD, other dioxins, and DLCs than humans.
Some discussion should also be included on the strengths and weaknesses of
using genetically homogeneous inbred mice to characterize immunotoxicological risk in the genetically variable human population. Expanding the
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
discussion to include the above crucial points would provide additional
balance to Part III of the Reassessment.
• EPA centers its risk characterization for adult immunological effects
on four studies conducted in mice (Burleson et al. 1996; Smialowicz et al.
1994; Narasimhan et al. 1994; Vecchi et al. 1983), and its developmental
immunotoxicological risk characterization on a single rat study (Gehrs and
Smialowicz 1999) (see Part III, Appendix A, Table A-1; for immune end
points; see Figures 12 to 15, pp. A-7 to A-9). Concerns about Table A-1 are
the following:
• The calculations of ED 0 j values and the scientific assumptions made
in deriving those values need further clarification. Likewise, EPA should
provide a clear scientific rationale for selecting ED01 as a benchmark dose.
• Considerations of the Burleson et al. (1996) study with no consider
ation of two similar studies—Nohara et al. (2002) and Warren et al.
(2000)—that yield very different results requires justification.
• On the basis of concerns discussed earlier, it would be prudent to
replicate the Gehrs and Smialowicz (1999) study before using its results for
characterizing developmental immunotoxicological risk of TCDD.
An important animal study by Oughton et al. (1995) was not included
in either Part II or the tables in the Executive Summary of the Reassessment.
The importance of the study is that it is the only low-level chronic exposure
investigation published (TCDD at 200 ng/kg/week once a week to Bb mice
at 2 to 16 months of age) in which immunotoxicological parameters have
been assessed—specifically, a phenotypic analysis by flow cytometry of
major cell subpopulations in the mouse spleen, thymus, and peripheral
blood. The study showed only subtle alterations in the immune system as
demonstrated by a modest increase in y-S T cells, which the authors consid
ered “ questionable biological relevance,” and a small decrease in the fre
quency of memory CD4 cells (by phenotype). However, these changes,
although of questionable biological relevance, have also been observed in
humans and in high-exposure animal studies.
REPRODUCTION AND DEVELOPMENT
Animal Data
EPA provided an overview of the effects of TCDD, other dioxins, and
DLCs on development and reproduction based on published animal studies
and accidental human exposures. Determination of the alterations in devel
opment and reproduction is a highly complex process because hormonal as
well as intracellular processes and compensatory mechanisms, including
hormonal feedback mechanisms, are affected. The Reassessment compre-
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hensively covers developmental aspects in a wide variety of models. Two
major rodent models have been used to study the effects of TCDD on
reproduction and development. In the first model, TCDD is given during
pregnancy (an in utero and lactational exposure model). This model tested
the ability of TCDD to disrupt development of the pups and assessed the
effects on reproduction and reproductive behavior later in life. A compre
hensive overview of the in utero and lactational exposure model is pre
sented, but the doses used and how the model relates to the human repro
ductive and developmental toxicity are not emphasized. For example,
maternal concentrations of TCDD in plasma are needed at designated times
during pregnancy and lactation in the rat dam; those data would allow
comparison to human data and determination of whether concentrations in
rodents are higher or lower than those in humans accidentally exposed to
TCDD. In the second model, adult rats and an immature gonadotropinprimed model are used to assess the effects of TCDD on ovulation. These
models were not adequately discussed. In addition, there is uncertainty in
the risk assessment based on differences in TEF reported by the World
Health Organization (WHO) (Van den Berg et al. 1998) when compared
with published data. For example, the WHO 1998 TEQs appear to be 2.5fold higher (Van den Berg et al. 1998) than actual potency data determined
with these models (Safe 1990; Gao et al. 1999, 2000a,b). Studies have been
conducted using the 1998 TEFs (e.g., Hamm et al. 2003), and the conclu
sions seemed to indicate that mixture doses two to three times higher than
the calculated TEQ appeared to be required to elicit the same alterations.
The study by Hamm et al. is comprehensive and revealed numerous adverse
effects on male and female reproduction, such as prolonged time to pu
berty, decreased seminal vesicle and ventral prostate weights, and, in the
female, increased the incidence of vaginal threads. The lowered responses
to the mixture of TCDD, other dioxins, furans, and coplanar PCBs were
attributed to decreased transfer of mixture components to the offspring,
whereas a miscalculation of the TEQ might have also contributed to the
lowered response (Hamm et al. 2003). In addition, if the mixture was
altered to favor what might have been present in the diet in nature, then the
true TEQ might not have been accurately represented in the Hamm et al.
study. However, given that the WHO TEFs are order-of-magnitude esti
mates of the relative potency of a chemical and derived from all toxicologi
cal outcomes in a variety of species, it is not surprising that there is a lack of
absolute concordance between a calculated TEQ and an actual TEQ, mea
sured in one species, for male and female reproduction end points. The
generation of end-point-specific TEFs would probably resolve the observed
differences between calculated and measured TEQs on these end points.
Examples of complete dose responses on various reproductive param
eters in females using various polychlorinated dibenzo-p-dioxins (PCDDs),
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polychlorinated dibenzofurans (PCDFs), and PCBs are given below. Trace
levels of PCDDs have been detected in fish, wildlife, and humans (Van den
Berg et al. 1985; Tiernan et al. 1985), and PCDDs have toxicological effects
on the reproduction and development in vertebrates, including rodents and
nonhuman primates (Van den Berg et al. 1994). Few studies have evaluated
the effects of complex mixtures of PCDDs and DLCs on the female repro
ductive system. Previous studies have validated the TEQ concept for several
PCDDs in acute and subchronic/chronic experiments using several biologi
cal end points (Stahl et al. 1992, Weber et al. 1992, 1994; Rozman et al.
1993, 1995; Viluksela et al. 1997a,b and 1998a,b).
Female reproductive toxicity of TCDD is evidenced by reduced ovulation
(Li et al. 1995a,b) and developmental defects (Heimler et al. 1998a), which
were orders of magnitude less than the cancer response (Kociba et al. 1978;
1979; Rozman et al. 1993), indicating that ovulation and development are
more sensitive end points because lower doses are needed to disrupt repro
ductive processes than to increase the incidence of cancer. Studies using
gonadotropin-treated immature rats revealed that complex mixtures of
PCDDs, such as TCDD, pentachlorodibenzo-p-dioxin (PeCDD), and
hexachlorodibenzo-p-dioxin (HxCDD), as well as each congener alone pro
duced dose responses that lowered ovarian weights and the number of ova
shed (Gao et al. 1999). In addition, the effects of PCDDs were additive when
an equipotent mixture of the PCDDs was given. The slopes of the doseresponse curves were not statistically different among the various congeners.
Thus, the additive effect and parallel dose-response curves indicated a similar
mechanism of action. The PCDFs and PCBs, with TCDD-like actions, also
have a similar inhibitory effect on ovulation. The studies of Gao et al. (1999)
and others (Krishnan and Safe 1993) are in close agreement and indicate a
TEF of 0.12 to 0.2 for PeCDD, which differs from the TEF of 0.5 proposed
by WHO (Van den Berg et al. 1998). The doses required in the ovulation
study for PCDDs (Gao et al. 1999) to produce the same effect increased
approximately 5-fold for each chlorine added to TCDD. Those observations
are consistent with prior studies (Stahl et al. 1992) and imply a TEF based on
female reproductive effects of 0.2 for PeCDD and 0.04 for HxCDD, which
differ from the WHO report in which a TEF of 1 was given for PeCDD (Van
den Berg et al. 1998) and used by Hamm et al. (2003) for numerous repro
ductive studies in males and females.
TEFs for the pentachloro-isomers of PCDPs and PCBs are in the same
range as those previously reported for other end points (Safe 1990; Van
Birgelen et al. 1994a,b, 1996). However, the WHO conference (Van den
Berg et al. 1998) reported values of 0.5 and 0.1 for 2,3,4,7,8pentachlorodibenzofuran (PeCDF) and 3,3',4,4',5-PCB, respectively, which
are twice as high as most studies report. The doses of the pentachloroisomers of PCDs and PCBs studied by Gao et al. (2000b) had 10-fold lower
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potency than the ED50 for TCDD in blocking ovulation (8 pg/kg) (Gao et
al. 2000b). Generally, TEF values are combined from all end points, and
development of end-point-specific TEFs might ultimately be useful. How
ever, it must be noted that TEFs are not expected to be exact, and the values
determine in the reproductive studies are well within an order of magni
tude.
The Reassessment was mainly directed at understanding the adverse
effects of TCDD administered during pregnancy on development of the
pups; that section was superbly written and covered numerous aspects of
exposure to TCDD in utero and during lactation. However, little risk esti
mate information is given. Also, an important part of the literature on the
adult female reproductive system was not addressed in the Reassessment.
This included mechanisms of ovulatory blockage at the level of the hypo
thalamic-pituitary axis and the ovary and endocrine disruption of repro
ductive processes by TCDD in adult rodents (Goldman et al. 2000; Petroff
et al. 2001; Valdez and Petroff 2004). The committee summarizes some of
those studies in the following section.
Effects of PCDDs on the Ovary
Studies have shown that PCDDs adversely affect ovarian function by
direct actions on the ovary and the hypothalamic-pituitary axis (Petroff et
al. 2000; Valdez and Petroff 2004). Human ovarian follicular fluid has
been found to contain PCDDs (Tsutsumi et al. 1998), implicating PCDDs
in possible adverse ovarian effects. Exposure of adult female rats to PCDDs
disrupted estrous cycles, delayed ovulation, and lowered ovarian weights
(Li et al. 1995a; Cummings et al. 1996). Irregular menstrual cycles were
observed in female rhesus monkeys fed TCDD in the diet (Allen et al. 1977;
Barsotti et al. 1979). Mice are less prone to the adverse ovarian effects of
PCDDs in some studies (Cummings et al. 1996), although TCDD caused
the formation of ovarian cysts in CD-1 mice (Gallo et al. 1986). In rats,
administration of TCDD before mating interrupts fertility by affecting both
ovulation and implantation (Giavini et al. 1983). In the immature gonadot
ropin-primed rat, the adverse effects of PCDDs on the ovary were charac
terized by small ovaries, the absence of corpora lutea, and numerous
unruptured preovulatory follicles (Gao et al. 1999, 2000b; Petroff et al.
2001). In the immature rat primed with gonadotropin, the number of ova
shed in response to PCDDs was dose-dependently inhibited with an ED50 of
TCDD at 8 pg/kg of body weight. This supported the TEQ for several other
AHR agonists, including PeCDD, HxCDD, PeCDF, and pentachlorobiphenyl (PeCB) (Li et al. 1995a,b; Gao et al. 1999, 2000b; Son et al. 1999;
Petroff et al. 2000, 2001). TCDD suppressed follicular development as
determined by a reduction in the number of antral and preantral follicles in
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the pups of pregnant rats exposed to TCDD in utero and during lactation
(Heimler et al. 1998a). The anovulatory effect of PCDDs was observed in
gonadotropin-primed hypophysectomized rats (Gao et al. 1999; Petroff et
al. 2000; Roby 2001), and direct application of TCDD to the ovary blocked
ovulation as well (Petroff et al. 2000). Thus, PCDDs have direct effects on
the ovulatory follicle that are sufficient to block ovulation.
The rat ovary expressed AHR mRNA (Son et al. 1999), and macaque
granulosa cells also expressed AHR mRNA which was increased by human
chorionic gonadotropin (Chaffin et al. 1999). p-Naphthoflavone
(Bhattacharyya et al. 1995) and TCDD (Son et al. 1999) also increased
ovarian Cytochrome P4501A1 protein (CYP1A1) mRNA in rats. The direct
effects of PCDDs on ovarian steroid production are less clear, despite consis
tent blockade of ovulation after systemic and local ovarian exposure to
PCDDs. In immature gonadotropin-primed female rats, pretreatment with
PCDDs increased serum estradiol during the preovulatory period and re
duced serum progesterone concentrations consistent with blockage of ovula
tion and reduced luteinization (Gao et al. 1999, 2000b). In addition, in the
immature rat model, PCDDs blocked the surges of follicle-stimulating hor
mone (FSH) and lutenizing hormone (LH) in sera on expected proestrus (Li
et al. 1995b; Gao et al. 1999, 2000b). Collectively, these results indicate that
the adverse effects of PCDDs may be due to effects on gonadotropin release
as well as to direct effects on the ovary (Son et al. 1999; Petroff et al. 2000;
Roby 2001). In CD-1 mice and avian species, PCDDs did not alter serum
concentrations of estradiol (DeVito et al. 1992; Janz and Bellward, 1996).
In vitro models have been used to assess the effects of PCDDs on
ovarian steroidogenesis. PCDDs decreased cellular uptake of glucose and
reduced protein kinase A activity and secretion of progesterone and estra
diol in human granulosa cells (Enan et al. 1996a,b). However, another
study reported an initial inhibition of estradiol in human luteinized granu
losa cells that was followed by increased estradiol accumulation at 36 and
48 hours (Heimler et al. 1998b). A decrease in aromatase activity and
reduced messenger ribonucleic acids (mRNAs) for P450ssc and P450arom
in FSH-stimulated rat granulosa cells exposed to PCDDs has been reported
(Dasmahapatra et al. 2000). In contrast, PCDDs failed to alter progester
one, androstenedione, or estradiol secretion in in vitro cultures of whole
ovarian dispersates, granulosa cells, or thecal-interstitial cells derived from
immature rats (Son et al. 1999) Although, this lack of in vitro action is also
seen in immune cells in vitro.
One target of PCDDs may be alterations in follicular proteolysis and
tissue remodeling during the periovulatory period as ovulation is blocked
after acute exposure to TCDD, other dioxins, and DLCs (Gao et al. 1999,
2000b; Petroff et al. 2001). Potential mechanisms blocking degradation of
the follicular wall may involve modulation of steroid action since decreased
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expression of ovarian cyclooxygenase-2 (COX-2) and AHR coincide with
increased plasminogen activator inhibitor-1 (PAI-1) and tissue plasminogen
activator (PA) (Mizuyachi et al. 2002). Because PA participates in ovulation
in the rat (Tsafriri 1995), TCDDs may increase PAI-1, reduce overall PA
activity, and block ovulation. It is well known that PA activity increases
after the ovulatory surges of LH and FSH as a result of increased granulosal
cell prostaglandin secretion, a process dependent on COX-2 (Richards et al.
1987). COX-2 has TCDD response elements. Thus, TCDD, other dioxins,
and DLCs may block ovulation by inhibiting granulosal prostaglandin se
cretion, reducing COX-2 in the preovulatory follicle, before reducing PA
activity after an increase in ovarian PAI-1.
TCDD reduces expression of the progesterone receptor (PR), and PR
null mice do not ovulate (Lyndon et al. 1996). TCDD is well known to
inhibit estradiol-induced PR in the breast cancer cell line MCF-7 through
an AHR-mediated mechanism (Harper et al. 1994). However, within 24
hours after administration of TCDD to immature rats, estrogen receptor
(ER)a, ERp, and PR were unaffected in the ovaries (Mizuyachi et al. 2002).
Thus, the role of the PR in the anovulatory effects of PCDDs is unresolved.
Effects of PCDDs on the Hypothalamus and Pituitary Gland
TCDD, other dioxins, and DLCs reduce pituitary secretion of LH and
FSH at the time of the LH and FSH surges but premature surges of LH and
FSH have been reported in immature rats (Gao et al. 1999; 2000a,b). In the
Han Wistar rat that is resistant to TCDD, (50 pg/kg) caused atrophy of the
pituitary with little to no loss of weight and no mortality (Pohjanvirta et al.
1993). However, exposure of fetal (in utero) or neonatal (via mother’s
milk) mice to TCDD reduced pituitary weights of male offspring (Theobald
and Peterson 1997).
LH synthesis in the pituitary is controlled by gonadotropin-releasing
hormone (GnRH) and gonadal steroids feed back negatively to reduce
secretion. LH and FSH secretion were altered in gonadotropin-primed fe
male rats pretreated with TCDD, other dioxins, and DLCs (Li et al. 1995b;
Gao et al. 1999, 2000a,b). TCDD-treated animals had reduced gonadotro
pin secretion during the preovulatory period compared with controls. Cul
ture of pituitary halves with TCDD dose-dependently reduced LH secre
tion, but no effect of TCDD was observed in primary pituitary cell cultures
(Li et al. 1997).
Preovulatory increases in estradiol are required through a positive
feedback mechanism for induction of the LH and FSH surges on proestrus.
TCDD has antiestrogenic effects and inhibits ovulation through blockage
of the LH and FSH surges. However, serum concentrations of estradiol in
intact control and TCDD-treated rats are similar during the preovulatory
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period, indicating the possibility that the lack of estradiol action was causal
in blocking the surges (Li et al. 1995b; Gao et al. 1999, 2000a,b). This
appeared to be the case, as a long-acting exogenous estradiol administered
during TCDD treatment overcame the blockade on ovulation and restored
the LH and FSH surges (Gao et al. 2001).
Exogenous GnRH also overcame the inhibitory effects of TCDD on
ovulation by restoring the LH and FSH surges in the immature gonadotro
pin rat model (Gao et al. 2000a). Controls exhibited normal LH and FSH
surges, whereas such surges diminished in rats treated with TCDD. GnRH
treatment increased secretion of LH and FSH to surge levels in TCDDtreated rats and partially restored ovulation. Those data indicate that GnRH
secretion may have been reduced by TCDD. The failure of the gonadotro
pin surges to completely restore ovulation in rats receiving TCDD and
GnRH indicates the possibility that adverse direct effects of TCDD on the
ovaries may have reduced the number of ovulations.
Effects of TCDD on the Cardiovascular and Pulmonary Systems
Since the publication of EPA’s draft Reassessment, a substantial body
of literature has emerged concerning the effects of TCDD on heart and
vascular development. The developing vascular system appears to be a
target very sensitive to TCDD in vertebrate embryos. Much of the work in
this area has been performed in zebrafish (Danio rerio) embryos, a model
that has the advantage over mammalian and avian models of allowing for
direct visual observation of many developing organ systems, including the
heart and associated vasculature. Studies have also been performed in avian
and rodent models.
Several studies have indicated a fundamental role for the AHR system
in vascular development and hence a theoretical basis for the TCDD sensi
tivity of vascular development. Lahvis et al. (2000) generated A hr/- mice
that displayed reduced liver size. Developing mice exhibited altered vascu
lar architecture, including massive portosystemic shunting due to a patent
ductus venosus, resulting in reduced blood flow to the liver and hence
reduced hepatocyte size and liver mass. This failure of the ductus venosus to
close in Ahr-/- mice was subsequently associated with major hepatic veins
failing to decrease in size, as observed in wild-type mice, which may result
in increased blood pressure or a failure in vasoconstriction (Lahvis et al.
2005). Walisser et al. (2004b) observed that mice engineered to contain a
hypomorphic Arnt allele (underexpressing ARNT, the AHR nuclear
translocator protein) demonstrated the same vascular phenotype and were
resistant to TCDD toxicity versus wild-type mice. Together with the AHR
studies, this indicated essential roles for ARNT and for AHR-ARNT dimer
ization for both the purported developmental and TCDD toxicity roles of
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the AHR pathway. TCDD exposure during a specific time frame of embry
onic development rescued vascular development in both Ahr and Arnt
hypomorphs, indicating the requirement for activation of the AHR-ARNT
heterodimer for normal vascular development (Walisser et al. 2004a).
Studies with fish models, particularly zebrafish, have demonstrated the
sensitivity of the cardiovascular system, including cardiomyocytes, to TCDD
during embryonic development (Antkiewicz et al. 2005). Studies with mor
pho-lino antisense oligonucleotides to knock down expression of specific
genes in the zebrafish embryo have supported the key role of AHR in the
developmental effects of TCDD. Knockdowns of AHR2 prevented TCDDinduced pericardial edema, trunk circulation failure, and anemia in devel
oping zebrafish (Prasch et al. 2003; Dong et al. 2004). (Due to gene dupli
cation, zebrafish have two AHRs, AHR1 and AHR2; TCDD-mediated
effects are associated with binding to AHR2, and not to AHR1.) In these
studies, the AHR2 morpholinos were highly effective at blocking TCDDinduced cytochrome P4501A protein (CYP1A) expression in the vascular
endothelium. Carney et al. (2004) showed that, whereas an AHR2
morpholino protected zebrafish from TCDD-mediated effects of reduced
blood flow to trunk segments and pericardial edema, the CYP1A morphlino
did not provide protection against TCDD toxicity in contrast to the find
ings of Teraoka et al. (2003). Collectively, these studies demonstrate that
these developmental effects of TCDD are AHR2 mediated in zebrafish, but
the role of CYP1A remains unresolved. TCDD has also been demonstrated
to perturb cardiovascular development in the chicken embryo (Sommer et
al. 2005) and in maternally exposed fetal mice (Thackaberry et al. 2005a,b).
In addition, cardiovascular function is compromised in Ahr-' mice (Lund et
al. 2005; Vasquez et al. 2003).
These studies addressing the effects of TCDD on cardiovascular devel
opment were not performed with the objectives of quantitative risk assess
ment in mind. However, given the sensitivity of this end point at a very
sensitive lifestage, EPA is encouraged to consider these and related studies
identifying adverse effects of TCDD on cardiovascular development and
function in its risk assessment for noncancer end points.
Human Data
The Reassessment extensively documents the known reproductive, de
velopmental, and ectodermal consequences of TCDD exposure in a variety
of animal species (Part II, Chapter 5) and describes to a lesser extent various
other noncancer consequences, including hepatic, thyroid, and cardiovas
cular effects observed in animals other than humans (Part II, Chapter 7,
part B). In assessing the potential for related risks in humans, EPA makes
several critical assumptions.
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Assumption: Because dioxins are proven causes of reproductive, devel
opmental, and other abnormalities in various animal species, they may,
therefore, cause similar effects in humans. (Part III, p. 2-33, lines 3 to 5; p.
6-1, lines 21 to 22; p. 6-3, lines 14 to 16).
For reproductive, developmental, and ectodermal effects, this assump
tion is readily justified given the nature and extent of the animal data.
Further, the profiles of reported human reproductive, developmental, and
ectodermal effects after exposures to TCDD, other dioxins, and DLCs are
similar to the effects found in animals, thus lending overall general support
to the assumption. Similarities in developmental effects are most compel
ling at the highest levels of exposure such as those reported in the Yusho
and Yu-Cheng poisonings (Part II, pp. 5-15 to 5-16) because “ all four
manifestations of developmental toxicity (reduced viability, structural al
terations, growth retardation, and functional alterations) have been ob
served to some degree” (Part II, p. 5-97, lines 1 to 3).
Even so, the developmental effects are not entirely consistent and the
Reassessment appropriately notes that other than the mouse “ no other
species develops cleft palate except at maternal doses that are fetotoxic and
maternally toxic” (Part II, p. 5-19, lines 10 to 11) and that “ studies in
humans have not clearly identified an association between TCDD exposure
and structural malformations” (Part II, p. 5-19, lines 15 to 17). As dis
cussed below, the effects of low-level TCDD exposure on reported human
developmental effects are less compelling. Although the spectrum of re
ported human reproductive and hormonal abnormalities following TCDD
exposure is generally similar to that found in animals, the strengths of the
individual associations in studies thus far, are weak, and confidence in the
causal nature of these associations while suggestive is not yet compelling.
In reference to other noncancer consequences of TCDD exposure, the
assumption remains equally valid, although the animal evidence for other
noncancer end points, such as adverse effects on hepatic enzymes (Part II,
section 7.15.1.2.3), pancreatic islet function (Part II, section 7.15.2.1.2),
thyroid hormone dysregulation (Part II, section 5.2.3.6; Part III, section
2.2.1.3) , lipid abnormalities (Part III, section 2.2.6.3), and cardiopulmo
nary or circulatory disturbances (Part II, section 7.15.3.1; Part III, section
2.2.6.3) , is often more limited in scope.
Assumption: Humans are neither more nor less sensitive than animals
as far as the adverse effects of dioxins are concerned (Part II, p. 8-4, lines 6
to 27; Part III, p. 2-3, lines 28 and 29; p. 2-32, lines 14 to 16]. Given the
paucity of systematic in vivo human data, this assumption is the parsimoni
ous choice and also the most defensible based on in vitro data (Part II, pp.
8-4 to 8-5). Nevertheless, EPA acknowledges the uncertainty and impreci
sion of this assumption noting that (1) “ for most toxic effects produced by
dioxin, there is marked species variation” (Part II, p. 8-5, line 31); (2)
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human epidemiological studies are confounded by the fact that the unex
posed “cohorts contain measurable amounts of background exposure to
PCDDs, PCDFs, and dioxin-like PCBs” (Part II, p. 8-5, line 35; p. 8-6, line
1); (3) “many epidemiological studies are hampered by small sample size,
and in many cases the actual amounts of TCDD and related compounds in
human tissues were not examined” (Part II, p. 8-6, lines 2 and 3); (4) “it is
often difficult, if not impossible, to assess in humans the same endpoints
that might be determined in experimental animals” (Part II, p. 8-6, lines 4
and 5); and (5) “it is essentially impossible to determine the contribution of
TCCD-like versus non-TCDD-like congeners to fetal/neonatal toxicity”
(Part II, p. 5-15, lines 14 and 15) in the poisoning episodes where complex
mixtures containing a variety of toxicants were ingested accidentally (Part
III, p. 2-23, lines 32 to 35).
Assumption: Noncancer effects can occur at body burden levels in
animals equal to or less than body burdens calculated for tumor induction
in animals (Part III, p. 5-25, lines 28 and 29). Although critical to the
discussion of noncancer end points in humans, the strength of this assump
tion is unknown and the uncertainty is possibly large. The propagated
uncertainties leading to this assumption are highly dependent on the inher
ent uncertainties in the use of TEQs, the calculation of the historical body
burden, and the modeling of dose-response effects, as discussed in detail in
Chapters 2 and 3. Because of limited epidemiological evidence, further
uncertainty is introduced by the inability to demonstrate convincing asso
ciations and dose-response relationships between TCDD exposure and
noncancer end points in humans (Part III, p. 2-23, lines 20 to 22), as
discussed below.
Assumption: ED 01 is an acceptable departure point for calculating the
risks o f noncancer end points. As noted above, the limitations of this as
sumption are highly dependent on the inherent uncertainties in the use of
TEQs, the calculation of body burden, and the modeling of dose-response
effects, as discussed in detail in Chapters 3 and 5.
The EPA Reassessment does not adequately discuss the level of confi
dence that should be accorded results whose statistical significance is asso
ciated with wide uncertainty limits. Attention should also be directed to
addressing the potential biological significance of very small statistically
significant physiological or biochemical changes that remain well within
the normal range of variation and adaptation.
Furthermore, the EPA Reassessment continues to rely on the approach
that diverse human data collected across disparate studies of different types
and inherent strengths can be interpreted with confidence without applying
the more formalized tools of evidence-based medicine. Thus, the EPA Reas
sessment (as well as Institute of Medicine [IOM] committee report) relies
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
largely on committee-based, consensus evaluation of the available data
rather than on specifically commissioned, rigorous analyses constructed
according to established criteria that both formally evaluate the strengths of
the available evidence and integrate, by quantitative systematic review, the
data across available studies (Sackett et al. 2000; NCI 2002; CEBM 2005;
Guzelian et al. 2005).
On the whole, the potential for increased risk of noncancer end points
after exposure to TCDD at or near background levels is cautiously pre
sented in the Reassessment. However, the Reassessment explicitly charac
terizes TCDD as “ developmental, reproductive, immunological, endocrino
logical, and carcinogenic hazards” (Part III, p. 6-3, lines 10 and 11),
although the formal criteria for defining human hazard in the context of
these noncancer end points are not defined precisely in the Reassessment.
Further, although the Reassessment acknowledges that “ some have argued
that in the absence of better human data, deducing that a spectrum of
noncancer effects will occur in humans overstates the science” (Part III, p.
6-3, lines 33 and 34), the EPA position is that an inference of human effects
“is reasonable given the weight of evidence from available data” (Part III, p.
6-4, lines 1 and 2). Nonetheless, as EPA concedes, available human data
currently do not permit resolution of these divergent evaluations.
Human Reproductive and Developmental Outcomes
The available human reproductive and developmental studies available
at the time of the Reassessment draft are presented in detail, although a
number of the more recent follow-up studies are obviously not reported, as
mentioned below. EPA provides an overall conclusion that “ subtle effects,
such as the impacts on ... developmental outcomes ... or the changes in
circulating reproductive hormones in men exposed to TCDD, illustrate the
types of responses that support the finding of subtle yet arguably adverse
effects at or near background body burdens” (Part III, p. 6-2, lines 6 to 11).
The committee agrees that the results are subtle but disagrees that the
reported effects are truly clinically adverse, especially when confidence in
the observations is low and the reported changes could be non-significant at
the biological level and clinical outcome. In this context, the Reassessment
also notes that “there is no reason to expect, in general, that humans would
not be similarly affected [as animals] at some dose, and a growing body of
data supports this assumption. On the basis of the animal data, current
margins of exposure are lower than generally considered acceptable, espe
cially for more highly exposed human populations. The human database
supporting this concern for potential effects near background body burdens
is less certain” (Part III, p. 6-32, lines 19 to 23).
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165
Male Reproductive Hormones
The Reassessment’s description of the National Institute of Occupa
tional Safety and Health (NIOSH) study report (Egeland et al. 1994) show
ing a significant positive correlation of serum LH and FSH levels with
serum TCDD does not discuss the weak nature of this correlation, the wide
confidence intervals (CIs) around the regression, or the hormone values
within the normal range (Part II, section 7.13.5.1). Similarly, the text fur
ther describes a two to four times higher prevalence of low testosterone
levels among workers exposed to TCDD but does not report that the CIs
around the risk ratios at the higher serum TCDD levels not only are very
broad but also cross 1.0, indicating limited confidence in the significance of
the relationships (Part II, section 7.13.5.1). Nor does the EPA Reassessment
report that no dose-response effect was observed (odds ratio = 3.9 at lowest
range of TCCD levels and 2.1 at highest levels), although the 95% CIs of
the odds ratios themselves are so broad as to raise significant uncertainty
about whether there is indeed a dose response relationship indicated by
these studies (Part II, section 7.13.5.1).
Similarly, the Reassessment states that the Ranch Hand study (Roegner
et al. 1991) (Part II, section 7.13.5.1) reported lower serum testosterone
levels in Ranch Hand veterans with current serum TCDD levels exceeding
33.3 pg/g, although the reported difference (10.2 ng/dL) was “ statistically
nonsignificant” and unlikely to have a measurable physiological effect. The
EPA Reassessment also describes three additional negative studies (CDC
1988; Grubbs et al. 1995; Henriksen et al. 1997), concluding that “ the
human data offer some evidence of alterations in male reproductive hor
mone levels associated with substantial occupational exposure to 2,3,7,8TCDD” (Part II, p. 7B-38). Thus, although “ some evidence” has been
reported, the bulk of the reported evidence is either negative or uncertain to
a degree.
The Department of Defense (DOD) released the latest report of the
Ranch Hand study in 2005. The committee did not have the opportunity to
review the report in detail because its release coincided with the end of the
committee’s deliberations. However, the document reports that “the differ
ence in adjusted free testosterone means in Ranch Hand versus Compari
sons was 10.95 versus 10.47, respectively. The LH means for Ranch Hand
and Comparison officers were 4.49 mIU/mL versus 4.09 mIU/mL, respec
tively. Both were well within one standard deviation of normal-age matched
populations. No evidence of a dose-response effect was seen based on
categorized dioxin or 1987 dioxin levels” (DOD 2005, p. 18-156). The
report concludes that “ the association of dioxin with ... gonadal abnor
malities appeared weak at best and unlikely to be clinically significant”
(DOD 2005, p. 18-156) and “ associations between dioxin level and ...
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
gonadal hormone abnormalities were unlikely to be clinically important” at
these levels (DOD 2005, p. 21-8).
Female Reproductive System
In the Reassessment’s discussion of potential effects of TCDD exposure
on endometriosis, the more recent Seveso data (Eskenazi et al. 2002a) are
not included. Compared with women with TCDD concentrations of s20
ppt, the relative risk of endometriosis is 2.1 in women with TCDD concen
trations >100 ppt, but the 90% CI ranges from 0.5 to 8.0, indicating little
confidence in the true magnitude of the rate ratio or the significance of the
reported average relative risk of 2.1. One conclusion from these data might
be that women whose serum TCDD levels were >20 ppt had no more
endometriosis than those whose serum TCDD concentrations were s20
ppt. Another defensible conclusion might be that the study did not have the
power to come to any convincing conclusion on this issue. The authors of
the study, Eskenazi et al. (2002a), chose to describe their findings as a
“ doubled, nonsignificant risk for endometriosis among women with serum
TCDD levels of 100 ppt or higher, but no clear dose response.” Finally, in
a recent review of the nonhuman primate and the human data assessing the
relationship between TCDD exposure and endometriosis Guo concluded
that “there are no solid, credible data available at this moment to support
the hypothesis that dioxin exposure may lead to the development of en
dometriosis” (Guo 2004).
Data published within the past 2 years on effects of exposure to TCDD,
other dioxins, and DLCs on the menstrual cycle in women are obviously
not referenced in the 2000 Reassessment. Thus, data from the Seveso inci
dent surveying women who were exposed to TCDD postnatally, but while
they were prepubertal, found “no change in the risk of onset of menarche
with a 10-fold increase in TCDD,” and there was “ no evidence of a doseresponse trend” (Warner et al. 2004). Likewise, postmenarchal women
exposed in Seveso showed no association of TCDD exposure with men
strual cycle length, but, in women exposed before menarche who had a 10
fold increase in serum TCDD concentrations, the menstrual cycle was
lengthened by 0.93 day, although the 95% CI ranged from -0.01 to 1.86,
and the strength of the relationship between menstrual cycle length and
serum TCDD concentration shown in Figure 1A of the report is not con
vincing, with widely scattered data points (Eskenazi et al. 2002b). An ob
servational study of wives and sisters of Swedish fishermen found a 0.49day shorter menstrual cycle (95% CI 0.03 to 0.89) in those with a high
dietary exposure to polychlorinated organochlorine compounds, including
TCDD, but found no association with early life exposure (Axmon et al.
2004).
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167
The discussion on spontaneous abortions briefly mentions the study on
the NIOSH cohort as “ in press” (Part II, section 7.15.3.4.5). This study has
now appeared (Schnorr et al. 2001) and found no effect on the incidence of
spontaneous abortion or on the sex ratio of offspring. The authors con
cluded that the study provided “ additional evidence that paternal TCDD
exposure does not increase the risk of spontaneous abortions at levels
above those observed in the general population.” Likewise, recent data
from the Seveso cohort (Eskenazi et al. 2003) showed no association of
TCDD with spontaneous abortions.
Other recent relevant studies include birth-weight results reported for
the NIOSH cohort (Lawson et al. 2004) and the Seveso cohort (Eskenazi et.
al. 2003). The recent NIOSH report (Lawson et al. 2004) found that pater
nal TCDD exposure had no effect on birth weight for term infants, and a
“ somewhat protective” association of preterm delivery with paternal TCDD
(odds ratio = 0.8), although the 95% CI ranged from 0.6 to 1.1. There was
no obvious increase in birth defects, although the results were descriptive
only. The authors concluded that “ because the estimated TCDD concentra
tions in this population were much higher than in other studies, the results
indicate that TCDD is unlikely to increase the risk of low birth weight or
preterm delivery through a paternal mechanism.” The recent Seveso follow
up (Eskenazi et al. 2003) also showed no association of TCDD concentra
tion with offspring birth weight or with the birth of infants small for
gestational age. Finally, the Reassessment does not discuss the female Viet
nam veterans study reported by Kang et al. (2000), which also reported no
increase in spontaneous abortions, stillbirths, low-birth-weight infants, or
infant deaths among women veterans who had served in Vietnam (and
possibly exposed) compared with those who had served in the United States,
although there are no body burden measurements made.
The data on birth-weight effects were described adequately (Part II,
section 7.13.12.8), and the summary comment (Part II, section 7.13.12.9)
reflected appropriately the uncertainty of whether there were any birthweight effects of exposure to TCDD at the time of the Reassessment. How
ever, the likelihood of TCDD exposure having a measurable effect on birth
weight has been substantially reduced by the recently reported studies of
Kang et al. (2000), Eskenazi et al. (2003), and Lawson et al. (2004). To
reflect and appropriately weigh this new information, EPA should corre
spondingly modify the summary comment (Part II, section 7.13.12.9).
For the state of available information in 2000, the Reassessment de
scribes adequately the observed effects of TCDD exposure on offspring sex
ratio (Part II, sections 7.13.12.7, 7.15.3.4.8). As noted in the report, in
creased female births were observed after the Seveso accident (Mocarelli et
al. 1996, 2000). They were also observed in a study of offspring of Russian
pesticide producers exposed to TCDD (Ryan et al. 2002). However, the
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Schnorr study mentioned in the Reassessment as “in press” has now been
published (Schnorr et al. 2001) and found no effect of TCDD exposure on
sex ratio of offspring in the NIOSH cohort.
In the United States, both exposure to TCDD and the male-to-female
sex ratio at birth have declined since the early 1970s (Matthews and
Hamilton 2005). This parallel decline is opposite that postulated from
TCDD poisoning incidents. Thus, because sex ratios at birth not only un
dergo temporal trends but also show racial and nationality differences and
are affected by both maternal age and infant birth order (Matthews and
Hamilton 2005), EPA should also acknowledge the uncertainties inherent
in evaluating sex ratios at birth without properly controlling for the afore
mentioned variables. The committee recognizes, however, that the TCDD
exposure studies that showed altered gender ratios at birth have reported
ratio values that were greater than the changes that might normally be
expected to be caused by the variables mentioned above.
Childhood Growth and Postnatal Development
The Reassessment text describes appropriately the cited growth data
and conveys adequately the uncertainty of whether TCDD exposure has
effects on postnatal growth in humans (Part III, section 7.13.12.9). The
issue would not be further clarified by including the omitted Swedish fish
exposure study (Rylander et al. 1995) that reported diminished height, but
not weight, at age 18, because this report includes neither TCDD nor TEQ
data. Two of the longest-term studies of chlorinated toxicant effects on
growth published subsequently (Blanck et al. 2002; Gladen et al. 2000) deal
with PCB exposure and thus do not contribute to resolving the debate
about the effects of TCDD on childhood growth.
Similarly, the longest neurodevelopmental follow-up studies (Jacobson
and Jacobson 1996; Gray et al. 2005) are reports on PCB exposure and do
not directly contribute to the current TCDD issues since no TEQ is derived.
However, the ongoing Dutch follow-up study referenced repeatedly in the
Reassessment has now published its findings in 6.5-year-old children
(Vreugdenhil et al. 2002a). At that age, there were no cognitive or motor
differences between breast-fed infants (primarily postnatally exposed) and
formula-fed infants (primarily exposed in utero with background postnatal
exposure), including no overall differences in global cognitive index,
memory, or motor performance, except when children from “less optimal
homes” were analyzed separately. This observation suggested to the au
thors that less optimal home environment may allow the effects of TCDD
on neurodevelopment to become manifest more readily while, in more
optimal home environments, the additional beneficial environmental influ
ences overcome the detrimental effects of exposure to TCDD. This is clearly
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169
a hypothesis at this stage, given the availability of only this single study that
has addressed the issues. In an additional report, Vreugdenhill et al. (2002b)
also described decreased masculinized play in boys and increased masculin
ized play in girls at age 7.5 years. Although statistically significant, the
biological relevance of these conclusions remains uncertain given the wide
scatter of the data and the regression coefficients reported.
Cardiovascular and Pulmonary Systems
The Reassessment discusses in detail the available data on potential
human cardiopulmonary consequences of TCDD exposure highlighting the
difficulty of supporting firm conclusions about the presence of a relation
ship (Part II, sections 7.13.9, 7.13.9.1, 7.13.10). Recently, from the latest
data on Ranch Hands (DOD 2005), DOD concluded that “no consistent
evidence suggested that herbicides or dioxin were associated with ill effects
on respiratory health” (p. 21-9). On the other hand, “the presence of heart
disease was found to be higher among Ranch Hands than Comparisons in
enlisted flyers” (p. 21-6), and “ an increased percentage of Ranch Hands in
the high dioxin category were found to have abnormally high diastolic
blood pressure. Ranch Hands in both the low dioxin category and the low
and high dioxin categories combined were found to have a lower mean
systolic blood pressure. Similarly, a smaller percentage of Ranch Hands in
both the low dioxin category and the low and high dioxin categories com
bined had an abnormally high systolic blood pressure” (p. 21-6). However,
the report notes that “the prevalence of cardiovascular disease was not
increased in the Ranch Hand cohort. In only one analysis, that of diastolic
blood pressure noted above, was there any evidence of an increased risk
with increased body burden of dioxin” (p. 21-7).
OTHER NONCANCER END POINTS
Diabetes
The Reassessment (Part II, sections 7.13.6, 7.13.6.1, 7.15.2.1.2; Part
III, section 2.2.5) presents in detail the then available data on the relation
ship between TCDD exposure and the development of Type 2 diabetes.
This relationship was evaluated in greater depth by an IOM committee
(IOM 2000), which concluded that “there is limited/suggestive evidence of
an association between exposure to the herbicides used in Vietnam or the
contaminant dioxin and Type 2 diabetes.” This is an adequate statement of
the state of the science concerning this noncancer end point, and the com
mittee recommends that EPA revise the Reassessment to include the analy
sis provided by the IOM committee. The Reassessment should also incor-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
porate the data of the recent Ranch Hand study report (DOD 2005) show
ing that “mean fasting insulin and the risk of diabetes requiring insulin
control increased with initial dioxin. C-peptide and time to diabetes onset
decreased as initial dioxin increased.” The risk of diabetes requiring insulin
control was increased in the Ranch Hand high-dioxin category. An increase
in the risk of diabetes requiring oral hypoglycemic or insulin control was
observed as 1987 dioxin levels increased. Time to diabetes onset decreased
as 1987 dioxin levels increased. The risk of an abnormally high hemoglobin
A1c increased with 1987 dioxin levels. Some findings in the DOD (2005)
report appeared inconsistent with the results presented above, such as a
decrease in the risk of 2-hour postprandial urinary glucose abnormalities
with 1987 dioxin levels. The findings appear consistent with the previously
noted association between Type 2 diabetes and dioxin in Ranch Hand
veterans. Increased risks of diabetes requiring insulin control were found
with initial dioxin, in the high-dioxin category, and with 1987 dioxin lev
els. In contrast, “ associations between dioxin level and thyroid and gonadal
hormone abnormalities were unlikely to be clinically important” (DOD
2005, p. 21-8).
These data led to the conclusion that “ the association noted at previous
Air Force Health Study examinations between Type 2 diabetes mellitus and
dioxin persisted. A higher prevalence of diabetes, as well as severity, as
dioxin increased was evident, even after adjustment for such factors as age
and body mass index” (DOD 2005, p. 18-156).
Thyroid Function
The Reassessment acknowledges that despite the fact that “ many ef
fects of TCDD exposure in animals resemble signs of thyroid dysfunction
or significant alterations of thyroid-related hormones” (Part III, p. 2-41,
lines 32 and 33), the results of human studies “ are mostly equivocal” (Part
III, p. 2-42, line 1). The Reassessment reports that Pavuk et al. (2003)
showed “ elevated TSH [thyroid stimulating hormone] means among the
high TCDD exposure group in the 1985 and 1987 follow-ups, with an
increasing trend across the decade 1982-1992, but no association with the
occurrence of thyroid disease” (Part III, p. 2-42, lines 2 to 4). The discus
sion does not address the fact that the TSH differences, although statisti
cally significant, are quantitatively extremely small and well within the
normal range of circulating TSH levels. Further, limitations of the study are
not described, including 86 exclusions “ from the longitudinal analyses be
cause they had undergone thyroidectomy, or had endocrine cancer, or were
on thyroid medication” (Pavuk et al. 2003), the exclusion of one Ranch
Hand because of an extremely low TSH value that might have indicated the
earliest sign of hyperthyroidism and the fact that “ over the five examina-
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NONCANCER END POINTS
171
tions, three different radioimmunoassays were used to measure TSH”
(Pavuk et al. 2003). In addition the Reassessment does not report that no
significant differences “with regard to mean levels of total thyroxine (T4),
triiodothyronine (T3)% uptake, or free thyroxine index were observed at
any examination” (Pavuk et al. 2003). Recently, DOD (2005) reaffirmed
that “ as for a dioxin effect related to thyroid disease, the 2002 examination
data did not support such a relation.”
Further, while the most recently published Ranch Hand follow-up re
port (DOD 2005) found a difference in adjusted mean TSH levels (1.653
microunits/mL versus 1.557 microunits/mL) in Ranch Hands and Compari
sons, respectively, this difference was “not considered clinically significant
because a 1% difference is difficult to measure. The same was true from the
free T4 values in enlisted flyers (mean of 1.115 ng/dL in Ranch Hands
versus 1.054 ng/dL in the comparison groups). If a primary thyroid effect
were present, one would expect the TSH to move in the opposite direction
of the free T4, which was not seen in these data” (DOD 2005, p. 18-156).
The draft Reassessment also highlights the higher TSH values reported
in human infants by Pluim et al. (1993) and by Koopman-Esseboom et al.
(1994) (Part III, 2.2.6.2 and Part 11,7.15.2.2.2), but does not discuss the fact
that the TSH changes were very small and possibly not of physiological or
clinical significance. Follow-up of the Dutch children’s cohort has now
been carried out for more than a decade, and changes in thyroid status in
this cohort have not been reported, although it is unclear whether they were
in fact thoroughly assessed. The text in the Reassessment does include
lengthy hypothetical discussions (Part III, 2.2.1.3 and Part III, 2.2.6.2) of
plausible mechanisms for perturbations of human thyroid function, al
though there is limited human data to support or refute such mechanisms.
Teeth
Because ectodermal abnormalities are common findings in animal stud
ies, including nonhuman primates, as well as in the human Yusho and YuCheng exposures, the enamel hypomineralization found in 6- to 7-year old
Finnish children (Alaluusua et al. 1996, 1999) is highlighted twice (Part II,
section 7.13.12.6.1; Part III, section 2.2.2.1). Because the enamel mineral
ization scores were largely subjective, it is imperative that the observers
were blinded to the prior breast-feeding status of the children, but this issue
is not specifically mentioned in the publications. Additionally, the Reassess
ment highlights several other limitations of this study and, in the related
commentary (Part II, section 7.13.12.6.2) EPA acknowledges that “ the
presentation of the results is incomplete.”
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Chloracne
The Reassessment adequately summarizes the uniformly agreed upon
and well-documented association between TCDD exposure and the devel
opment of chloracne.
Elevated y-Glutamyl Transferase
The Reassessment adequately summarizes the data showing “ a consis
tent pattern of increased y-glutamyl transferase (GGT) levels among indi
viduals exposed to TCDD-contaminated chemical.” (Part III, p. 2-40, lines
23 and 24) and notes that “long-term pathological consequences of el
evated GGT have not been illustrated by excess mortality from liver disor
ders or cancer; or in excess morbidity in the available cross-sectional stud
ies” (Part III, p. 2-41, lines 23 and 24). The report also acknowledges that
“the consistency of the findings in a number of studies suggests that the
finding may reflect a true effect of exposure but for which the clinical
significance is unclear” (Part II, p. 7B-116, lines 20 to 22).
The most recent report of the Ranch Hand study found no relationship
between TCDD exposure and GGT (DOD 2005, pp. 13-51 to 13-57).
Lipid Levels
The Reassessment accurately notes that “neither adults nor children
from Seveso had lipid levels above the referent level” (Part II, p. 7B-118)
despite very high exposures and that “the most recent data suggest that
high exposure to 2,3,7,8-TCDD contaminated substances are not related
significantly to increased lipid concentrations, specifically total cholesterol
and triglycerides” (Part II, p. 7B-118). The Reassessment adds that “ slight
but chronic elevations in serum lipids may put an individual at increased
risk for disorders such as atherosclerosis and other conditions affecting the
vascular system” (Part II, p. 7B-118) and that “risk factors such as dietary
fat intake, familial hypercholesterolemia, alcohol consumption, and exer
cise” (Part II, p. 7B-118) were not considered in the Seveso study, even
though no effects of TCDD exposure were found.
The most recent Ranch Hand follow-up showed no relationship be
tween TCDD exposures and total high-density-lipoprotein cholesterol, al
though ranch hands had an increased percentage of individuals with in
creased triglyceride values (DOD 2005, pp. 13-78 to 13-103). This report
concluded that “ based on the analysis of triglycerides, a subtle relation
between dioxin and lipid metabolism cannot be excluded” (DOD 2005,
p. 13-218).
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NONCANCER END POINTS
173
The various remaining noncancer end points, for which there are fewer
data and even less suggestive evidence than the associations discussed above,
are adequately summarized throughout the Reassessment.
CONCLUSIONS AND RECOMMENDATIONS ON THE
REPRODUCTIVE, DEVELOPMENTAL, AND OTHER NONCANCER
END POINTS OF TCDD, OTHER DIOXINS, AND DLCS
• Embryonic and fetal development and female and male reproduction
are sensitive end points of toxicity from TCDD, other dioxins, and DLCs in
rodents because, as discussed earlier, responses occur at lower administered
doses than other end points. However, the sensitivity of these end points in
humans is less apparent.
• The fetal rodent is more sensitive than the adult rodent to adverse
effects of TCDD.
• The human equivalent intake (pg/kg-day) for some adverse effects
related to reproduction and development based on ED01 is not adequately
supported (Chapters 2, 3, and 4).
• In humans, there is a clear association of TCDD exposure with chloracne and available studies have shown suggestive associations of TCDD
exposure with Type 2 diabetes, but the latter data are not yet robust.
• In humans, the association of TCDD exposure with other reported,
detrimental noncancer effects has not been convincingly demonstrated. The
available studies have not yet shown clear associations among TCDD expo
sures and the risks of individual, clinically significant, noncancer end points.
• In reference to human disease risks, the overall conclusions about
noncancer risks due to TCDD exposure are, in general, cautiously stated,
and the uncertainty of suspected relationships is acknowledged. Nonethe
less, the limitations of specific human studies are not uniformly addressed,
and the broad 95% CIs accompanying some reported statistically signifi
cant effects are not discussed in the context of the uncertainty that these
broad confidence limits imply. Conversely, statistically insignificant effects
are sometimes highlighted.
• The divergent data across the diverse studies assessing human
noncancer end points have not been subjected to systematic review accord
ing to currently accepted approaches, including meta-analysis when appro
priate, nor has there been formal grading of the quality of the evidence
according to accepted principles currently applied in other areas of clinical
pathophysiology, including one report of the relationship of TCDD expo
sure to cancer end points (Crump et al. 2003).
• EPA should discuss how the ED used in the in utero and lactational
exposure rat models relates to human reproductive and developmental tox
icity and risk information, including TEFs and TEQs.
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• EPA should more clearly describe how and why ED01 values were
determined in animals and transferred to human equivalents for the various
noncancer end points and address risk estimate calculations using alterna
tive assumptions (e.g., ED05). Whereas ED01 is conceptually a viable POD,
the committee has concerns about how the ED01 is computed and whether
there are adequate data at the ED01 level to ensure an acceptable level of
confidence in the conclusions derived from using the ED01. The dynamic
range approach EPA used to compute ED01 for continuous response is
flawed in that the change of 1% total range may not identify any meaning
ful toxic effects, that 1% change may be well within random variation in
the absence of exposure, and that the use of total range is less sensitive than
use of a control range because total range can be much wider.
• EPA should provide a discussion of the dose-response effects of
TCDD, other dioxins, and DLCs on the adult female reproductive system
that result in endocrine disruption in animals. The impact of the doseresponse data provided in these studies on human risk assessment should be
presented.
• With respect to human noncancer end points, the Reassessment text
should be revised to include the relevant, more recent data and, when
appropriate as discussed above, to reflect study quality and data uncer
tainty of the studies referenced.
• For available human, clinical, noncancer end point data, EPA should
establish formal principles of and a formal mechanism for evidence-based
classification and systematic statistical review, including meta-analysis when
possible. The application of systematic review, followed by evidence-based
classification, leads to a more explicit statement of, and concrete apprecia
tion of, the level of certainty (and, correspondingly, of uncertainty) that can
be accorded the answers to specific questions in a particular field.
• When the mechanism is established, currently available and newly
available human clinical studies should be subject to such systematic review
and formal evidence-based assessment. The quality of the available evi
dence should be reported, and the strength or weakness of a presumptive
association should be classified according to currently accepted criteria for
levels of evidence. Animal studies have shown that TCDD can cause a
variety of noncancer effects. These studies support both the EPA position of
the plausibility of corresponding human effects and the need to devise
adequately designed investigations that will answer the questions in man.
• In making its final recommendations, EPA should incorporate and
integrate the relevant data from both human and animal studies, as appro
priate, according to the levels-of-evidence hierarchy devised.
• EPA is encouraged to review newly available studies on the effects of
TCDD on cardiovascular development in its risk assessment for noncancer
end points.
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7
R eview o f R isk C h a ra c te riz atio n
The Reassessment1 (Part III, Chapter 6) considers risk characterization
under a series of headings, many of which represent summaries of the
inputs to risk characterization instead of the output of risk characterization
and its formulation of advice to risk managers. For convenience, this chap
ter uses the same headings for the committee’s review of the Reassessment’s
risk characterizations before presenting its conclusions. Because Chapter 6
in the Reassessment summarizes data from previous chapters, many of the
committee’s comments here were raised in previous chapters of this report.
REVIEW
TCDD, Other Dioxins, and DLCs Can Produce a Wide Variety of Effects
in Animals and May Produce Many of the Same Effects in Humans
Reassessment (Part III, pp. 6-1 to 6-4)
This introductory text sets the scene by stating:
Effects will likely range from detection of biochemical changes at or near
background levels of exposure to detection of adverse effects with increas
ing severity as the body burdens increase above background levels. (Part
III, p. 6-1, lines 28-30)
^The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-Dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
175
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
Clearly adverse effects, including, perhaps, cancer, may not be detectable
until exposures contribute to body burdens that exceed current back
grounds by one or two orders of magnitude (10 to 100 times). (Part III, p.
6-2, lines 11-13)
The rationale for those statements is not clearly defined, although the
Reassessment states later that few clinically significant effects were detected
in the small number of human cohorts studied, nearly all members of which
had body burdens significantly above background levels.
The text considers species differences in sensitivity to 2,3,7,8-tetrachlorodibenzo-p-dioxin (TCDD, also referred to as dioxin), other dioxins,
and dioxin-like compounds (DLCs) and concludes that “humans most likely
fall in the middle rather than at either extreme of the range of sensitivity for
individual effects among animals” (p. 6-2, lines 25-26). A general compari
son across several species is not relevant to the focused issue of risk charac
terization. The comparisons of importance are those between humans and
the species and strains used in the specific studies that revealed adverse
effects at the lowest levels of exposure, the so-called critical effects (see
below). This general statement on interspecies differences detracts from a
critical and quantitative assessment of differences in sensitivity between
humans and the species used in the key toxicological studies for risk charac
terization.
Overall the committee considered this introductory section to be rea
sonable but unfocused.
TCDD, Other Dioxins, and DLCs are Structurally Related and
Elicit Their Effects Through a Common Mode of Action
Reassessment (Part III, pp. 6-4 to 6-5)
The text is uncontroversial and concludes that binding to the aromatic
hydrocarbon receptor (AHR) appears to be necessary but is not sufficient to
elicit the various TCDD-induced effects. The committee agrees with this
conclusion.
EPA and the International Scientific Community Have Adopted
Toxic Equivalency of TCDD, Other Dioxins, and DLCs as a
Prudent Scientific Policy
Reassessment (Part III, pp. 6-5 to 6-6)
The text summarizes the current situation and is uncontroversial. Obvi
ously, given the date of the Reassessment, the text has not considered
planned updates to toxic equivalency factor (TEF) values and whether these
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will be used in future risk assessments of TCDD, other dioxins and DLCs.
The committee’s recommendations related to TEFs appear in Chapter 3.
Complex Mixtures of TCDD, Other Dioxins, and DLCs
Are Highly Potent, Likely To Be Carcinogens
Reassessment (Part III, pp. 6-6 to 6-12)
The Reassessment states that “ because TCDD, other dioxins, and DLCs
always occur in the environment and in humans as complex mixtures of
individual congeners, it is appropriate that the characterization [likely car
cinogen] apply to the mixture” (p. 6-6, lines 19-21). Therefore, despite the
attention given by EPA and hence by this committee to consideration of
whether TCDD is “ carcinogenic to humans” or “likely to be carcinogenic
to humans,” the reality is that TCDD is always present as part of a mixture
of TCDD, dioxins, and DLCs, and, therefore, the practical hazard charac
terization of human exposure to TCDD is in effect considered by EPA
“likely to be carcinogenic.” In consequence, the focus on qualitative classi
fication of the nature of the cancer hazard by EPA has been a somewhat
futile exercise. The text then further discusses this issue and concludes that
the “ likely to be carcinogenic” classification could differ in strength, de
pending on the constituents in the mixture. Subsequent text reconfirms that
TCDD is classified by EPA as “ carcinogenic to humans” and outlines the
evidence used to reach this conclusion, including the presence of “ strong
and consistent” evidence from occupational epidemiological studies (a point
on which the committee does not agree; see Chapter 5). The committee
concluded that such detailed consideration of hazard classification was of
little value in a section on risk characterization, especially as the difference
between “carcinogenic” and “likely to be carcinogenic” would not have a
significant impact on the formulation of advice under risk characterization.
This section continues by presenting the upper bound of the cancer risk
estimate of 1 x 10_3 per pg of toxic equivalent quotient (TEQ)/kg of body
weight/day for both background intakes and incremental intakes above
background. The value is based on the range of cancer slope factors (CSFs)
developed from linear modeling of the occupational cohort data. The Reas
sessment states, “Evaluations of shape parameters...for biochemical effects
that can be hypothesized as key events in a generalized dioxin mode-ofaction model do not argue for significant departures from linearity below a
calculated ED01, extending down to at least one to two orders of magnitude
lower exposure” (p. 6-8, lines 31-33, to 6-9, lines 1-2). This sentence ap
pears to be critical to the risk characterization approach for cancer adopted
by EPA, but there is no scientific assessment of the strength of the available
evidence to support that statement, of the ability of the model-fitting meth-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
ods used by EPA to detect a departure from linearity, were one to exist; or
of the indications of nonlinearity in the dose-response relationships for
many noncancer effects also considered to be mediated via an interaction
with the AHR. The Reassessment attempts to explain the decision to use a
linear approach (Part III, p. 5-15, lines 27-29) by stating that “The linear
default is selected on the basis of the agent’s mode of action when the linear
model cannot be rejected and there is insufficient evidence to support an
assumption of nonlinearity.” Quantitative evidence of nonlinearity below
the point of departure (POD), the ED01 (effective dose), will never be avail
able because the POD is chosen to be at the bottom end of the available
dose-response data. As discussed in Chapter 5 of this report, EPA should
give greater weight to knowledge about the mode of action and its impact
on the shape of the dose-response relationship. The committee considers
that the absence of evidence that argues against linearity is not sufficient
justification for adopting linear extrapolation, even over a dose range of
one to two orders of magnitude or to the assumption of linearity through
zero, which would not normally be applied to receptor-mediated effects.
This view is supported by the results of the recent cancer bioassay (NTP
2004), which was not available to EPA at the time of the Reassessment but
which could have a major impact on the risk assessment approach adopted
by EPA.
The text compares the current estimate with previous EPA estimates
and uses previous evaluations with the same approach to support the out
come of the Reassessment. The difference between EPA’s evaluation and
that of the Food and Agricultural Organization of the United Nations
(FAO)/World Health Organization (WHO) Joint Expert Committee on
Food Additives (JEFCA) (which considered TCDD to be a nongenotoxic
carcinogen and an uncertainty factor approach to be adequate to account
for both cancer risk and noncancer effects) was thought to “reflect differ
ences in science policy” (p. 6-11, line 2). The Reassessment did not attempt
to explain why EPA has chosen to use an uncertainty factor approach for
the risk characterization of other nongenotoxic, receptor-mediated carcino
gens with a known mode of action (such as for thyroid carcinogens) but not
for TCDD. The Reassessment suggests that a margin-of-exposure (MOE)
approach should be adopted for both cancer and noncancer effects but does
not explore the implications of the estimated MOE for cancer or the ability
of the MOE approach to refine the advice for population groups.
The use of different methods for the risk characterization of end points
that result from the same basic underlying mode of action is scientifically
illogical, a conclusion that seems to be supported by EPA in an earlier part
of the Reassessment (Part III, p. 5-3, lines 18 to 28).
Although the Reassessment defines a slope factor and cancer risk esti
mate, it does not spell out clearly the health implications of emphasizing
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this approach for the U.S. population. The Reassessment states that the
slope factor has a “ public health-conservative nature” (p. 6-9, line 14), but
such risk management considerations should not be used to support an
approach to risk characterization or detract from selection of the most
appropriate, scientifically justifiable approach.
The text discusses the consistency of the present slope factor with
previous EPA evaluations and further discusses the issue of hazard classifi
cation, as if the decision to define a CSF affected the hazard classification
(or vice versa), which illustrates the lack of clarity and focus in this part of
the Reassessment.
The Reassessment recognizes that “ the shape of the dose-response curve
below the range of observation can be inferred only with uncertainty” (p. 6
12, lines 1 to 2), and therefore the Reassessment should have given equal
weight and critical evaluation to the derivation of a CSF and to the calcula
tion of the MOE with a discussion of the adequacy of the MOE of an
exposure in relation to the remaining uncertainties.
In future revisions of the Reassessment, aspects that should be dis
cussed for both approaches include the known mode of action, the ad
equacy of the occupational cohorts to represent the whole population, the
integration of data from the animal cancer bioassays (including the most
recent study) in relation to the spectrum of cancers detected, and the shape
of the dose-response relationship.
Use of a MOE Approach to Evaluate Risk for
Noncancer and Cancer End Points
Reassessment (Part III, pp. 6-12 to 6-18)
Despite the title of this section, there is no focused discussion in the
Reassessment of the MOE in relation to cancer or to each of the end points,
using exposure data relevant to that end point (Part III, Appendix A, Table
A-1). In addition, there is no discussion of the areas of uncertainty that
would need to be taken into account for each study and end point. For ex
ample, the MOE for cancer, and possibly for immune and neurodevelopmental
effects, would be based on epidemiological data, whereas MOEs for noncancer
effects would be based largely on data from animal studies. Other issues
that should be discussed in the interpretation of the MOE for each end
point are the relevance of the effect to the general population and to popu
lation groups and life stages and, most important, the clinical significance
of the magnitude of the effect detected at the ED01 (if this is retained as a
point of comparison on the dose-response relationship; see committee com
ments earlier in this report).
The Reassessment concludes that setting a reference dose (RfD) is not
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
appropriate because of the relatively high background levels of exposure
compared with effect levels (Reassessment, Part III, p. 6-14) and that “ any
RfD that the Agency would recommend using a traditional approach for
setting an RfD using uncertainty factors to account for limitations of knowl
edge is likely to be below—perhaps significantly below (by a factor of 10 or
more)—current background intakes and body burdens.” EPA thus con
cluded that setting an RfD would be of little value for evaluating possible
risk management options when the RfD has already been exceeded by
average background exposure. This issue is not resolved by simply replac
ing the RfD by an MOE without analyzing whether the estimated MOE is
adequate for that particular end point based on the data used to derive the
point of comparison on the dose-response relationship.
The magnitude of this apparent problem arises from two aspects of the
Reassessment: the use of ED 0 1 as the point of comparison with exposure for
continuous variables, which in many cases is two orders of magnitude
below the lowest-observed-adverse-effect level (LOAEL) (Reassessment,
Appendix A, Table A-1), and the use of the usual default uncertainty fac
tors despite the wealth of data available on TCDD. The issue of the deriva
tion and suitability of the ED01 for continuous variables was discussed in
Chapter 2 of this report. EPA has not justified its use for risk assessment
and its replacement of traditional measures, such as the no-observed-ad
verse-effect level (NOAEL), LOAEL, or BM D10 (benchmark dose) or
BMD05, as a point for establishing an RfD or for comparison with human
exposures by calculation of MOEs. Selection of appropriate uncertainty
factors is discussed further below.
An additional problem identified by the Reassessment is that “ the cal
culation of an RfD (with its traditional focus on a single “critical” effect)
distracts from the large array of effects associated with similar body bur
dens of dioxin” (Part III, p. 6-14, lines 20-22). This statement appears to
contradict EPA’s well-established approach of focusing on the critical effect
as a basis for setting health protective values. The problem applies to some
degree to many other chemicals that show multiple effects over a narrow
dose range and does not invalidate the selection of a “ critical effect” from
the TCDD database, which would lead to a more focused discussion and
risk characterization. A critical effect of postnatal reproductive changes
after in utero exposure was identified from the available dose-response data
by the European Scientific Committee on Food (SCF 2000, 2001) and by
JECFA (2002). These bodies concluded that developing a health-based guid
ance value (equivalent to an RfD) from reproductive effects in rats after in
utero exposure would also cover the risks of other effects (including cancer)
detected at slightly higher body burdens. In principle, the case of TCDD is
no different from that of other contaminants that produce multiple adverse
effects. Because the exposures of a proportion of the U.S. population would
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be above any RfD, it would have been useful for EPA to define the nature
and magnitude of the risks at different levels of intake, the groups of the
population most at risk, and the major sources of exposure for any at-risk
groups. Alternatively, if MOEs were calculated for noncancer effects, then
the risk characterization should describe the nature of the adverse effect
and the uncertainties and variability inherent in both the BMD (ED) esti
mate and the relevant exposure estimate. It would have been useful if MOE
values had been calculated and discussed for different exposure scenarios.
The Reassessment discusses the approaches adopted by the SCF and
WHO (IPCS1998b) and by the Agency for Toxic Substances and Disease
Registry (ATSDR 1998) (Reassessment, Part III, pp. 6-16 to 6-18). The
more recent JECFA (2002) evaluation was considered with reference to the
date of the meeting in 2001. The Reassessment highlights three sources of
differences:
1. An initial focus on cancer or noncancer effects.
2. The use of intake or body burden.
3. The “ safety” or “ uncertainty” factors used.
The recent evaluations by SCF and JECFA used the approach pro
posed by the WHO International Programme on Chemical Safety (IPCS)
in which the usual 10-fold default uncertainty factors are subdivided into
toxicokinetic and toxicodynamic subfactors, which can be replaced when
suitable chemical-specific data are available (IPCS 1994, 1999, 2004;
WHO 2005). In this approach, the 10-fold interspecies factor is divided
into 4.0 for toxicokinetics and 2.5 for toxicodynamics, the product (10)
being used in the absence of chemical-specific data to replace either of the
default values; the 10-fold human variability factor is subdivided equally
into 3.2 for toxicokinetics and 3.2 for toxicodynamics. Subdividing the
10-fold default uncertainty factors was done by EPA in its recent evalua
tion of boron (EPA 2004b), although a slightly different split of the 10
fold interspecies factor was used. (This difference between EPA and WHO
would not have altered significantly the health-based guidance value for
TCDD, other dioxins, and DLCs that was derived by SCF and JECFA.)
The Reassessment considers briefly the rationale for the uncertainty
factors used in the recent SCF and JECFA evaluations. These evaluations
concluded that interspecies differences in toxicokinetics had been taken
into account by the use of body burden as the dose metric instead of the
external dose, and therefore this subfactor would become 1 instead of 4.0.
The Reassessment does not discuss the explanation of why SCF and JECFA
concluded that “no uncertainty factor needed to be applied for differences
in toxicodynamics between experimental animals and humans and for
interindividual variation [in toxicodynamics] among humans” (Reassess-
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
ment, Part III, p. 6-17, lines 21-23). The rationale given in the JECFA
monograph (JECFA 2002, p. 590) was that, in general, rats are more
sensitive than humans to the adverse effects of TCDD, and therefore the
interspecies factor might be less than 1, but that “ it cannot be excluded
that the most sensitive humans might be as sensitive to the adverse effects
of TCDD as rats were in the pivotal studies. Therefore, it was concluded
[by the JEFCA] that no safety factor in either direction need to be applied
for differences in toxicodynamics among humans.” In other words, any
possible variability in toxicodynamics among humans would be compen
sated for by the higher inherent sensitivity of the rat strains used in the
pivotal studies compared with average humans, and each of these
subfactors would become 1. Of the four aspects for which the usual 100
fold uncertainty factor is applied, the only one for which data was consid
ered to be inadequate related to human variability in toxicokinetics. SCF
and JECFA applied the default value of 3.2 for human variability in
toxicokinetics.
While the approach adopted by the SCF and JECFA is open to criti
cism because of its simplicity, the attempt to incorporate the wealth of
data on TCDD into the risk assessment process contrasts with EPA’s
assumption that default values would be used, and hence the RfD would
be below the current levels of exposure. The Reassessment (Part III, p. 6
18, lines 6-9) states, “ In particular, the focus on accounting for residual
toxicodynamic differences in cross-species scaling and interindividual vari
ability in the general population to account for sensitive individuals, in
cluding children” would suggest larger uncertainty factors than have been
proposed by these groups if EPA were to set an RfD. However, EPA does
not discuss how the usual uncertainty factors might be modified using the
TCDD database and does not give an analysis of the uncertainty factors
that it would use and justification for their use. The Reassessment does
not discuss whether or not the EPA considered how the uncertainty fac
tors or other aspects of risk characterization could be revised based on
probabilistic approaches.
The Reassessment does not evaluate critically the extent of species
differences in target organ sensitivity, especially in relation to the pivotal
studies and critical effects. Overall, there is inadequate discussion of the
relative affinities of the AHRs in rats and humans and of the possible
impact of polymorphisms in AHR and other sources of sensitivity differ
ences within humans. The Reassessment (Part III, p. 6-18, line 4-5) states,
“ Traditional approaches that might be applied by EPA or that have been
applied by ATSDR would likely require additional information to support
the choice or removal of uncertainty factors as performed by WHO, SCF
and JECFA.” However, there is no critical discussion of the limitations of
the available data that might be used to move away from the traditional
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uncertainty factors. The Reassessment does not give the rationale for EPA’s
decision not to replace the default uncertainty factors by chemical-specific
data, despite the enormity of the TCDD database.
The Reassessment (Part III, p. 6-18, lines 10-14) concludes that “ any
composite uncertainty factor greater than 10 applied to effect levels based
on body burden ... would result in TDI or MRLs below the current back
ground intakes. The use of uncertainty factors in the range of 30 to 100 or
more, as traditionally used by EPA, would result in values even further
below some current background body burdens.”
The Reassessment concludes the risk characterization section (Part III,
p. 6-34) by stating that the MOEs based on body burden are less than 1 for
enzyme induction in rats and mice and less than 4 for developmental effects
in rats and endometriosis in nonhuman primates. The reader is left to
compare those values with uncertainty factors in the range of 30 to 100,
which EPA would traditionally use, with no clear and concise guidance on
the interpretation of this information. However, these judgments are based
on the nontraditional use of ED01 in place of a BMD5, BMD10, NOAEL, or
LOAEL.
Children’s Risk from Exposure to TCDD, Other Dioxins, and DLCs May
Be Increased, but More Data Are Needed to Address This Issue
Reassessment (Part III, pp. 6-18 to 6-21)
The Reassessment highlights the greater susceptibility of in utero,
perinatal, and neonatal life stages on the basis of animal and human
epidemiological data. The Reassessment does not clarify the additional
data that would be required before an RfD could be established or before
definitive advice could be given about the adequacy or inadequacy of the
MOE for adverse effects detected in animal studies after in utero expo
sure. Following these general doubts about the possible heightened sus
ceptibility of neonates and children, the Reassessment comments on the
greater exposure of nursing infants and children but concludes that, be
cause the risk characterization is based on body burden, the short-term
intake levels will have little impact on risk compared with overall lifetime
exposure. The committee noted that EPA did not define the MOE for
these life stages and that, overall, this section raises concerns about hypo
thetical, additional, undefined susceptibility while allaying concerns about
the considerably greater exposures of infants and children compared with
adults.
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Background Exposures to TCDD, Other Dioxins, and DLCs Need To Be
Considered When Evaluating Hazard and Risk
Reassessment (Part III, pp. 6-21 to 6-23)
This section of the Reassessment provides a summary of the extent of
background exposures but does not adequately integrate the information
into an MOE or an estimate of population cancer risk using the slope
factor.
Evaluating the Exposure of “ Special” Populations and Developmental
Stages Is Critical to Risk Characterization
Reassessment (Part III, pp. 6-23 to 6-25)
The Reassessment describes sources of variability in exposure and intake—for example, contaminated poultry feed, increased consumption of
fish, and occupational exposures. This section concludes that a high intake
would be about three times the population mean, but again there is no
quantification of the M OE or any attempt to link high-exposure groups to
specific end points (except for breast-feeding, which is covered in the fol
lowing section).
Breast-Feeding Infants Have Higher Intakes of TCDD, Other Dioxins,
and DLCs for a Short but Developmentally Important Part of Their
Lives but the Widely Recognized Benefits of Breast-Feeding
Outweigh the Risks
Reassessment (Part III, pp. 6-26 to 6-27)
The Reassessment reiterates the information on breast-feeding and
points out that the average daily intake by the infant over the first year of
suckling would be 87 times the adult daily intake. It correctly points out
that this would not result in an 87-fold higher body burden because of the
rapid increase in body weight and more rapid elimination. The Reassess
ment reiterates the advantages of breast-feeding in general and concludes
that reevaluation of TCDD, other dioxins, and DLCs does not alter the
previous advice, especially because the risk assessment is based on body
burden. While not disagreeing with the conclusion, the committee considers
the Reassessment to be superficial on this point. It does not support its
position with well-founded evidence, it does not consider the impact of
body composition (e.g., percent body fat) on distribution of the body bur
den in infants, and, most important it makes no attempt to compare the
intakes by infants with the doses producing adverse effects in the relevant
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animal studies (that is, on those involving in utero exposure and subsequent
assessment of developmental parameters in early life).
Many Dioxin Sources Have Been Identified and Emissions to the
Environment Have Been Reduced
Reassessment (Part III pp. 6-27 to 6-29)
This summary of previously presented information on sources and emis
sions is adequate. (The committee noted, however, that it is largely irrel
evant to this part of the Reassessment because it does not consider or
contribute to risk characterization.) See Chapter 4 for the committee’s
recommendations on sources and emissions.
TCDD, Other Dioxins, and DLCs Dioxins Are Widely Distributed in the
Environment at Low Concentrations Primarily as a Result of
Air Transport and Deposition
Reassessment (Part III, pp. 6-29 to 6-30)
This summary of previously presented information on sources and emis
sions is adequate. (The committee noted, however, that it is largely irrel
evant to this part of the Reassessment because it does not consider or
contribute to risk characterization.)
Environmental Levels, Emissions, and Human Exposures Have Declined
During Recent Decades
Reassessment (Part III, p. 6-30)
This summary of previously presented information on sources and emis
sions is adequate. (The committee noted, however, that it is largely irrel
evant to this part of the Reassessment because the data are not interpreted
in the context of risk characterization.)
Risk Characterization Summary Statement
Reassessment (Part III, pp. 6-30 to 6-34)
This section provides a reasonable summary of the preceding parts of
Chapter 6 of Part III.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
CONCLUSIONS AND RECOMMENDATIONS
The committee considered Chapter 6 of Part III of the Reassessment to
be the most important section, but in many ways it was the weakest and
least scientifically rigorous in its support of the decisions made.
EPA used linear extrapolation from the POD, the ED01, derived from the
cancer epidemiological studies to calculate a CSF. The resulting cancer risk
estimate of 1 x 10_3 per pg TEQ/kg of body weight per day for both back
ground intakes and incremental intakes above background was considered
by EPA to be the most appropriate approach. Using a linear extrapolation
approach in the Reassessment was one of the most critical decisions by EPA.
Use of this approach was not supported by a scientifically rigorous argument,
nor was there a balanced presentation of arguments using the same data to
support the calculation and interpretation of an MOE. EPA did not ad
equately discuss the risk management implications of the cancer risk esti
mate, which might be interpreted to indicate the need to reduce the current
exposure of the general population between 10-fold and 1,000-fold to limit
the calculated cancer risk between 1 in 10,000 and 1 in 1,000,000. Such a use
and interpretation of the slope factor would require EPA to consider the
validity of the linear model over many orders of magnitude.
The Reassessment stated that it used an MOE approach for noncancer
effects, but the discussion did not focus on the MOE values for different
adverse effects. An important improvement over past EPA practice was the
reliance in the Reassessment on an estimated ED (BMD) for noncancer
effects rather than on the traditional NOAEL and LOAEL as the POD. An
ED can be calculated mathematically from a fitted dose-response model
and is not limited to the experimental doses, thus representing a significant
advance in dose-response assessment. However, the computation of the
ED01 for continuous noncancer effects was critical, where the ED01 was not
the dose associated with a 1% incremental incidence of an adverse effect
but was the dose associated with a change in the mean response from the
background level that was 1% of the maximum possible total response
range. EPA made no attempt to present the biological significance of such
changes for each of the different continuous end points of studies subject to
dose-response modeling.
The adoption of such a novel approach gave extremely low general
MOE values and was used by EPA as justification for not analyzing and
interpreting the MOE values for each end point and also for not using the
massive TCDD database to identify an RfD.
Because EPA decided not to define an RfD, the Reassessment lacked
detailed risk characterization information—for example, the proportion of
the population with intakes above the RfD, detailed assessment of popula
tion groups, and contributions of the major food sources for those individu-
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REVIEW OF RISK CHARACTERIZATION
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als with high intakes. The lack of such a focus in the Reassessment results in
a diffuse risk characterization that is difficult to follow and that does not
provide clear guidance to risk managers.
The Reassessment should describe clearly the following aspects:
1. The effects seen at the lowest body burdens that are the primary
focus for any risk assessment—the “ critical effects.”
2. The modeling strategy used for each noncancer effect, paying par
ticular attention to the critical effects, and the selection of a point of com
parison based on the biological significance of the effect; if the ED01 is
retained, then the biological significance of the response should be defined
and the precision of the estimate given.
3. The precision and uncertainties associated with the body burden
estimates for the critical effects at the point of comparison, including the
use of total body burden rather than modeling steady-state concentrations
for the relevant tissue.
4. The committee encourages EPA to calculate RfDs as part of its effort
to develop appropriate margins of exposure for different end points and
risk scenarios, including the proportions of the general population and of
any identified groups that might be at increased risk (See Table A-1 in the
Reassessment, Part III Appendix, for the different effects; appropriate expo
sure information would need to be generated.) Interpretation of the calcu
lated values should take into consideration the uncertainties in the POD
values and intake estimates.
5. Consideration of individuals in susceptible life stages or groups (e.g.,
children, women of childbearing age, and nursing infants) who might re
quire an estimation of a separate MOE using specific exposure data.
6. Distributions that provide clear insights about the uncertainty in the
risk assessments, along with discussion about the key contributors to the
uncertainty.
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C o n c lu sio n s an d R e c o m m e n d a tio n s
CLASSIFICATION OF TCDD AS CARCINOGENIC TO HUMANS
In its charge, the committee was requested to comment specifically on
the U.S Environmental Protection Agency (EPA) conclusion that 2,3,7,8tetrachlorodibenzo-p-dioxin (TCDD, also referred to as dioxin) is best char
acterized as “carcinogenic to humans.” Both EPA and the International
Agency for Research on Cancer (IARC), an arm of the World Health Orga
nization (WHO), have established criteria for qualitatively classifying chemi
cals into various categories based on the weight of scientific evidence from
animal, human epidemiological, and mechanism or mode-of-action studies.
In 1997, an expert panel convened by IARC concluded that the weight of
scientific evidence for TCDD carcinogenicity in humans supported its clas
sification as a Class 1 carcinogen— “carcinogenic to humans.” In 1985,
EPA classified TCDD as a “probable human carcinogen” based on the data
available at the time, but in the latest Reassessment (2003 ),1 EPA con
cluded that TCDD was “ best characterized as ‘carcinogenic to humans.”
The National Toxicology Program (NTP 2000) also classified TCDD as
“known to be a human carcinogen.”
After reviewing EPA’s 2003 Reassessment and other scientific informa
tion and in light of EPA’s recently revised 2005 Guidelines for Carcinogen
1The Exposure and Human Health Reassessment of 2,3,7,8-Tetrachlorodibenzo-p-dioxin
(TCDD) and Related Compounds (EPA 2003a, Part I; 2003b, Part II; 2003c, Part III) is
collectively referred to as the Reassessment.
188
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CONCLUSIONS AND RECOMMENDATIONS
189
Risk Assessment (cancer guidelines), the committee concludes that the clas
sification of TCDD as “carcinogenic to humans”—a designation suggesting
the greatest degree of certainty about carcinogenicity—versus “ likely to be
carcinogenic to humans”—the next highest designation—is somewhat sub
jective and depends largely on the definition and interpretation of the crite
ria used for classification. The true weight of evidence lies on a continuum,
with no obvious point or “ bright line” that readily distinguishes between
those two categories.
Referring to the specific definitions in EPA’s 2005 cancer guidelines for
qualitative classification of chemical carcinogens, the NRC committee was
split on whether the evidence met all the criteria necessary for classification
of TCDD as “carcinogenic to humans,” although the committee unani
mously agreed on a classification of at least “likely to be carcinogenic to
humans.” The committee concludes that the weight of epidemiological evi
dence that TCDD is a human carcinogen is not strong, but the human data
available from occupational cohorts are consistent with a modest positive
association between relatively high body burdens of TCDD and increased
mortality from all cancers. Positive animal studies and mechanistic data
provide additional support for classification of TCDD as a human carcino
gen. The committee recommends that EPA summarize its rationale for
concluding that TCDD satisfies the criteria set out in its cancer guidelines
for designation as either “carcinogenic to humans” or “likely to be a hu
man carcinogen.”
If EPA continues to designate TCDD as “carcinogenic to humans”
under the new guidelines, it should explain whether this conclusion reflects
a finding that there is a strong association between TCDD exposure and
human cancer or between TCDD exposure and key precursor events of
TCDD’s mode of action (presumably aromatic hydrocarbon receptor [AHR]
binding). If its finding reflects the latter association, EPA should explain
why that end point (e.g., AHR binding) represents a “key precursor event.”
As noted above, the committee concludes that the distinction between
these two categories is based more on semantics than on science and recom
mends that EPA focus its energies and resources on more carefully delineat
ing the assumptions used in quantitative risk estimates for TCDD, other
dioxins, and dioxin-like compounds (DLCs) derived from human and ani
mal studies.
The committee agrees that other dioxins and DLCs are most appropri
ately classified as “likely to be carcinogenic to humans.” If EPA continues
to classify TCDD as “carcinogenic to humans,” more justification will be
required to explain why a mixture containing TCDD would not also meet
the classification of “carcinogenic to humans.”
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
USE OF LOW-DOSE LINEAR VERSUS THRESHOLD (NONLINEAR)
EXTRAPOLATION MODELS FOR QUANTITATIVE
CANCER RISK ESTIMATIONS
The committee unanimously agrees that the current weight of evidence
for TCDD, other dioxins, and DLCs carcinogenicity favors the use of non
linear methods for extrapolation below the point of departure (POD) of
mathematically modeled human or animal data. However, the committee
recognizes that it is not scientifically possible to exclude totally a linear
response at doses below the POD, so it recommends that EPA provide risk
estimates using both approaches and describe their scientific strengths and
weaknesses to inform risk managers of the importance of choosing a linear
vs. nonlinear method of extrapolation. To the extent that EPA favors using
default assumptions for regulating dioxin as though it were a linear car
cinogen, such a conclusion should be made as part of risk management.
EPA should strictly adhere to the distinction between risk assessment, which
is a scientific activity, and risk management, which takes into account other
factors.
USE OF THE 1% RESPONSE LEVEL AS A POINT OF DEPARTURE
FOR LOW-DOSE RISK ESTIMATION
The Reassessment adopts the benchmark dose (BMD) method to re
place the traditional, less quantitative approach of using no-observed-ad
verse-effect level (NOAEL) and lowest-observed-adverse-effect level
(LOAEL) to characterize noncancer effects. A BMD (or an effective dose)
can be calculated mathematically from a fitted dose-response model and is
not limited to the experimental doses. The BMD method is a significant
advance in dose-response modeling, and EPA’s use of BMD is highly com
mendable. However, the determination of an ED at the 1% response level
(ED01) for continuous noncancer effects is not without significant limita
tion. Specifically, the ED01 was the dose associated with a change in mean
response away from the background level by 1% of the maximum possible
total response range. For some noncancer end points, the significance of
such a change may be difficult to identify both clinically and statistically
and can be well within the variation of the control data. The biological
significance of this magnitude of change represented by the ED01 values for
different continuous noncancer end points should be evaluated.
The adoption of such a novel approach gave extremely low margin-ofexposure (MOE) values compared with background exposures and was
used by EPA as justification for not analyzing and interpreting the MOE
values for each end point and also for not using the massive dioxin database
to set a reference dose (RfD).
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CONCLUSIONS AND RECOMMENDATIONS
191
In its evaluation of the ED01 used for cancer risk assessment, the com
mittee concluded that EPA had not adequately justified use of the 1%
response level as the POD for the analysis of either the epidemiological or
the animal bioassay data. Even though it is necessary to demonstrate that
the POD is within the range of the observed data, that by itself is not
sufficient to justify use of the ED01. Other conditions, such as demonstrat
ing that the POD is relatively insensitive to functional form (as noted in the
cancer guidelines [EPA 2005a]), must also be satisfied. EPA should ac
knowledge the larger extrapolation from justifiable POD values down to
environmentally relevant doses that would be necessitated by use of a higher
response-level POD.
With regard to EPA’s review of the animal cancer bioassay data, the
committee recommends that EPA establish clear criteria for the inclusion of
different data sets. The reliance on data for one site from one gender of one
species, as reported by a single study, does not adequately represent the full
range of data available. The committee recommends that EPA consider the
full range of data, including the new NTP animal bioassay studies on
TCDD, for quantitative dose-response assessment.
For the various noncancer end points, EPA should describe more clearly
how and why the ED01 values were determined in animals and translated to
human equivalents. At the least, the risk assessment should provide more
apparent and parallel calculations using a 5% response level as the POD to
demonstrate the impact that this assumption might have on both the point
estimates of risk at low doses and the range of uncertainty surrounding that
point estimate. This recommendation applies to extrapolation for cancer
risk estimates, for which an ED01 was also used, as well as for noncancer
risk estimates.
Although the committee commends EPA’s extensive efforts on doseresponse modeling of a large number of data sets, particularly those of
noncancer end points, it is concerned that selection of the final model for
computing POD values was not based on a statistical assessment of model
goodness of fit, particularly at low doses. An inadequately fitted model
could substantially alter extrapolation to low doses and therefore is a source
of error that can result in significant uncertainty. The committee recom
mends using statistically rigorous methods for assessing model fit to control
and reduce this source of uncertainty related to selection of a POD. Al
though the committee encourages EPA to use thorough statistical analyses
of data, it also cautions that “ statistical significance” does not always
equate with “ biological significance,” and thus sound scientific judgment,
in addition to statistical analysis, is a critical element of data interpretation.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
CHARACTERIZATION OF UNCERTAINTY FOR RISK ESTIMATES
Overall, the committee found that the Reassessment qualitatively ad
dressed many sources of uncertainty and variability but that it failed quan
titatively to sufficiently address uncertainty and variability that resulted
from the numerous decisions EPA made in deriving point estimates of risk
in the comprehensive risk assessment. In contrast, EPA used concerns about
uncertainties and uncertainty factors as part of the justification for not
setting an RfD for noncancer effects (see Chapter 7 for further discussion).
The committee recommends that EPA provide statistical estimates of
the upper-, lower-, and central-bound risk estimates for all quantitative risk
estimates. In light of the magnitude of this uncertainty, the committee
considers identification of a point estimate value for the dioxin cancer slope
factor (CSF), even a point estimate designated as an upper bound, to confer
a false sense of precision. EPA should identify the sources of uncertainty
and quantitatively characterize their impact on the probability distribution
that describes the set of plausible CSF values. If necessary, EPA should
acknowledge that the information available is not sufficient to support
designation of a meaningful point estimate.
The committee recommends that EPA more completely characterize
uncertainty associated with cancer risk estimates inferred from the epide
miological data (1) by taking into account the full range of ED values
statistically consistent with the data (not only the central and lower esti
mates); (2) by considering alternative PODs; (3) by considering biologically
plausible alternative dose-response functional forms consistent with the
data; and (4) by considering uncertainty associated with the half-life esti
mates of TCDD in humans for the purpose of back-extrapolating exposures
in occupational cohort studies.
The Reassessment did not provide details about the magnitudes of the
various uncertainties surrounding the decisions that EPA makes about dose
metrics (e.g., the impact of species differences in percentage body fat on the
steady-state concentrations present in nonadipose tissues). The committee
recommends that the Reassessment use simple physiologically based phar
macokinetic models to define and characterize the uncertainty of any differ
ences between humans and rodents in the relationship between total body
burden at steady state (as calculated from the intake, half-life, and
bioavailability) and tissue concentrations; EPA should modify the estimated
human equivalent intakes when necessary. While PBPK modeling may itself
introduce uncertainty, the process of building the PBPK model should help
to reduce the far greater uncertainty and likelihood of error that arises
when PBPK considerations are not included explicitly. Many opportunities
exist to further characterize sources of uncertainty and variability related to
the dose metric choices, and the committee recommends that EPA improve
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CONCLUSIONS AND RECOMMENDATIONS
193
the Reassessment by providing a clear evaluation of the impacts of possible
choices on the risk estimates.
The committee recommends that EPA make greater use of mechanistic
information to assess the biological plausibility of different mathematical
models, use more rigorous criteria (e.g., goodness-of-fit test) for selecting a
model for deriving a POD, and clearly identify the benchmark response
level of toxicological significance for noncancer end points.
USE OF TOXIC EQUIVALENCY FACTORS FOR RISK ESTIMATION
OF DLCS AND MIXTURES OF DLCS
Overall, even given the inherent uncertainties, the toxic equivalency
factor (TEF) method provides a reasonable, scientifically justifiable, and
widely accepted method to estimate the relative toxic potency of dioxins,
other than TCDD, and DLCs, relative to TCDD, on human and animal
health. However, the Reassessment should acknowledge the need for better
uncertainty analysis of the TEF values. The committee also supports a
previous recommendation from the EPA Science Advisory Board (SAB)
“that, as a follow up to the Reassessment, EPA should establish a task force
to build consensus probability density functions for the ... chemicals for
which TEFs have been established, or to examine related approaches such
as those based on fuzzy logic.”
USE OF BODY BURDEN AS THE PRIMARY DOSE METRIC FOR
CROSS-SPECIES EXTRAPOLATION
Although the committee agrees that use of body burden as the dose
metric is the most reasonable and pragmatic approach at the present time, a
number of uncertainties in using body burden to develop risk estimates should
be addressed. The magnitudes of the various uncertainties are not clearly
defined. The most significant impact is the species differences in percentage
body fat on the relationship between body burden and the concentrations
present in nonadipose tissues. An analysis of the impact of possible uncertain
ties in the dose metric on the final risk estimates would be informative.
It remains to be determined whether the current WHO TEFs, which
were developed to assess the relative toxic potency of a mixture to which an
organism is directly exposed by dietary intake, are appropriate for body
burden toxic equivalent quotient (TEQ) determinations, which are derived
from the concentrations of different congeners measured in body fat. If
body burdens are to be used as the dose metric, a separate set of body
burden TEFs should be developed and applied for this evaluation. Without
these corrected values, the overall TEQs estimated by use of intake TEFs
might be substantially in error.
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
EPA’S EXPOSURE ASSESSMENT OF TCDD, OTHER DIOXINS, AND
DLCS IN THE UNITED STATES
To assess total emissions of TCDD, other dioxins, and DLCs, EPA used
a “ bottom-up” approach, which attempted to identify all source categories
and then estimated the magnitude of emissions for each category. However,
a “ top-down” approach that attempts to account for the levels measured in
receptors (e.g., people, animals, and plants) could give rise to substantially
different information. Such alternative approaches are likely to give rise to
significantly different estimates of the historical levels of dioxin emissions.
Both approaches come with uncertainties, and EPA could benefit signifi
cantly from using them simultaneously to set plausible bounds on the his
torical and current trends in emissions.
Although beyond the scope of the review of the EPA Reassessment, the
committee noted that it would be useful for EPA to set up an active conge
ner-specific database of typical concentrations for the whole range of poly
chlorinated dibenzo-p-dioxins (PCDDs), polychlorinated dibenzofurans
(PCDFs), and dioxin-like polychlorinated biphenyls (PCBs) (included in the
WHO TEF list) present in food. This database should be based on a com
pendium of all available data that would be updated on a regular basis with
new data as they are published in the peer-reviewed literature. Such a
database should have clear requirements of data quality and traceability
(e.g., chemical analysis, representative and targeted sampling, representa
tive of consumer exposure, presentation of data, and handling and presen
tation of nondetects).
The committee suggests that in the future EPA define a strategy for
collecting samples and reanalyzing archived samples to answer a number of
remaining questions about exposure trends and to fill some important data
gaps.
EPA’S EVALUATION OF IMM UNOTOXICITY OF TCDD,
OTHER DIOXINS, AND DLCS
Present clinical findings are inconclusive about whether or in what way
TCDD, other dioxins, and DLCs are immunotoxic in humans, and EPA
acknowledges that human data are sparse. A series of studies from a Dutch
children’s cohort showed an association between prenatal exposure to DLCs
and changes in immune status. The effects were modest, and laboratory
values did not fall significantly outside the full range of normal. Some
clinically relevant adverse effects seen in this perinatal study are also seen at
higher levels of exposure, although these do not seem to persist. A number
of animal studies suggest that the developing immune system is especially
sensitive. In light of the large database showing that TCDD, other dioxins,
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CONCLUSIONS AND RECOMMENDATIONS
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and DLCs are immunotoxic in laboratory animal studies—together with
limited human data—EPA is prudent in concluding that these compounds
are likely to be human immunotoxicants in the absence of more definitive
human data.
However, EPA’s conclusion that TCDD and related compounds are
immunotoxic at “ some dose level” by itself is inadequate. At a minimum, a
section or paragraph should be added that discusses the immunotoxicology
of TCDD, other dioxins, and DLCs in the context of current AHR biology.
Likewise, some discussion should also be included on the strengths and
weaknesses of using genetically homogeneous inbred mice to characterize
immunotoxicological risk in the genetically variable human population. Ex
panding the discussion to include the above crucial points would provide
additional balance to Part III, Integrated Summary and Risk Characterization.
Additional comments and recommendations relating to the use of spe
cific data sets for risk assessment of the immunotoxic effects of TCDD,
other dioxins, and DLCs are provided in Chapter 6.
EPA’S EVALUATION OF REPRODUCTIVE AND DEVELOPMENTAL
TOXICITY OF TCDD, OTHER DIOXINS, AND DLCS
As clearly described in the Reassessment, embryonic and fetal develop
ment and reproductive effects are sensitive end points of TCDD toxicity in
rodents. It is clear that the fetal rodent is more sensitive than the adult
rodent to adverse effects of TCDD. Comparable human data are generally
lacking, and the sensitivity of humans to these end points is less apparent.
The committee recommends that EPA address more thoroughly how
the effective doses used in the animal pregnancy models relate to human
reproductive and developmental toxicity and risk information, including
TEFs and TEQs. For available human clinical data on reproductive and
developmental end points, EPA should establish formal principles of, and a
formal mechanism for, evidence-based classification and systematic statisti
cal review, including meta-analysis when possible.
Finally, EPA should discuss the dose-response effects of TCDD, other
dioxins, and DLCs on the adult female reproductive system that result in
endocrine disruption in animals. Based on the dose-response data provided
in these studies, the impact on human risk assessment should be presented.
EPA’S EVALUATION OF OTHER TOXIC END POINTS
In general, the committee determined that the Reassessment adequately
addressed the available data on whether exposures to TCDD, other diox
ins, and DLCs are likely to be significant risk factors for other toxic end
points, such as chloracne, thyroid function, liver function, diabetes, lipid
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
disorders, and cardiovascular diseases. In humans, the relationship between
dioxin exposure and risk of individual, clinically significant, noncancer end
points remains uncertain, except for chloracne.
The overall conclusions in the Reassessment about noncancer risks due
to exposure to TCDD, other dioxins, and DLCs are, in general, cautiously
stated, and the uncertainty of suspected relationships is acknowledged.
Nonetheless, the limitations of individual human studies are not uniformly
addressed, and the broad 95% confidence intervals accompanying some
reported statistically significant effects are not discussed in the context of
the uncertainty that these broad confidence limits imply. Conversely, statis
tically nonsignificant effects are sometimes highlighted, presenting an im
plied potential for unobserved detrimental effects without a firm evidence
base. For available human clinical data for other noncancer end points,
EPA should establish formal principles of, and a formal mechanism for,
evidence-based classification and systematic statistical review, including
meta-analysis when possible.
With respect to human noncancer end points, the committee deter
mined that the Reassessment text should be revised to include the relevant,
more recent data and, when appropriate, the quality and data uncertainty
of the studies referenced. When the mechanism is established, currently
available and newly available human clinical studies should be subject to
such systematic review and formal evidence-based assessment. The quality
of the available evidence should be reported, and the strength or weakness
of a presumptive association should be classified according to currently
accepted criteria for levels of evidence.
New studies on effects of TCDD on the developing vascular system
suggest that this system could be a highly sensitive target and suggest that
this area be identified as an important data gap in the understanding of the
potential adverse effects of TCDD, other dioxins, and DLCs.
EPA’S OVERALL APPROACH TO RISK CHARACTERIZATION
As discussed above, EPA used linear extrapolation from the POD (the
ED01) derived from the cancer epidemiological studies and animal bioas
says to calculate a CSF. The selection of the default linear extrapolation
approach was one of the most critical decisions in the Reassessment, but the
decision to use this approach was not supported by a scientifically rigorous
argument, nor was there a balanced presentation of arguments that would
support the calculation and interpretation of a MOE with the same data.
The committee determined that a balanced presentation of available data
could support the use of a nonlinear model consistent with a receptormediated mode of action with subsequent calculations and interpretation of
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CONCLUSIONS AND RECOMMENDATIONS
197
MOE values. (For cancer risk assessment, the threshold approach should be
used in addition to the linear approach.)
Because EPA decided not to define an RfD, the Reassessment lacked
detailed risk characterization information—for example, the proportion of
the population with intakes above the RfD; detailed assessment of popula
tion groups, such as those with occupational exposures; and the contribu
tions of major food sources and other environmental sources for those
people with high intakes. The lack of such a focus in the Reassessment
results in a diffuse risk characterization that is difficult to follow and that
does not provide clear advice to risk managers.
The committee points out particular areas that could be improved in
Part III of the Reassessment. In particular, the risk characterization chapter
of the Reassessment should describe concisely and clearly the following
aspects.
1. The effects seen at the lowest body burdens that are the primary
focus for any risk assessment—the “ critical effects.”
2. The modeling strategy used for each noncancer effect modeled, pay
ing particular attention to the critical effects, and the selection of a point of
comparison based on the biological significance of the effect; if the ED01 is
retained, then the biological significance of the response should be defined
and the precision of the estimate given.
3. The precision and uncertainties associated with the body burden
estimates for the critical effects at the point of comparison, including the
use of total body burden rather than modeling steady-state concentrations
for the relevant tissue.
4. The committee encourages EPA to calculate RfDs as part of its effort
to develop appropriate margins of exposure for different end points and
risk scenarios, including the proportions of the general population and of
any identified groups that might be at increased risk (See Table A-1 in the
Reassessment, Part III Appendix, for the different effects; appropriate expo
sure information would need to be generated.) Interpretation of the calcu
lated values should take into consideration the uncertainties in the POD
values and intake estimates.
5. Consideration of individuals in susceptible life stages or groups (e.g.,
children, women of childbearing age, and nursing infants) who might re
quire estimation of a separate MOE using specific exposure data.
6. Distributions that provide clear insights about the uncertainty in the
risk assessments, along with discussion of the key contributors to the uncer
tainty.
The committee recognizes that it will require a substantial amount of
effort by EPA to incorporate all the changes recommended in this review;
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HEALTH RISKS FROM DIOXIN AND RELATED COMPOUNDS
however, it does not advocate a substantial expansion in the length of the
Reassessment. Rather, the committee encourages EPA to address the major
concerns raised in this review and to finalize the current Reassessment as
quickly, efficiently, and concisely as possible. The committee agreed that it
is important for EPA to recognize that new advances in the understanding
of the toxicity of TCDD, other dioxins, and DLCs could require reevalua
tion of key assumptions in the risk assessment document. The committee
recommends that EPA routinely monitor new scientific information with
the understanding that future revisions may be required to maintain a risk
assessment that is based on current state-of-the-science. However, the com
mittee also recognizes that stability in regulatory policy is important to the
regulated community and thus expects that science-based changes in regu
latory policy on TCDD, other dioxins, and DLCs will be invoked only in
the face of compelling new information that would warrant revision of its
final risk assessment. Such substantial gains in knowledge are not likely to
occur frequently.
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
http://www.nap.edu/catalog/11688.html
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Viluksela, M., B.U. Stahl, L.S. Birnbaum, and K.K. Rozman. 1997b. Subchronic/chronic
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Viluksela, M., B.U. Stahl, L.S. Birnbaum, and K.K. Rozman. 1998a. Subchronic/chronic tox
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Viluksela, M., Y. Bager, J.T. Tuomisto, G. Scheu, M. Unkila, R. Pohjanvirta, S. Flodstrom,
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Walisser, J.A., M.K. Bunger, E. Glover, and C. Bradfield. 2004a. Gestational exposure of Ahr
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Walker, N.J., P.W. Crockett, A. Nyska, A.E. Brix, M.P. Jokinen, D.M. Sells, J.R. Hailey, M.
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A ppendixes
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A
B io g ra p h ic a l In fo rm a tio n on
C o m m ittee M e m b e rs
From left to right: Nancy Kim, Alvaro Puga, Malcolm Pike, Michael Denison,
Andrew Renwick, Thomas McKone, Richard Di Giulio, David Savitz, David Eaton,
Norbert Kaminski, Joshua Cohen, Paul Terranova, Allen Silverstone, Gary Will
iams, Yiliang Zhu, Kimberly Thompson, Dennis Bier
David L. Eaton, Chair, is a professor in the Department of Environmental
and Occupational Health Sciences and the Public Health Genetics Program
in the school of Public Health and Community Medicine, and associate
vice provost for research at the University of Washington in Seattle. He is
also the director of the Center of Ecogenetics and Environmental Health at
the university and an associate director of the Fred Hutchinson Cancer
Research Center-University of Washington-Childrens’ Hospital and Medi
cal Center Cancer Center Research Consortium. He earned a B.S. in pre227
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APPENDIX A
medicine from Montana State University in 1974 and a Ph.D. in pharma
cology and toxicology from the University of Kansas Medical Center in
1978. Dr. Eaton’s research interests include the molecular basis of chemi
cally induced cancers and understanding how human genetic variation in
biotransformation enzymes may increase or decrease individual suscepti
bility to natural and synthetic chemicals found in the environment. He has
served on numerous boards and committees, including service as president
of the Society of Toxicology in 2001-2002 and as a member of the NRC
Board on Environmental Studies and Toxicology (BEST). Dr. Eaton has
served as chair of the NRC Committee on Emerging Issues and Data on
Environmental Contaminants and as a member of the Panel on Arsenic in
Drinking Water. Dr. Eaton has been awarded many distinguished fellow
ships and honors, including the Achievement Award from the Society of
Toxicology in 1990. He is an elected fellow of the Academy of Toxicologi
cal Sciences and the American Association for the Advancement of Science.
Dennis M. Bier is professor of pediatrics and director of the Children’s
Nutrition Research Center and program director of the General Clinical
Research Center at Baylor College of Medicine. Dr. Bier earned a B.S. from
Le Moyne College in 1962 and an M.D. from New Jersey College of Medi
cine in 1966. Dr. Bier’s research interests include the role of nutrition in
human health and in the prevention and treatment of disease and the role of
maternal, fetal, and childhood nutrition on the growth, development, and
health of children through adolescence. He also has professional interests in
the long-term consequences of nutrient inadequacy during critical periods
of embryonic and fetal life through infancy and childhood and on the
pathogenesis of adult chronic diseases. Dr. Bier has expertise in macronutri
ents (carbohydrate, lipid, and protein), intermediary metabolism, tracer
kinetics, diabetes, obesity, and endocrine disorders. Dr. Bier was elected to
the Institute of Medicine in 1997 and was a member of IOM’s Food and
Nutrition Board. He also served on the IOM Committee on Implications of
Dioxin in the Food Supply.
Joshua T. Cohen is a lecturer at Tufts New England Medical Center in the
Institute for Clinical Care Research and Health Policy Studies. He earned
his B.A. (1986) in applied mathematics and Ph.D. (1994) in decision sci
ences from Harvard University. Dr. Cohen’s research focuses on the appli
cation of decision analytical techniques to environmental risk management
problems with a special emphasis on the proper characterization and analy
sis of uncertainty. He was the lead author on a study comparing the risks
and benefits of changes in population fish consumption patterns, an analy
sis of the risks and benefits of cell-phone use while driving, and a study
comparing the costs and health impacts of advanced diesel and compressed
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229
natural gas urban transit buses. He has also played a key role in a risk
assessment of bovine spongiform encephalopathy (“mad cow disease” ) in
the United States.
Michael Denison is a professor in the Department of Environmental Toxi
cology at the University of California at Davis. He earned a B.S. from Saint
Francis College in 1977, an M.S. from Mississippi State University in 1980,
and a Ph.D. from Cornell University in 1983. Dr. Denison completed
postdoctoral training at the Hospital for Sick Children in Toronto, Canada,
and the Department of Pharmacology at Stanford University. He began his
professional career as an assistant professor in the Department of Biochem
istry at Michigan State University in 1988 and relocated to the University of
California in 1992. His research interests include the biochemical and
molecular mechanisms by which xenobiotics (particularly dioxins and re
lated chemicals and endocrine disruptors) interact with ligand-dependent
transcription factors to produce biological and toxicological effects in ani
mals. Dr. Denison is also examining the molecular and structural character
istics of the Ah receptor responsible for its binding and activation by dioxins
and structurally diverse xenobiotics. The application of molecular biological
approaches for the development of rapid high-throughput bioassay systems
for detection and characterization of ligands for xenobiotic receptors present
in environmental, biological, and food samples is another major research
area. Dr. Denison is co-chair of the International Advisory Board of the
annual International Dioxin Symposium and was the organizer and co-chair
of the 2003 U.S.-Vietnam Scientific Workshop on Methodologies of Dioxin
Screening, Remediation, and Site Characterization in Hanoi, Vietnam.
Richard Di Giulio is a professor of environmental toxicology at the Nicho
las School of the Environment and Earth Sciences, director of the Integrated
Toxicology Program, director of the Superfund Basic Research Center, and
director of the Center for Comparative Biology of Vulnerable Populations
at Duke University. He earned a B.A. from the University of Texas at
Austin in 1972, an M.S. from Louisiana State University in 1978, and a
Ph.D. from Virginia Polytechnic Institute and State University in 1982. Dr.
Di Giulio’s professional experience began as an assistant professor and
research associate at the School of Forestry and Environmental Studies at
Duke University in 1982. His research focuses on biochemical and molecu
lar responses of aquatic animals to environmental stressors, particularly
contaminants. Of particular concern are mechanisms of oxidative metabo
lism of aromatic hydrocarbons; mechanisms of free radical production and
antioxidant defense, and mechanisms of chemical carcinogenesis, develop
mental perturbations, and adaptations to contaminated environments by
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APPENDIX A
fishes. Dr. Di Giulio also has interests in the area of interconnections be
tween ecological and human health.
Norbert Kaminski is the director of the Center for Integrative Toxicology,
formerly known as the Institute for Environmental Toxicology at Michigan
State University. He is also a professor of pharmacology and toxicology in
the Department of Pharmacology and Toxicology. Dr. Kaminski earned a
B.A. in chemistry from Loyola University in 1978, an M.S. in toxicology in
1981, and a Ph.D. in toxicology and physiology in 1985 from North Caro
lina State University. Dr. Kaminski’s postdoctoral training was in
immunotoxicology at the Medical College of Virginia. He continued at the
Medical College of Virginia as a faculty member until 1993. His research
interests are in the areas of immunotoxicology and immunopharmacology
and, in particular, the molecular mechanisms by which dioxins alter B-cell
differentiation and function. Dr. Kaminski served on the IOM Committee to
Review the Health Effects in Vietnam Veterans of Exposure to Herbicides.
Nancy Kim is the director of the Division of Environmental Health Assess
ment, within the New York State Department of Health, and an associate
professor at the University of Albany School of Public Health. She earned a
B.A. in chemistry from the University of Delaware in 1964, and an M.S.
and Ph.D. in chemistry from Northwestern University in 1966 and 1969,
respectively. Her interests include toxicological evaluations, exposure as
sessments, risk assessment, structural activity correlations, and quantitative
relationships between toxicological parameters. Dr. Kim has held numer
ous panel memberships and is now a member of the National Center for
Environmental Health/Agency for Toxic Substances and Disease Registry
Board of Scientific Counselors. She has received several awards and honors,
including the Women in Government Award presented by the New York
State Department of Health. In 1999, Dr. Kim was inducted into the Delta
Omega Society, a national honorary public health society.
Antoine Keng Djien Liem is scientific coordinator of the Scientific Commit
tee of the European Food Safety Authority (EFSA) in Parma. Dr. Liem
earned an M.Sc. degree in environmental chemistry and toxicology from
the University of Amsterdam (1984) and a Ph.D. in biology from the Utrecht
University (1997). Following his university study in Amsterdam, Dr. Liem
began his career at the Department of Industrial Contaminants of the Dutch
National Institute of Public Health and the Environment (RIVM) in
Bilthoven. After the discovery of increased levels of dioxins in milk in cows
grazing in the vicinity of municipal waste incinerators, Dr. Liem was leader
of various multidisciplinary dioxin projects. He was appointed chairman of
the Dutch Working Group on Dioxins in Food and the Dutch Working
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231
Group on Dietary Intakes and acted as temporary adviser and national
delegate in the framework of studies of the World Health Organization
related to dioxins and related compounds. In 1998-2000, Dr. Liem acted as
the leader of the Dutch delegation coordinating a European Scientific Co
operation (SCOOP) Task on the assessment of dietary intake of dioxins and
related PCBs by the population of EU member states. This EU task was
jointly coordinated by RIVM and the Swedish National Food Administra
tion. The outcomes of the EU-SCOOP project were used in the risk assess
ments of dioxins and dioxin-like compounds in food carried out by the
EU’s Scientific Committee on Food (SCF), in which Dr. Liem contributed to
the Task Force preparing the opinion, and the Joint FAO/WHO Expert
Committee on Food Additives in 2001.
Thomas McKone is senior staff scientist and deputy department head at the
Lawrence Berkeley National Laboratory and an adjunct professor and re
searcher at the University of California at Berkeley School of Public Health.
He earned a Ph.D. from the University of California at Los Angeles in 1981.
Dr. McKone’s research interests include risk assessment methods, mass
transfer at environmental and human-environmental boundaries, model
uncertainty and reliability in exposure risk assessment, environmental and
occupational radioactivity, and biotransfer and bioconcentration. He is
very active in many research and professional organizations and is a mem
ber of the NRC Committee on the Selection and Use of Models in the
Regulatory Decision Process and was a member of the NRC Committee on
Toxicants and Pathogens in Biosolids Applied to Land. Dr. McKone is also
a member of the Advisory Council of the American Center for Life Cycle
Assessment and a member of the Organizing Committee for the Interna
tional Life-Cycle Initiative, a joint effort of the United Nations Environ
ment Program and the Society for Environmental Toxicology and Chemis
try. One of Dr. McKone’s most recognized achievements was his
development of the CalTOX risk assessment framework for the California
Department of Toxic Substances Control.
Malcolm Pike is a professor in the Department of Preventive Medicine at
the Norris Comprehensive Cancer Center at the University of Southern
California Keck School of Medicine. As a native of South Africa, he earned
a B.S. (honors) in mathematics from the University of Witwaterstand in
Johannesburg, South Africa, in 1956. He then studied statistics at Birkbeck
College of the University of London and earned a diploma in mathematical
statistics from Cambridge University in 1958. Dr. Pike received a Ph.D. in
mathematical statistics from Aberdeen University in Aberdeen, Scotland, in
1963. From 1963 to 1969, he was at the Statistical Research Unit of the
Medical Research Council at University College, London, and from 1969
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APPENDIX A
through 1973, he was the Regius Professor of Medicine at Oxford Univer
sity. Between 1973 and 1983, he held the position of professor of preven
tive medicine at the University of Southern California School of Medicine.
Following this, Dr. Pike was the director of the ICRF Cancer Epidemiology
and Clinical Trials Unit at Oxford University for 4 years. His research areas
include the epidemiology of breast, endometrial, and ovarian cancer. Dr.
Pike has received many distinguished honors, including the Brinker Interna
tional Award of the Susan G. Komen Breast Cancer Foundation in 1994
and the American Association for Cancer Research Award for Research
Excellence in Cancer Epidemiology and Prevention in 2004. He was elected
to the Institute of Medicine in 1994.
Alvaro Puga is a professor of molecular biology and environmental health
in the Department of Environmental Health at the University of Cincinnati,
director of the Center for Environmental Genetics and deputy director of
the Superfund Basic Research Program at the University of Cincinnati. In
Spain, he earned a Licenciate in Biology degree in 1966 from the Universidad
Complutense in Madrid. In the United States, Dr. Puga earned a Ph.D in
1972 from Purdue University and completed his postdoctoral training in
1976 with Scripps Clinic and Research Foundation in La Jolla, California.
His research interests include the molecular mechanisms of dioxin and
other environmental contaminants with the purpose of elucidating the sig
nal transduction pathways that underlie the biological responses
postexposure to these contaminants. He is also investigating the genetic
diversity on the response to exposure, specifically the genes that code for
transcription factors with a regulatory role in the expression of detoxifica
tion enzymes. Before joining the University of Cincinnati, he was the head
of the Unit on Pharmacogenetics, Laboratory of Developmental Pharma
cology, at the National Institute of Child Health and Human Development
(NICHHD) and the deputy chief of Laboratory of Developmental Pharma
cology at NICHHD. Dr. Puga was the recipient of the Society of Toxicol
ogy Award in 1999 and of the University of Cincinnati College of Medicine
Richard Akeson Award for Excellence in Teaching in 2002.
Andrew Renwick is an emeritus professor at the University of Southampton,
having retired from his position as professor of biochemical pharmacology
in September 2004. Dr. Renwick earned a B.Sc. degree in zoology and
chemistry in 1967, a Ph.D. in biochemistry in 1971, and a D.Sc. (medicine)
in pharmacology with toxicology in 1991 from the University of London.
He was appointed lecturer in biochemistry at St. Mary’s Hospital Medical
School from 1969 until 1976. Dr. Renwick was senior lecturer in clinical
pharmacology from 1976 to 1987 and was promoted to reader in clinical
pharmacology in 1987 and professor of biochemical pharmacology in 1997.
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Dr. Renwick’s research interests focused on the absorption and metabolism
of drugs and other foreign chemicals in humans following ingestion, inhala
tion, and dermal administration, in addition to species differences in the
fate of chemicals in the body. His U.K. governmental advisory committee
memberships have included the Medicines Commission, the Committee on
Toxicity, and the Committee on Carcinogenicity of the Department of
Health. In 2000, he was awarded an OBE (Officer [of the Order] of the
British Empire) for services to U.K. Medicines Licensing Authority and
Pharmacology and the Toxicology Forum George H. Scott Memorial Award
in 2002. This award was presented in recognition of Dr. Renwick’s efforts
to promote the advancement and application of the science of toxicology
with government, academics, and industry.
David Savitz is the Charles W. Bluhdorn Professor of Community and
Preventive Medicine and the director of the Center of Excellence in Epide
miology, Biostatistics, and Disease Prevention at Mount Sinai School of
Medicine. He earned a B.A. in 1975 from Brandeis University. In 1978, Dr.
Savitz earned an M.S. from the Department of Preventive Medicine and
then continued his education at the University of Pittsburgh, earning a Ph.D
from the School of Public Health’s Department of Epidemiology in 1982.
He began his professional career as an assistant professor in the Depart
ment of Preventative Medicine at the University of Colorado School of
Medicine. He joined the Department of Epidemiology at the University of
North Carolina School of Public Health in 1985 and became chair of the
department in 1996 and named Cary C. Boshamer Distinguished Professor
in 2003. His research covers the areas of reproductive, environmental, and
occupational epidemiology. Dr. Savitz is a member of many organizations
and has served as president of the Society for Epidemiologic Research.
Currently, Dr. Savitz is president of the Society for Pediatric and Perinatal
Epidemiologic Research. He has authored over 200 peer-reviewed journal
articles and is an editor of Epidemiology.
Allen Silverstone is professor of microbiology and immunology at SUNYUpstate Medical University and adjunct professor of environmental medi
cine at the University of Rochester School of Medicine. Dr. Silverstone
earned a B.A. from Reed College in 1965 and a Ph.D. from the Massachu
setts Institute of Technology in 1970. His research interests include the
cellular and molecular biology of how dioxins, estrogens, and estrogenic
compounds affect the immune system. Having identified the particular tar
get cell in T-cell development that is affected by dioxin, Dr. Silverstone’s lab
is now identifying the specific gene program activated by this agent in these
cells. He was a member of the review panel and a consultant to the Science
Advisory Board for EPA’s reassessment of dioxin and related compounds.
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APPENDIX A
Paul F. Terranova is professor of molecular and integrative physiology and
obstetrics and gynecology. He is director of the Center for Reproductive
Sciences at the University of Kansas Medical Center. He earned a B.S. in
1969 and an M.S. in 1971 in biology from McNeese State University and a
Ph.D. from Louisiana State University in 1975. Dr. Terranova is an interna
tionally recognized researcher in reproductive biology and has written or
co-written more than 100 peer-reviewed original research papers, 19 chap
ters in books or symposium proceedings, and numerous articles in interna
tional scientific journals. He has served on numerous review panels of the
National Institutes of Health, the National Science Foundation, and EPA. Dr.
Terranova’s research focuses on factors regulating follicular development
and ovulation. Recently, he has found that an environmental contaminant,
dioxin, prevents follicular rupture and he is assessing the endocrine and
molecular mechanisms by which this blockage occurs. Dr. Terranova has
also developed a mouse model of ovarian cancer and is determining which
growth regulators are involved in the spontaneous transformation of the
ovarian surface epithelial cells into a malignant phenotype.
Kimberly M. Thompson is an associate professor of risk analysis and deci
sion science at the Harvard School of Public Health and Children’s Hospital
Boston. Professor Thompson recently joined the systems dynamics group at
the Massachusetts Institute of Technology Sloan School of Management as a
visitor. She earned a B.S. and an M.S. in chemical engineering from the
Massachusetts Institute of Technology in 1988 and 1989, respectively, and
an Sc.D. in environmental health from Harvard School of Health in 1995.
Her research interests focus on issues related to developing and applying
quantitative methods for risk assessment and risk management in addition to
consideration of the public-policy implications associated with uncertainty
and variability in risk characterization. Dr. Thompson is a member of many
organizations and societies, including the Society for Risk Analysis and the
International Society for Exposure Analysis. She was a Sigma Xi Distin
guished Lecturer for 2003-2005 and has been the recipient of several honors,
including recognition in 2003 by the Society of Toxicology for an outstand
ing published paper demonstrating an application of risk assessment with
fellow colleagues and the 2004 Society for Risk Analysis Chauncey Starr
Award.
Gary M. Williams is the director of environmental pathology and toxicol
ogy, head of the program on medicine, food, and chemical safety; and a
professor of pathology at the New York Medical College since 1975. Dr.
Williams earned a B.A. from Washington and Jefferson College in 1963
and an M.D. from the University of Pittsburgh School of Medicine in 1967.
Following his residency at Massachusetts General Hospital, Dr. Williams
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APPENDIX A
235
began his career as assistant professor in the Department of Pathology at
Temple University School of Medicine in 1971. He is board certified in
pathology and toxicology. Dr. Williams’ research focuses on mechanisms
of chemical carcinogenesis and risk assessment. He has served on numerous
working groups and committees of the NRC, EPA, International Agency
for Research on Cancer, and World Health Organization. He has received
many honors, including the Arnold J. Lehman and Enhancement of Animal
Welfare Awards from the Society of Toxicology, and was elected fellow of
the Royal College of Pathologists (U.K.).
Yiliang Zhu is a professor and director of the biostatistics Ph.D. program and
the Center for Collaborative Research in the Department of Epidemiology
and Biostatistics, College of Public Health, University of South Florida. Dr.
Zhu earned a B.S. in computer science and applied mathematics from Shang
hai University of Science and Technology (1982), an M.S. in statistics from
Queen’s University (1987), and a Ph.D. in statistics from the University of
Toronto (1992). Dr. Zhu’s research includes benchmark dose methods, dose
response and PBPK modeling, and general methods in health risk assessment.
Dr. Zhu has served on several EPA committees, including the Peer Review
Committee on Neurobehavioral Dose-Response and Benchmark Method
Guidance, Peer Review Committee on Benchmark Dose Software, Toxico
logical Review for 2-Methylnaphthalene, STAR Program Grant Review Com
mittee for Global Change for Aquatic Ecosystems, and Peer Review Commit
tee on Benchmark Doses Technical Guidance Document. Dr. Zhu is a member
of the Advisory Committee on Organ Transplantation to the secretary of the
U.S. Department of Health and Human Services.
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Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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B
EPA’s 2 0 0 5 G u id elin es fo r
C a rc in o g e n R isk A sse ssm en t
“ CARCINOGENIC TO HUMANS”
This descriptor indicates strong evidence of human carcinogenicity. It
covers different combinations of evidence.
• This descriptor is appropriate when there is convincing epidemio
logic evidence of a causal association between human exposure and cancer.
• Exceptionally, this descriptor may be equally appropriate with a
lesser weight of epidemiologic evidence that is strengthened by other lines
of evidence. It can be used when all of the following conditions are met: (a)
there is strong evidence of an association between human exposure and
either cancer or the key precursor events of the agent’s mode of action but
not enough for a causal association, and (b) there is extensive evidence of
carcinogenicity in animals, and (c) the mode(s) of carcinogenic action and
associated key precursor events have been identified in animals, and (d)
there is strong evidence that the key precursor events that precede the
cancer response in animals are anticipated to occur in humans and progress
to tumors, based on available biological information. In this case, the nar
rative includes a summary of both the experimental and epidemiologic
information on mode of action and also an indication of the relative weight
that each source of information carries, e.g., based on human information,
based on limited human and extensive animal experiments.
236
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APPENDIX B
237
“LIKELY TO BE CARCINOGENIC TO HUMANS”
This descriptor is appropriate when the weight of the evidence is ad
equate to demonstrate carcinogenic potential to humans but does not reach
the weight of evidence for the descriptor “ Carcinogenic to Humans.” Ad
equate evidence consistent with this descriptor covers a broad spectrum. As
stated previously, the use of the term “likely” as a weight of evidence
descriptor does not correspond to a quantifiable probability. The examples
below are meant to represent the broad range of data combinations that are
covered by this descriptor; they are illustrative and provide neither a check
list nor a limitation for the data that might support use of this descriptor.
Moreover, additional information, e.g., on mode of action, might change
the choice of descriptor for the illustrated examples. Supporting data for
this descriptor may include
• an agent demonstrating a plausible (but not definitively causal) asso
ciation between human exposure and cancer, in most cases with some
supporting biological, experimental evidence, though not necessarily carci
nogenicity data from animal experiments;
• an agent that has tested positive in animal experiments in more than
one species, sex, strain, site, or exposure route, with or without evidence of
carcinogenicity in humans;
• a positive tumor study that raises additional biological concerns
beyond that of a statistically significant result, for example, a high degree of
malignancy, or an early age at onset;
• a rare animal tumor response in a single experiment that is assumed
to be relevant to humans; or
• a positive tumor study that is strengthened by other lines of evi
dence, for example, either plausible (but not definitively causal) association
between human exposure and cancer or evidence that the agent or an
important metabolite causes events generally known to be associated with
tumor formation (such as DNA reactivity or effects on cell growth control)
likely to be related to the tumor response in this case.
“ SUGGESTIVE EVIDENCE OF CARCINOGENIC POTENTIAL”
This descriptor of the database is appropriate when the weight of evi
dence is suggestive of carcinogenicity; a concern for potential carcinogenic
effects in humans is raised, but the data are judged not sufficient for a
stronger conclusion. This descriptor covers a spectrum of evidence associ
ated with varying levels of concern for carcinogenicity, ranging from a
positive cancer result in the only study on an agent to a single positive
cancer result in an extensive database that includes negative studies in other
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Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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238
APPENDIX B
species. Depending on the extent of the database, additional studies may or
may not provide further insights. Some examples include:
• a small, and possibly not statistically significant, increase in tumor
incidence observed in a single animal or human study that does not reach
the weight of evidence for the descriptor “ Likely to Be Carcinogenic to
Humans.” The study generally would not be contradicted by other studies
of equal quality in the same population group or experimental system (see
discussions of conflicting evidence and differing results, below);
• a small increase in a tumor with a high background rate in that sex
and strain, when there is some but insufficient evidence that the observed
tumors may be due to intrinsic factors that cause background tumors and
not due to the agent being assessed. (When there is a high background rate
of a specific tumor in animals of a particular sex and strain, then there may
be biological factors operating independently of the agent being assessed
that could be responsible for the development of the observed tumors.) In
this case, the reasons for determining that the tumors are not due to the
agent are explained;
• evidence of a positive response in a study whose power, design, or
conduct limits the ability to draw a confident conclusion (but does not make
the study fatally flawed), but where the carcinogenic potential is strengthened
by other lines of evidence (such as structure-activity relationships); or
• a statistically significant increase at one dose only, but no significant
response at the other doses and no overall trend.
“INADEQUATE INFORMATION TO ASSESS
CARCINOGENIC POTENTIAL”
This descriptor of the database is appropriate when available data are
judged inadequate for applying one of the other descriptors. Additional
studies generally would be expected to provide further insights. Some ex
amples include:
• little or no pertinent information;
• conflicting evidence, that is, some studies provide evidence of carci
nogenicity but other studies of equal quality in the same sex and strain are
negative. Differing results, that is, positive results in some studies and
negative results in one or more different experimental systems, do not
constitute conflicting evidence, as the term is used here. Depending on the
overall weight of evidence, differing results can be considered either sugges
tive evidence or likely evidence; or
• negative results that are not sufficiently robust for the descriptor,
“Not Likely to Be Carcinogenic to Humans.”
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APPENDIX B
239
“NO T LIKELY TO BE CARCINOGENIC TO HUMANS”
This descriptor is appropriate when the available data are considered
robust for deciding that there is no basis for human hazard concern. In
some instances, there can be positive results in experimental animals when
there is strong, consistent evidence that each mode of action in experimen
tal animals does not operate in humans. In other cases, there can be con
vincing evidence in both humans and animals that the agent is not carcino
genic. The judgment may be based on data such as:
• animal evidence that demonstrates lack of carcinogenic effect in
both sexes in well-designed and well-conducted studies in at least two
appropriate animal species (in the absence of other animal or human data
suggesting a potential for cancer effects),
• convincing and extensive experimental evidence showing that the
only carcinogenic effects observed in animals are not relevant to humans,
• convincing evidence that carcinogenic effects are not likely by a
particular exposure route (see Section 2.3), or
• convincing evidence that carcinogenic effects are not likely below a
defined dose range.
A descriptor of “not likely” applies only to the circumstances sup
ported by the data. For example, an agent may be “Not Likely to Be
Carcinogenic” by one route but not necessarily by another. In those cases
that have positive animal experiment(s) but the results are judged to be not
relevant to humans, the narrative discusses why the results are not relevant.
MULTIPLE DESCRIPTORS
More than one descriptor can be used when an agent’s effects differ by
dose or exposure route. For example, an agent may be “ Carcinogenic to
Humans” by one exposure route but “Not Likely to Be Carcinogenic” by a
route by which it is not absorbed. Also, an agent could be “Likely to Be
Carcinogenic” above a specified dose but “ Not Likely to Be Carcinogenic”
below that dose because a key event in tumor formation does not occur
below that dose.
Copyright © National Academy of Sciences. All rights reserved.
Health Risks from Dioxin and Related Compounds: Evaluation of the EPA Reassessment
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Copyright © National Academy of Sciences. All rights reserved.