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Saturday, April 29
8:00am–11:00am
2100—Adult Stem Cells—A Primer for the
Clinician
PAS/ASPHO Mini Course
Room 3014, Moscone West
Chairs: Jakub Tolar, University of Minnesota, Minneapolis, MN;
and Mervin C. Yoder, Jr., Indiana University School of
Medicine, Indianapolis, IN
Target Audience:
Hematologists/oncologists, endocrinologists, basic scientists
and neurologists.
Adult stem cells represent a
technology that is being intensively investigated currently,
and this research may have wide implications for human health.
This mini course will focus on recent research and potential
applications in human health.
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Introduction
Jakub Tolar, University of Minnesota, Minneapolis, MN
Mervin C. Yoder, Indiana
University School of Medicine, Indianapolis, IN
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Multipotent Adult Progenitor
Cell: Hype or Reality?
Catherine M. Verfaillie, University of Minnesota, Minneapolis, MN
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Mesenchymal Stem Cell: Harnessing
the Power of Adult Stem Cells To Repair Tissues
Darwin Prockop, Tulane University Health Science Center, New Orleans,
LA
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Hierarchy of Endothelial
Progenitors in Human Blood and Blood Vessels
David A. Ingram, Indiana University School of Medicine, Indianapolis,
IN
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Cancer Stem Cell: Concept of
Human Leukemic Development
Craig T. Jordan, James P. Wilmot Cancer Center, University of Rochester
School of Medicine, Rochester, NY
Sponsored jointly by
the American Society of Pediatric Hematology/Oncology and the
Pediatric Academic Societies
8:00am–11:00am
2125—New Considerations for the Growth Rate
of the Preterm Infant: Too Fast or Not Fast Enough?—A Review
of the Evidence
PAS Mini Course
Room 3002-3008, Moscone West
Chairs: Frank R. Greer, University of Wisconsin, Madison, WI;
and William W. Hay, Jr., University of Colorado, Aurora, CO
Target Audience: Neonatologists,
hospitalists who take care of preterm infants, nutritionists
and general pediatricians.
Recent nutritional emphasis in
the NICU has been to achieve the normal intrauterine growth
rate with more aggressive nutritional support for the low
birth weight infant. In general, this has been difficult to
achieve, and new evidence from long-term follow up studies
shows that preterm infants are at an increased risk of
developing the metabolic syndrome including obesity, type 2
diabetes and cardiovascular disease. This implies that the
organs in the early life of the preterm infant may be
programmed adversely by nutritional therapy. This raises the
questions of how fast these infants should grow (including
catch up growth), the importance of the composition of this
growth and the urgency for defining the necessary balance
between growth of the brain and the rest of the body.
Ultimately, providers may want to revise the long-term and
short-term goals for feeding very low birth weight or
extremely low birth weight infants. This mini course will
present evidence to help answer these questions and provide
discussion about related practice recommendations.
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Overview
Frank R. Greer, University of Wisconsin, Madison, WI
William W. Hay, University of
Colorado Health Sciences Center, Aurora, CO
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Macronutrient Requirements for
Growth of Preterm Infants—Upper and Lower Limits
(Energy, Fat, CHO, Protein)
Scott C. Denne, Indiana University School of Medicine, James Whitcomb
Riley Hospital, Indianapolis, IN
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Aggressive Nutritional Support of
the Preterm Infant Revisited—Evidence for Efficacy and
Safety
Patti J. Thureen, University of Colorado Health Sciences Center,
Denver, CO
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Adverse Outcomes of Rapid Somatic
Growth and Alterations of Body Composition in the Low
Birth Weight Infant
Frank R. Greer, University of Wisconsin, Madison, WI
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Fatty Acids and Neuronal
Development
Susan E. Carlson, University of Kansas Medical Center, Kansas City, KS
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Iron and Development of the Brain
Michael K. Georgieff, University of Minnesota School of Medicine,
Minneapolis, MN
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Nutritional Influences on
Structural and Functional Maturation of the Developing
Brain During Extended Postnatal Period
Steve H. Zeisel, The University of North Carolina, Chapel Hill, NC
8:00am–11:00am
2130—Newborn Hearing Screening: From the
Bedside to Beyond
PAS/PIDS Mini Course
Room 3010, Moscone West
Chairs: Mark R. Schleiss and Lisa Ann Schimmenti, University
of Minnesota Medical School, Minneapolis, MN
Target Audience: General
pediatricians, geneticists and infectious disease specialists.
Sensorineural hearing loss (SNHL)
in infants is the most common birth defect, and early
detection improves outcome. Evidence from the CDC reveals that
less than one half of screened babies are followed up. One
possible reason is the low positive predictive value of
bedside screening. There is a critical need to augment current
strategies to prevent late diagnosis of SNHL. One solution is
to propose second-tier testing for the most common causes of
SNHL, as the most common causes of newborn hearing loss are
infectious and genetic. Of infectious causes, cytomegalovirus
(CMV) is the most common. Evidence of CMV infection can be
found in 1% of newborns, with 10–15% developing hearing loss
or other CNS abnormalities. Of the genetic causes, mutations
in GJB2/GJB6 are the most common and are identified in up to
one half of individuals with SNHL. The goal of this program
will be to examine evidence for inclusion of infectious and
genetic screening to augment current newborn screening
protocols.
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Diagnostic Evaluation and
Management of Childhood Hearing Loss
Margaret Alene Kenna, Children's Hospital Boston, Boston, MA
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Range of Mutations in
GJB2-Associated Hearing Loss
Lisa Ann Schimmenti, University of Minnesota Medical School,
Minneapolis, MN
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Congenital Cytomegalovirus
Infection and Hearing Loss
Karen B. Fowler, University of Alabama at Birmingham, Birmingham, AL
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Newborn Hearing Screening:
Audiologic Assessment
Yvonne Sininger, University of California Los Angeles, Los Angeles, CA
Sponsored jointly by
the Pediatric Infectious Diseases Society and the Pediatric
Academic Societies
8:00am–12:00pm
2180A—LWPES Plenary Session I
LWPES Plenary Session
Room 3007-3009, Moscone West
Chairs: Lynne Levitsky, Massachusetts General Hospital,
Boston, MA; Henry Anhalt, Saint Barnabas Medical Center,
Livingston, NJ; and Alan D. Rogol, University of Virginia,
Charlottesville, VA
Target Audience:
Endocrinologists, nephrologists, cardiologists, general
pediatricians, immunologists, geneticists and molecular
biologists.
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Opening Remarks
Lynne L. Levitsky, Massachusetts General Hospital, Boston, MA
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Lawson Wilkins Lecture:
Recent years have witnessed a
significant revision of the traditional view of fat cells
as simple stores of excess energy. Studies in the
speaker's lab as well as many others have clearly
demonstrated that adipocytes produce and regulate many
metabolic and hormonal signals, which generate profound
effects on systemic endocrine equilibrium. In his earlier
studies, he also demonstrated that these cells exhibit an
inflammatory capacity that is abnormal in obesity and key
to the pathogenesis of insulin resistance and diabetes.
Recently, he identified a key molecular mechanism
underlying the link between inflammatory responses and
insulin action. This pathway involves obesity-related
activation of the serine, threonine kinase, JNK, and the
consequent inhibition of insulin receptor signaling via
phosphorylation of a substrate of insulin receptor, IRS-1.
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Integration of Metabolic and
Inflammatory Pathways in Metabolic Disease
Gokhan S. Hotamisligil, Harvard School of Public Health, Boston, MA
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Robert Blizzard Lecture:
One of the greatest questions
asked of physicians caring for children with autoimmune
diabetes is "why did this happen?" This session
will unravel some of the mysteries surrounding the
etiology and pathogenesis of autoimmune diabetes from an
investigator who has dedicated his life to this issue.
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On the Unravelling of the
Etiopathogenesis of Type 1 Diabetes: Are We Stuck or
Are We Winning?
Gian Franco Bottazzo, Ospedale Pediatrico Bambino Gesú, Scientific
Institute, Rome, Italy
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Break
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Esoterix Lecture:
The attendee will familiarize
him/herself with newer molecular mechanisms of growth
failure that are due to abnormalities in receptor and
post-receptor translation of GH signaling.
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Molecular Mechanisms and Defects
in Growth Hormone Receptor Signaling
Peter Rotwein, Oregon Health and Science University, Portland, OR
9:00am–11:00am
2196—Modulators of Bronchopulmonary
Dysplasia
PAS Platform Session
Room 3003-3005, Moscone West
Chairs: Suhas G. Kallapur and Lawrence M. Nogee
Supported by an unrestricted educational grant from Dey,
L.P.
12:00pm–3:00pm
2505—Embryonic Stem Cells: A Primer for
Clinicians
PAS Mini Course
Room 3014, Moscone West
Chair: Michael T. Longaker, Stanford University, Stanford, CA
Embryonic stem cells offer
incredible promise for treating diseases affecting both
children and adults. This mini course will provide an overview
of stem cells and a basic understanding of how to derive human
embryonic stem cells, recent research and ethical
considerations. After attending this session, attendee will
have a better understanding of: 1) what are embryonic stem
cells; 2) how human embryonic stem cells are derived; 3)
recent progress in human embryonic stem cell research; 4)
ethical considerations in human embryonic stem cells.
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Stem Cells: Embryonic, Adult and
Cancer
Michael T. Longaker, Stanford University, Stanford, CA
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What It Takes Clinically To Get
an Embryonic Stem Cell
Linda C. Giudice, University of California, San Francisco, San
Francisco, CA
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What Can You Do with an Embryonic
Stem Cell in Research
Renee Reijo Pera, University of California, San Francisco, San
Francisco, CA
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Ethical and Oversight
Considerations in Human Embryonic Stem Cell Research
Hank Greely, Stanford University, Stanford, CA
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Panel Discussion
Supported in part by an unrestricted educational grant from
Treuman Katz Center for Pediatric Bioethics - Seattle
Children's Hospital
12:00pm–3:00pm
2510—Inherited Disorders Caused by
Inappropriate Apoptosis
PAS Mini Course
Room 3010, Moscone West
Chairs: Cynthia J. Tifft, Children's National Medical Center,
Washington, DC; and Hans Andersson, Tulane University Medical
Center, New Orleans, LA
Target Audience: Pediatric
researchers interested in genetic basis of disease and
apoptosis.
This session will describe the
recent findings of the role of apoptosis in the pathogenesis
of genetic diseases. Inappropriate apoptosis and acquired
resistance to apoptosis are important mechanisms in some
genetic disorders and a better understanding of this role is
expected to lead to potential therapies.
Inappropriate apoptosis has been
implicated in the causation of several inherited disorders
with specific interest for pediatricians. The pathophysiology
of inherited neurodegenerative disorders have long eluded
explanation and recent studies suggest that storage of
abnormal compounds in lysosomes act as a trigger for
apoptosis. Additionally, nephropathic cystinosis has recently
been shown to be caused by inappropriate onset of apoptosis
caused by abnormal cystinylation. This session will provide a
clinical perspective on the role of apoptosis in genetic
disorders affecting the pediatric population.
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Overview
Hans C. Andersson, Tulane University Medical School, New Orleans, LA
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Microglial Activation and
Inflammation Precedes Apoptosis in Tay-Sachs Disease
Cynthia J. Tifft, Children's National Medical Center, Washington, DC
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Lysosomal Cystine Enhances
Apoptosis and Yields the Nephropathic Cystinotic Phenotype
Jess G. Thoene, Tulane University School of Medicine, New Orleans, LA
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Niemann Pick Disease, Type C:
Glycolipid Gridlock and Apoptosis
Marc C. Patterson, Columbia University Medical Center, New York, NY
- Role of GM1-Ganglioside
in ER-and Mitochondrial-Mediated Neuronal Apoptosis
Alessandra D'Azzo, St. Jude
Children’s Research Hospital, Memphis, TN
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Discussion
1:00pm–3:00pm
2610—Genetics and Epigenetics of Neonatal
Disease
PAS Platform Session
Room 3022, Moscone West
Chairs: Aaron Hamvas and Jeffrey C. Murray
1:30pm–3:30pm
2670A—Controversies in Care in Pediatric
Endocrinology—The Great Debates
LWPES Workshop
Room 3001, Moscone West
Chairs: William Clarke, University of Virginia,
Charlottesville, VA; and Henry Anhalt, St. Barnabas Medical
Center, Livingston, NJ
Target Audience:
Endocrinologists, general pediatricians and adolescent
medicine specialists.
The attendee will be part of a
lively debate on a number of areas of controversy in pediatric
state-of-the-art diabetes management.
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Is Primary Prevention of Type 1
Diabetes Possible?
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Pro—Desmond A. Schatz, University of Florida, Gainesville, FL
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Con—Dorothy J. Becker, Children's Hospital of Pittsburgh, Pittsburgh, PA
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Should Glucose Sensors Be
Routinely Used?
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Pro—Stuart Alan Weinzimer, Yale School of Medicine, New Haven, CT
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Con—Darrell M. Wilson, Stanford University Medical Center, Stanford, CA
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Should Metformin Be Used To Treat
Pediatric Patients with Insulin Resistance?
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Pro—Michael S. Freemark, Duke University Medical Center, Durham, NC
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Con—Philip Scott Zeitler, University of Colorado at Denver and Health
Sciences Center, Denver, CO
3:00pm–5:00pm
2710A—Hematopoietic Cell
Transplantation—An Update
ASPHO Symposium
Room 3016-3018, Moscone West
Chairs: Jakub Tolar and K. Scott Baker, University of
Minnesota, Minneapolis, MN
The program will begin with a
review of unrelated umbilical cord blood transplantation, now
a common practice in the treatment of pediatric malignancies.
The program will follow with a presentation of the most recent
data on reduced intensity hematopoietic cell transplantation
for treatment of malignant and non-malignant diseases in
children. The symposium will conclude with an overview of
immune implications of mesenchymal stem cell infusion,
including their use for graft versus host disease prophylaxis
and treatment. Cellular therapy has yielded notable successes
in the past decade and holds considerable promise, and one
should walk away from the session with a realistic overview of
the possibilities and limitations of cellular therapy for
childhood cancer.
After attending this session, it
is expected that the learner will be able to:
1. Identify efficacious cellular
therapy approaches.
2. Recognize the limitations of cellular therapy for childhood
cancer.
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Introduction
Jakub Tolar, University of Minnesota, Minneapolis, MN
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Umbilical Cord Blood
Transplantation: Current Practice and Future Innovations
John E. Wagner, University of Minnesota Medical School, Minneapolis, MN
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Non-myeloablative Hematopoietic
Cell Transplantation in Children
Morris Kletzel, Northwestern University Feinberg School of Medicine,
Chicago, IL
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Immunobiology of Mesenchymal Stem
Cells
Katarina Le Blanc, Karolinska University, Stockholm, Sweden
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Question and Answer Session
3:15pm–5:15pm
2725—Integrating Genetic Susceptibility and
Environmental Influences in Pediatric Research
PAS Topic Symposium
Room 2008, Moscone West
Chair: Bruce P. Lanphear, Cincinnati Children's Environmental
Health Center, Cincinnati Children’s Hospital Medical
Center, Cincinnati, OH
Target Audience: A broad
pediatric audience with the goal of promoting
interdisciplinary understanding and greater integration of
genetic and environmental research.
Asthma, preterm birth, ADHD and
other prevalent pediatric conditions are widely recognized to
result from interactions of environmental influences and
genetic susceptibility. Tremendous progress has been made in
measuring both environmental and genetic risk factors.
Increasingly, researchers are moving beyond ecological methods
(e.g., questionnaires, air monitoring) to directly measure in
humans hundreds of environmental chemicals, from nicotine to
metals to DDT and phthalates. Similarly, unprecedented
innovation has led rapidly to high-throughput methods that
assess DNA variation across large cohorts. New
interdisciplinary collaborations that integrate state of the
art approaches to both environmental and genetic influences
should greatly improve our ability to predict and prevent
disease and disability. Such studies will be critical for
understanding mechanistic pathways, defining susceptible
subpopulations and developing effective interventions. This
session will provide an overview of gene–environment
research, describe recent advances in biomarkers of
environmental exposure and review new methods for measuring
genetic variability.
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Gene–Environment Interaction in
Common Pediatric Conditions: Conceptual Overview and
Recent Evidence
Robert S. Kahn, Cincinnati Children’s Hospital Medical Center,
University of Cincinnati College of Medicine, Cincinnati,
OH
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Advances in Biomarkers of
Environmental Exposure in Pediatric Research
Bruce P. Lanphear, Cincinnati Children's Environmental Health Center,
Cincinnati Children’s Hospital Medical Center,
Cincinnati, OH
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Measuring Genetic Susceptibility
to the Environment: Study Designs and Genotyping Methods
Robert O. Wright, Harvard Children's Environmental Health Center,
Boston Children's Hospital and the Harvard School of
Public Health, Boston, MA
3:15pm–5:15pm
2730—Mechanisms of Hypertension in the
Molecular Era
PAS/ASPN/IPHA/LWPES Topic Symposium
Room 2003-2005, Moscone West
Chairs: Bruce Z. Morgenstern, Phoenix Children's Hospital,
Phoenix, AZ; and Julie R. Ingelfinger, Massachusetts General
Hospital, Boston, MA
Target Audience: General
pediatricians, nephrologists, endocrinologists and
neonatologists.
Our understanding of the
pathophysiology of hypertension has been changing rapidly due
to advances in molecular genetics, most notably the
identification of several single-gene defects that cause
hypertension. This session will update participants on the
latest advances in our knowledge of molecular mechanisms of a
variety of forms of hypertension.
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Role of Dopamine Receptors
Pedro A. Jose, Georgetown University Medical Center, Washington, DC
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Perinatal Programming and the
Development of Hypertension
Lori Woods, Oregon Health & Science University, Portland, OR
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Low Renin Hypertension in
Childhood
Maria I. New, Mount Sinai School of Medicine, New York, NY
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WNK Kinases and Blood Pressure
Regulation
Richard Lifton, Yale University School of Medicine, New Haven, CT
Sponsored jointly by
the American Society of Pediatric Nephrology, the
International Pediatric Hypertension Association, the Lawson
Wilkins Pediatric Endocrine Society and the Pediatric Academic
Societies
3:15pm–5:15pm
2763—New Approaches in Stem Cell Technology
PAS Educational Workshop
Golden Gate Hall A1, SF Marriott
Leader: Kathleen Sakamoto, Los Angeles, CA; Co-leaders: Ed
Horwitz, Harley Kornblum, and Punam Malik
Recent advances in molecular and
cellular techniques have provided new approaches to studying
the role of gene function in a variety of cell types,
including stem cells. It is critical for pediatricians and
pediatric subspecialists to understand the basis and use of
these emerging technologies. This workshop will provide an
overview of new methods that are currently being used to study
stem cells. The topics include RNA interference (RNAi),
isolation and purification of stem cells, gene expression
profiling, and gene therapy. Upon completion of this workshop,
participants will be able to describe (a) uses of RNAi in stem
cells, (b) approaches to isolate and purify mesenchymal stem
cells, (c) characterization of neural stem cells using
expression profiling, and (d) applications of gene therapy and
stem cells.
3:15pm–5:15pm
2767—How Do I Incorporate Proteomics
Technology Into My Research?
PAS Educational Workshop
Golden Gate Hall A3, SF Marriott
Leader: Anne Murphy, Johns Hopkins University School of
Medicine, Baltimore, MD; and Co-leader: James Schilling,
Stanford University School of Medicine, Stanford, CA
Laboratory scientists,
physician-scientists and trainees as well as clinical
scientists interested in disease biomarkers.
Proteomics is a technology driven
field. Many investigators not currently employing these
technologies would love to incorporate them into their
research. This workshop will address some strategies for
investigators contemplating the use of the gel,
mass-spectrometry, and array based approaches. Discussions of
strategies to choose technologies to match the question,
strategies for assessing post-translational modifications, and
how to best work with a core facility will be addressed. The
workshop attendees should emerge with an understanding of the
strengths and weaknesses of various broad-based proteomic
technologies. This will permit them to match techniques with
an experimental question and to maximize interactions with
core facilities already in place within their institutions.
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Approaches To Analyze The
Phosphoproteome
Anne M. Murphy, Johns Hopkins University School of Medicine, Baltimore,
MD
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Overview of Approaches
David R. Graham, Johns Hopkins University-Bayview Campus, Baltimore, MD
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Sample preparation
James Malone, Washington University School of Medicine, St. Louis, MO
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Mass Tagging Approaches to
Biomarker Discovery
K.W. Michael Siu, Centre for Research in Mass Spectrometry, York
University, Toronto, Ontario Canada
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Statistics and Data Analysis
Robert Tibshirani, Stanford University, Stanford, CA
Supported in part by an unrestricted educational grant from
Applied Biosystems and GE Healthcare
3:45pm–5:15pm
2785A—Celiac Disease for the
Non-gastroenterologist
LWPES Workshop
Room 3010, Moscone West
Chair: Michelle M. Pietzak, Children's Hospital of Los
Angeles, Los Angeles, CA
Target Audience:
Gastroenterologists and endocrinologists.
Celiac disease affects
approximately 10-15% of children with diabetes. Often times
the screening tests are vexing. This workshop is aimed at
clarifying the disease process and how to diagnose it.
-
Celiac Disease for the
Non-gastroenterologist
Michelle M. Pietzak, Children's Hospital of Los Angeles, Los Angeles,
CA
3:45pm–5:15pm
2790A—Hyperthyroidism
LWPES Workshop
Room 3000, Moscone West
Chair: Scott A. Rivkees, Yale School of Medicine, New Haven,
CT
Target Audience: Generalists.
Much controversy exists about the
most effective and safest treatments for hyperthyroidism in
children. This workshop will clarify some of the newer
evidence based approaches to the diagnosis and management of
hyperthyroidism, with a special emphasis on radioactive
ablation.
3:45pm–5:15pm
2795A—Neonatal Diabetes
LWPES Workshop
Room 3001, Moscone West
Chair: Mark A. Sperling, Children's Hospital of Pittsburgh,
Pittsburgh, PA
Target Audience: Endocrinologists
and neonatologists.
Over the past few years much has
been learned about the pathogenesis of neonatal diabetes. This
workshop will impart knowledge on important considerations in
the diagnosis and work-up of this rare condition.
Sunday, April 30
8:00am–10:00am
3100—Advances in Pathogenesis, Diagnosis
and Management of Kawasaki Disease
PAS/PIDS Topic Symposium
Room 3001, Moscone West
Chairs: Marian Melish, University of Hawaii, Kapiolani
Children's Hospital, Honolulu, HI; and Stanford T. Shulman,
Children's Memorial Hospital, Northwestern University,
Feinberg School of Medicine, Chicago, IL
Target Audience: Infectious
disease specialists, cardiologists, rheumatologists,
immunologists and primary care pediatricians.
Cloning the IgA antibody response
in acute Kawasaki Disease has led to exciting new insights
into the etiology and pathogenesis of this enigmatic illness.
The diagnosis of incomplete Kawasaki Disease remains a
significant clinical problem, and new guidelines have been
published to help the clinician in making this diagnosis.
Approximately 10–15% of children with acute Kawasaki Disease
do not respond to conventional intravenous gammaglobulin and
aspirin therapy, and new data regarding treatment with
steroids and Remicade are emerging. Knowledge regarding
optimal management of cardiac complications and long-term
outcome continues to evolve as patients diagnosed with
Kawasaki Disease in the 1970s and 1980s age.
-
Overview
Marian E. Melish, University of Hawaii, Kapiolani Children's Hospital,
Honolulu, HI
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IgA Response in Acute Kawasaki
Disease Targets Inclusion Bodies in Acute Kawasaki Disease
Bronchial Epithelium
Anne H. Rowley, Northwestern University, Children's Memorial Hospital,
Chicago, IL
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Clinical Dilemma of Diagnosing
Incomplete Kawasaki Disease
Jane C. Burns, University of California, San Diego, CA
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Treatment of Refractory Kawasaki
Disease
Stanford T. Shulman, Children's Memorial Hospital, Northwestern
University, Feinberg School of Medicine, Chicago, IL
-
Management of Cardiac
Complications and Long-Term Outcome
Jane W. Newburger, Harvard University, Children’s Hospital of Boston,
Boston, MA
Sponsored jointly by
the Pediatric Infectious Diseases Society and the Pediatric
Academic Societies
8:00am–10:00am
3110—Probiotics in Necrotizing
Enterocolitis—Their Clinical Effect and Possible Mechanisms
PAS/ASPR/JPS/NASPGHAN Topic Symposium
Room 3003-3005, Moscone West
Chairs: W. Allan Walker, Harvard Medical School, Boston, MA;
and Yuichiro Yamashiro, Juntendo University School of
Medicine, Tokyo, Japan
Target Audience: Neonatologists,
gastroenterologists, pediatric surgeons, NICU nurses and
bacteriologists in perinatal medicine.
Necrotizing enterocolitis (NEC)
is a serious gastrointestinal disease seen predominantly in
very low birth weight (VLBW) and extremely low birth weight (ELBW)
infants. NEC is probably a complex, multifactorial disease.
Currently, the precise pathogenic mechanisms remain to be
elucidated; however, clinical use of probiotics has been
reported to be useful for preventing NEC development in VLBW
and ELBW infants. This session will provide us the current
knowledge about the role of probiotics in the management of
NEC.
-
Fifteen-Year's Experience of
Early Administration of Bifidobacterium Breve to Preterm
Infants
H. Kitajima, Osaka Medical Center and Research Institute for Maternal
and Child Health, Osaka, Japan
-
Oral Probiotics Reduces Incidence
of NEC in VLBW Infants
H. C. Lin, China Medical University, Taichung, Taiwan
-
Effects of Probiotics on the
Immunological Development and Short Chain Fatty Acids in
ELBW and VLBW Infants
Yoshikazu Ohtsuka, Juntendo University School of Medicine, Bunkyo-ku,
Japan
-
Possible Role of Probiotic
Supplementation for Prevention from NEC
Michael S. Caplan, Northwestern University, Evanston, IL
Sponsored jointly by
the Asian Society for Pediatric Research; Japan Pediatric
Society; North American Society for Pediatric
Gastroenterology, Hepatology & Nutrition and the Pediatric
Academic Societies
8:00am–10:00am
3125—Developmental Origins of Adult
Disease—Metabolism
PAS Platform Session
Room 3010-3012, Moscone West
Chairs: William W. Hay and Rebecca A. Simmons
8:00am–10:00am
3145—Genetic Basis of Disease: Pathogenesis
and Treatment
PAS Platform Session
Room 3000, Moscone West
Chairs: George A. Diaz and
Brendan H. Lee
Includes
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SPR Fellow's Basic Research
Award: Conditional Mutagenesis of the Homeobox Gene [italic]Hhex[/italic]
Reveals Novel and Essential Roles in Development of the
Liver and Extrahepatic Biliary Tract
Michael Hunter, Yale University School of Medicine, New Haven, CT
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SPR Student Research Award: Germ
Line [italic]KRAS[/italic] Mutations Encoding Proteins
with Novel Biochemical and Functional Properties Cause
Disorders of the Noonan Syndrome Spectrum
Suzanne Schubbert, University of California, San Francisco, CA
8:00am–11:00am
3240—Manuscript Preparation and the Process
of Peer-Reviewed Publication
PAS Educational Workshop
Willow, SF Marriott
Leader: Stephen Daniels, Denver, CO; Co-leaders: Thomas Welch,
Robert Wilmott, Sarah Long
Target Audience: Trainees,
fellows, junior faculty, mid-level faculty, senior faculty,
community practitioners.
This interactive workshop will
address multiple aspects of publication in scientific journals
and provides insights from editors of The Journal of
Pediatrics on the publication process. Presenters will discuss
preparation of materials, including the initial decision that
the data are sufficient to justify publication. Issues related
to manuscript writing will include length, focus, adherence to
journal formats, and referencing. The editorial process, from
submission to publication, will be described in depth, with
particular attention to ways in which authors can interact
with journal editors. Another section of the workshop will
cover ethical issues in publication including review boards,
authorship, duplicate publication, intellectual property
rights, and conflict of interest. There will be open
discussion of sample cases and questions derived from the
experiences of the participants.
Objectives:
– To learn about preparing and
submitting work for publication in peer-reviewed journals.
– To discuss ethical issues related to publication of
research and clinical work.
– To have the opportunity to ask and have answered questions
about publication and to offer insights.
Format: Open discussion,
question-and-answer.
Designed to meet elements of the
core curriculum for pediatric fellowship subspecialty
training.
9:45am–11:45am
3300A—Pure Red Cell Aplasia
ASPHO Symposium
Room 3016-3018, Moscone West
Chair: Jeffrey M. Lipton, Schneider Children’s Hospital, New
Hyde Park, NY
The pure red cell aplasias (PRCA)
represent a form of bone marrow restricted to the erythroid
lineage. Research has led to new insights into the molecular
basis of erythroid development. Transcriptional regulation of
erythropoiesis will be discussed in this symposium. In
pediatrics, intrinsic stem/progenitor cell defects are the
most important cause of red cell failure. One of the most
common forms of pure red cell aplasia in pediatric patients is
Diamond-Blackfan Anemia. The clinical and molecular basis of
this disease will be presented. Congenital dyserythropoietic
anemias (CDA) are rare forms of erythroid cytopenias. New
information on the pathophysiology of CDA will be presented
during this symposium.
After attending this session, it
is expected that the learner will be able to:
1. Describe the biology of
erythropoiesis.
2. Describe the clinical presentation and molecular basis of
Diamond-Blackfan Anemia.
3. Describe the clinical presentation and pathophysiology of
congenital dyserythropoietic anemia.
-
Introduction: How
"Pure" Is Pure Red Blood Cell Aplasia?
Jeffrey M. Lipton, Schneider Children's Hospital, New Hyde Park, NY
-
Biology of Erythroid Development
Mitchell Weiss, Children's Hospital of Phiadelphia, Philadelphia, PA
-
Clinical and Molecular Biology of
Diamond-Blackfan Anemia
Adrianna Vlachos, Schneider Children's Hospital, New Hyde Park, NY
-
Congenital Dyserythropoietic
Anemias 2006: Where Are We Now?
Bertil E. Glader, Stanford University School of Medicine, Stanford, CA
-
Question and Answer Session
2:00pm–4:00pm
3705—Infections at the
Maternal–Placental–Fetal Interface: Immunopathogenesis of
Group B Streptococcus, Listeria monocytogenes and
Cytomegalovirus
PAS/PIDS Topic Symposium
Room 3022-3024, Moscone West
Chairs: John R. Schreiber, University of Minnesota Medical
School and University of Minnesota Children's
Hospital/Fairview, Minneapolis, MN; and Robert F. Pass,
University of Alabama at Birmingham, Birmingham, AL
Target Audience: Neonatologists,
infectious disease specialists, immunologists, developmental
biologists and general pediatricians.
Infections in newborns commonly
result from acquisition either during the delivery process or
transplacentally. The host and pathogen factors that
contribute to acquisition of infections at the
maternal–placental–fetal interface are poorly understood.
This symposium will review the basic science and
immunopathogenesis of three diverse pathogens that all share
the ability to cause infections at the placental level:
cytomegalovirus, group B streptococcus, and Listeria
monocytogenes.
-
Intrauterine Cytomegalovirus
Infection, Transplacental Spread of Virus and Control by
Maternal Immunity
Lenore Pereira, University of California San Francisco, San Francisco,
CA
-
Host and Bacterial Factors in
Invasive Group B Streptococcal Infection
Craig E. Rubens, University of Washington, Seattle, WA
-
Listeriosis in the Pregnant
Guinea Pig: A Model of Vertical Transmission
Daniel A. Portnoy, University of California Berkeley, Berkeley, CA
-
Discussion
Sponsored jointly by
the Pediatric Infectious Diseases Society and the Pediatric
Academic Societies
2:00pm–4:00pm
3722—Neonatal Lung Inflammation: Mechanisms
and Clinical Implications
PAS Platform Session
Room 3014, Moscone West
Chairs: Rose M. Viscardi and Stephen E. Welty
Supported by an unrestricted educational grant from Dey,
L.P.
2:00pm–4:00pm
3732—Pulmonary and Cardiac Development:
Transcriptional Control and Stem Cells
PAS Platform Session
Room 2004, Moscone West
Chairs: Lawrence M. Nogee and George A. Porter
Includes
-
SPR Student Research Award:
Critical Requirement of C/EBP[alpha] for Lung Maturation
and Funct
Prithy Martis, Cincinnati Children's Hospital Medical Center,
Cincinnati, OH
-
Role of MicroRNAs in
Cardiogenesis
Yong Zhao, Gladstone Institute of Cardiovascular Disease, University of
California San Francisco
2:00pm–5:00pm
3750—Endocrine Disrupters
PAS/LWPES Mini Course
Room 3010-3012, Moscone West
Chairs: Mary M. Lee, University of Massachusetts Medical
School, Worcester, MA; and Henry Anhalt, Saint Barnabas
Medical Center, Livingston, NJ
Target Audience:
Endocrinologists, generalists, neonatologists and basic
scientists.
Concerns regarding clinical
consequences of endocrine disrupting chemicals have increased
over the past decade as researchers have documented
detrimental effects in wildlife. Federal attention to
endocrine disrupters began in earnest in 1996 when the U.S.
Congress passed the Food Quality Protection Act and amended
the Safe Drinking Water Act. These laws mandated testing to
determine if pesticides and industrial chemicals might behave
like hormones; therefore, the U.S. EPA formed the Endocrine
Disrupters Screening and Advisory Committee. In addition to
direct effects, some environmental disrupters act through
non-genomic actions, some of which persist for several
generations. This program presenting innovative studies on
mechanisms of action of endocrine disruptors will be of
critical interest to endocrinologists, both clinical and basic
scientists, as well as public health experts.
-
Prenatal Programming with
Estrogen/Estrogen Mimetics
Kenneth S. Korach, National Institute of Environmental Health Sciences,
Research Triangle Park, NC
-
Epigenetic Transgenerational
Actions of Endocrine Disruptors on Male Fertility and
Other Diseases
Michael K. Skinner, Washington State University, Pullman, WA
-
Prenatal Programming with Native
and Environmental Steroids
Vasantha Padmanabhan, University of Michigan, Ann Arbor, MI
Sponsored jointly by
the Lawson Wilkins Pediatric Endocrine Society and the
Pediatric Academic Societies
4:15pm–5:45pm
3805—Fetal Homeland Security: New Insights
into Old Threats
PAS State of the Art Plenary
Room 3002-3008, Moscone West
Chairs: Phil W. Shaul, University of Texas Southwestern
Medical Center, Dallas, TX; and Rashmin C. Savani, University
of Texas Southwestern Medical Center, Dallas, TX
Target Audience: Neonatologists,
pediatricians and researchers interested in perinatal biology.
In addition to premature birth,
there are a select number of maternal conditions that have
marked negative impact on the well being of the fetus and
newborn. This symposium will highlight recent advances in our
understanding of these classical threats to our most
vulnerable pediatric patient population.
First, new knowledge of the
mechanisms by which maternal diabetes alters embryonic and
fetal development will be discussed. Second, the newly
discovered role of circulating anti-angiogenic proteins of
placental origin in the pathogenesis of preeclampsia will be
presented. Finally, novel mechanisms by which biochemical
events in the fetal lung trigger the initiation of labor will
be discussed. Further advances in each of these realms will
ultimately lead to new therapies to protect the fetus and
yield healthy outcomes at term.
-
Mechanisms by Which Maternal
Diabetes Modifies Embryonic and Fetal Development
Kelle H. Moley, Washington University School of Medicine, St. Louis, MO
-
Role of Circulating Anti-angiogenic
Proteins of Placental Origin in the Pathogenesis of
Preeclampsia
S. Ananth Karumanchi, Harvard Medical School, Beth Isreal Deaconess
Medical Center, Boston, MA
-
Fetal–Maternal Signaling in the
Initiation of Labor
Carole R. Mendelson, University of Texas Southwestern Medical Center,
Dallas, TX
4:15pm–5:45pm
3810—RNA Interference, Technological
Development of siRNAs and Potential Treatments for Childhood
Diseases
PAS State of the Art Plenary
Room 3016-3018, Moscone West
Chair: R. Alan B. Ezekowitz, Harvard Medical School,
Massachusetts General Hospital, Boston, MA
Target Audience: Basic scientists
studying a broad range of childhood diseases, translational
scientists of all disciplines studying clinical implications
of basic science research, clinical scientists studying
childhood and other diseases in need of improved therapies and
clinicians interested in cutting-edge science and its medical
implications.
RNA interference is a recently
discovered, naturally occurring intracellular process that
regulates gene expression through the silencing of specific
mRNAs. Methods of harnessing this natural pathway are being
developed that allow the catalytic degradation of targeted
mRNAs using specifically designed complementary small
inhibitory RNAs (siRNA). siRNAs are being chemically modified
to acquire drug-like properties. Numerous recent high-profile
publications have provided proofs of concept that RNA
interference may be useful therapeutically. Much of the design
of these siRNAs can be accomplished bioinformatically, thus
potentially expediting drug discovery and opening new avenues
of therapy for many childhood diseases including uncommon
pediatric and orphan diseases. A discussion of the science
behind RNA interference will be followed by a presentation of
the potential practical issues in applying this technology to
disease. The program then describes two therapeutic programs
currently under way with applications to pediatric diseases. A
question-and-answer time will follow each discussion.
-
The Science of RNA Interference
John J. Rossi, Beckman Research Institute of City of Hope, Duarte, CA
-
RNA Interference and Its
Potential Applications for Controlling Disease
Judy Lieberman, CBR Institute for Biomedical Research and Harvard
Medical School, Boston, MA
-
Silencing the VEGF Pathway with
siRNAs and the Potential Application to Retinopathy of
Prematurity
Pamela Pavco, Sirna Therapeutics, Boulder, CO
-
siRNA as Therapy for Respiratory
Syncytial Virus
John P. DeVincenzo, University of Tennessee School of Medicine,
Memphis, TN
4:15pm–6:15pm
3840—Cell Biology of Lung Disease
PAS Platform Session
Room 2004, Moscone West
Chairs: Michael M. Grunstein and Craig M. Schramm
4:15pm–6:15pm
3850—Human Milk and Breastfeeding
PAS Poster Symposium
Room 3001, Moscone West
Chairs: Sheela R. Geraghty and Ardythe L. Morrow
4:15pm–6:15pm
3855—Infectious Diseases I
PAS/PIDS Platform Session
Room 3022-3024, Moscone West
Chairs: Sheldon L. Kaplan and David W. Kimberlin
4:15pm–6:15pm
3865—Neonatal Neurology—Neural Stem Cells
and Neurotrophins
PAS Poster Symposium
Room 3020, Moscone West
Chairs: Sandra E. Juul and Patrick S. McQuillen
4:15pm–6:15pm
3875—Neonatal Pulmonary Hypertension
PAS Poster Symposium
Room 2002, Moscone West
Chairs: Steven H. Abman and Bernard Thebaud
Monday, May 1
8:00am–10:00am
4110—Pediatric Fluids and Hyponatremia: Are
We Giving Too Much Water?
PAS/ASPN/LWPES Topic Symposium
Room 3007-3011, Moscone West
Chairs: John W. Foreman, Duke University Medical Center,
Durham, NC; and D. Michael Foulds, University of Texas Health
Science Center at San Antonio, San Antonio, TX
Target Audience: Nephrologists,
general pediatricians, emergency room doctors, intensivists,
hospitalists, endocrinologists and anyone who administers IV
maintenance fluids.
In the 1950s, Holiday and Segar
devised formulae for calculating intravenous maintenance
fluids for infants and children who were unable to drink.
These formulae have been taught and used now for over 40 years
and have generally stood the test of time. However, several
recent investigators have challenged these formulae and argued
that they put children at risk of hyponatremia. Since Holiday
and Segar devised these formulae, new information has arisen,
such as the concept of non-osmotic stimulation of ADH release
in sick children and our ability to measure ADH levels in
plasma on a routine basis. Arieff and Ayus were the first to
point out that children and women are at particular risk for
developing hyponatremic encephalopathy. Moritz and Ayus have
subsequently argued that hypotonic parenteral fluid should not
be used unless there are ongoing free water losses or
hypernatremia. In addition to this new clinical data,
Verkman’s group has exciting data identifying molecular
mechanisms of cerebral edema, including after water
intoxication. Dr. Arieff will review who is at risk and why.
Dr. Verkman’s group has developed data regarding mechanisms
of cerebral edema in experimental animals. Dr. Moritz will
describe the new concepts of maintenance fluids. Dr. Friedman
will defend the current practice. At the end there will be
time for an exchange between the speakers and the audience on
the right fluid to use in today’s children.
-
Hyponatremic Encephalopathy:
Special Risk Factors for Children and Women
Allen I. Arieff, University of California San Francisco, San Francisco,
CA
-
Aquaporin 4 and Cerebral Edema
Alan S. Verkman, University of California San Francisco, San Francisco,
CA
-
0.9% Sodium Chloride: The New
Approach to Maintenance Fluids in Pediatrics
Michael L. Moritz, Children's Hospital of Pittsburgh, Pittsburgh, PA
-
Maintenance Therapy: Tried and
True
Aaron L. Friedman, Brown Medical School, Hasbro Children's Hospital,
Providence, RI
Sponsored jointly by
the AAP Section on Nephrology, the American Society of
Pediatric Nephrology, the Lawson Wilkins Pediatric Endocrine
Society and the Pediatric Academic Societies
8:00am–10:00am
4132—Mechanisms of Neonatal Lung Injury
PAS Platform Session
Room 3003-3005, Moscone West
Chairs: Vineet Bhandari and Vasanth H.S. Kumar
8:00am–11:00am
4150—The Skinny on the Adipocyte
PAS/LWPES Mini Course
Room 3010-3012, Moscone West
Chairs: Silva A. Arslanian, University of Pittsburgh,
Pittsburgh, PA; and Robert H. Lustig, University of
California, San Francisco, CA
Target Audience: General
pediatricians, gastroenterologists, endocrinologists,
cardiologists, pulmonologists and adolescent medicine
specialists.
Over the past five years much has
been learned about the adipocyte. The ability of the adipocyte
to function as an endocrine gland, elaborating inflammatory
cytokines that result in free radical formation and premature
apoptosis of the beta cell, is a relatively new concept. This
mini course will comprehensively address many of the newest
concepts in adipocyte function and their impact on health and
disease. Further discussion will include new concepts on the
interactions of IGF-II and other peptides' interactions with
the adipocyte. Lastly, there will be a call for new approaches
to the pediatric obesity epidemic.
-
Adipose Tissue as an Endocrine
Organ
Susan Fried, University of Maryland School of Medicine, Baltimore VA
Medical Center, Baltimore, MD
-
Obesity and Inflammation
Christopher Hug, Whitehead Institute and Children's Hospital,
Cambridge, MA
-
Effects of GH, IGF-I and Insulin
Therapies on Adiposity
Zvi Laron, Schneider Children's Medical Center of Israel, Petah Tikva,
Isreal
-
Pathology and Sequelae of
Childhood Obesity in Adult Life
Dennis M. Styne, University of California, Davis Medical Center,
Sacramento, CA
Sponsored jointly by
the Lawson Wilkins Pediatric Endocrine Society and the
Pediatric Academic Societies
9:00am–12:00pm
4230—Recognizing Common Biostatistical
Errors: A Case-Based Approach
PAS Educational Workshop
Yerba Buena Gardens Salon 12, SF Marriott
Leader: Thomas Newman, San Francisco, CA; Co-leader: Susan
Fisher-Owens
Target Audience: Trainees,
fellows, junior faculty, mid-level faculty, and community
practitioners.
This workshop uses multiple real
examples from the pediatric literature to teach participants
how to be more discriminating consumers of statistics. Topics
to be covered include standard deviation vs. standard error of
the mean, commonly violated assumptions of statistical tests
including normality and independent sampling, between- vs.
within-groups comparisons, "type 3" (dumb or
careless) errors, odds ratios vs. relative risks, relative vs.
absolute effect sizes, effect size exaggeration, and multiple
comparisons. In the last part of the seminar, participants
will have the opportunity to test what they have learned on a
set of "unknown" examples.
Objectives:
– Choose the correct
statistical test.
– Recognize common errors in biostatistics.
– Avoid common errors in biostatistics.
Format: Case-based
question-and-answer period.
Designed to meet elements of the
core curriculum for pediatric fellowship subspecialty
training.
9:00am–12:00pm
4258—Practice-Based Research Networks
APA Special Interest Group
Room Pacific Suite C, SF Marriott
Chair: Robert Siegel, robertsiegel56@pol.net.
The Practice-Based Research
Network SIG is highlighted by presentations of recent work
done by the SIG members and works in progress. There will be
an open discussion of issues facing practice-based research as
an opportunity to discuss collaboration on projects and ways
of fostering practice-based research network development.
10:15am–12:15pm
4350—Mechanisms of Brain Injury
PAS Poster Symposium
Room 3020, Moscone West
Chairs: Maria Delivoria-Papadopoulos and Jeffrey M. Perlman
10:15am–12:15pm
4365—Nitric Oxide and Oxygen: A Marriage
Made in Heaven or Hell?
PAS Poster Symposium
Room 3003-3005, Moscone West
Chairs: Satyan
Lakshminrusimha and Leif D. Nelin
1:00pm–2:45pm
4500—March of Dimes Prize in Developmental
Biology Lecture
PAS Award
Room 3002-3008, Moscone West
Dr. Varshavsky is a pioneer in
the study of ubiquitin, a tiny protein that has a very big
job. Ubiquitin (from the Latin ubique meaning
"everywhere," the source of the word
"ubiquitous") is so named because it is essential to
nearly every major activity in the life cycle of cells,
including cell growth and division during embryo development,
DNA repair, programmed cell death, immune response, and the
nervous system. The ubiquitin system is the housekeeping
mechanism by which the cell maintains a proper and healthy
balance of proteins. Ubiquitin's role was unknown until the
1980s, when Dr. Varshavsky and colleagues elucidated it. This
discovery revolutionized our understanding of the control of
human cells, and ubiquitin quickly became one of the major
areas of study in genetics, developmental biology, cell
biology, and biochemistry. Today ubiquitin is a cornerstone of
medical research into the causes and treatments of birth
defects, neurodegenerative disease, infections, and cancer.
Dr. Varshavsky receives the 2006 March of Dimes Prize for
revealing and characterizing the biological significance of
the ubiquitin system in the regulation of living cells.
-
Regulation by Proteolysis: The
N-End Rule Pathway in Yeast and Mammals
Alexander Varshavsky, Smits Professor of Cell Biology, California
Institute of Technology, Pasadena, CA
Presented by the
March of Dimes Birth Defects Foundation
2:00pm–4:00pm
4580—Application of Translational Science
to Vaccinology: Varicella-Zoster Virus and Human
Papillomavirus
PAS/PIDS State of the Art Plenary
Room 3003-3005, Moscone West
Chairs: Ann M. Arvin, Stanford University School of Medicine,
Stanford, CA; and Anna-Barbara Moscicki, University of
California, San Francisco, CA
Target Audience: Infectious
disease specialists, primary care pediatricians, immunologists
and adolescent medicine physicians.
One of the major goals of
infectious diseases research is to understand the pathogenesis
of disease and to use this knowledge to prevent the illness
through vaccination. An understanding of varicella
pathogenesis led to the development of a successful vaccine,
and further insights into long-term success of the vaccine and
the future of varicella immunization are emerging. A more
recent success story is that of human papillomavirus, in which
basic science studies of pathogenesis led to the development
of vaccines based on virus-like particles. These two examples
serve as models of the success of translational science in
combating infectious diseases.
-
MMRV and the Future of
Immunization Against Varicella-Zoster Virus
Anne A. Gershon, Columbia University, New York, NY
-
Pathogenesis of Human
Papillomavirus Infections
Anna-Barbara Moscicki, University of California, San Francisco, CA
-
Development of Virus-like
Particles for Immunization Against Human Papillomavirus
John T. Schiller, National Cancer Institute, National Institutes of
Health, Bethesda, MD
Sponsored jointly by
the Pediatric Infectious Diseases Society and the Pediatric
Academic Societies
3:00pm–4:00pm
4600A—LWPES Trans-Pacific Lecture
LWPES Award
Room 3002-3008, Moscone West
Chair: Mark Sperling, Children's Hospital of Pittsburgh,
Pittsburgh, PA
Target Audience: Geneticists,
endocrinologists and molecular biologists.
This new lecture recognizes one
outstanding scientist from the Pacific Rim. This talk will
illuminate congenital adrenal disorders with particular focus
on the relationship between newborn screening and molecular
mechanisms.
-
Congenital Adrenal Disorders:
From Newborn Screening to Molecular Mechanism
Kenji Fujieda, Asahikawa Medical College, Asahikawa, Japan
3:00pm–5:00pm
4610—Cardiac Stem Cell Biology and
Therapeutics
PAS Topic Symposium
Room 3001, Moscone West
Chair: Harold S. Bernstein, University of California, San
Francisco, CA
Target Audience: Physicians,
scientists and trainees with interest in pediatric cardiology,
stem cell biology and reparative medicine.
This topic symposium is directed
towards educating interested members about the state of the
art in cardiac stem cell research, both the underlying biology
and initial attempts in animals and humans at cardiac
regenerative therapy. The discussion will range from
hematopoietic stem cells to cardioblasts, as well as to how
one assesses the results of stem cell infusion trials.
-
Recent History of Secondary
Cardiac Myogenesis
Harold S. Bernstein, University of California, San Francisco, CA
-
Mesenchymal Stem-Cell-Based
Cardiac Regeneration
Andrew Boyle, Johns Hopkins University, Baltimore, MD
-
Cardioblasts
Kenneth R. Chien, Mass General Hospital, Harvard Medical School,
Boston, MA
-
Functional Assessment of Myogenic
Stem Cells and Cardiomyocytes for Cardiac Cell Therapy
Loren J. Field, Indiana University School of Medicine, Indianapolis, IN
3:00pm–5:00pm
4630A—Molecular Control of the Formation of
the Renal Collecting System
ASPN Symposium
Room 3010-3012, Moscone West
Chairs: Lisa M. Satlin, Mount Sinai School of Medicine, New
York, NY; and Norman D. Rosenblum, The Hospital for Sick
Children, Toronto, Ontario, Canada
Target Audience: Clinicians,
clinician-scientists and scientists interested in development,
nephrology and human disease involving the urinary tract.
Kidney development depends on
embryonic processes which pattern the collecting system
consisting of the ureter, renal pelvis and calyces, and
collecting ducts. Disruption of these processes in humans
results in a spectrum of anomalies including vesicoureteral
reflux, malformations of the pelvis and calyces, a decreased
number of collecting ducts and cystic malformation of these
ducts. Presentations in this symposium will highlight newly
elucidated genetic mechanisms that control different aspects
of collecting system formation. Analyses of genetic mouse
models are demonstrating a critical role for Fibroblast Growth
Factor Receptors in controlling growth and branching of the
ureteric buds that give rise to collecting ducts. Recent
evidence reveals a critical role for HNF1b, a transcription
factor, in controlling collecting duct terminal
differentiation and cyst formation via mechanisms involving
PKDH1, the gene mutated in human autosomal recessive kidney
disease. New genetic approaches are being harnessed to define
molecular mechanisms that control formation of the
vesico-ureteric orifice. Together, these discoveries and
approaches are providing novel molecular insights into
developmental nephrology and human disease.
-
Overview
Norman D. Rosenblum, Professor of Paediatrics and Canada Research Chair
in Developmental Nephrology, Division of Nephrology &
Program in Developmental Biology, The Hospital for Sick
Children, Toronto, Ontario, Canada
-
Role of Fibroblast Growth Factor
Receptors in Kidney Development
Carlton M. Bates, Children's Hospital of Columbus, Columbus, OH
-
Transcriptional Control of the
Bradykinin B2 Receptor
Samir S. El-Dahr, Tulane University School of Medicine, New Orleans, LA
-
Roles of HNF-1beta in Kidney
Development and Disease
Peter Igarashi, University of Texas Southwestern School of Medicine,
Dallas, TX
-
Genes and VUR
Ali Gharavi, Columbia University, New York, NY
3:00pm–5:00pm
4650—CPCCRN: The NICHD Collaborative
Pediatric Critical Care Research Network
PAS Educational Workshop
Room 2004, Moscone West
Leader: Carol Nicholson, Bethesda, MD; Co-leader: Douglas
Willson
Target Audience: Trainees,
fellows, junior faculty, mid-level faculty, and community
practitioners.
There are 10,500 critically ill
and injured children admitted each year to the PICUs covered
in the Network. We would welcome the opportunity to interact
with all of the pediatric subspecialties in the context of
PAS. Our work and our research are interwoven with each
pediatric subspecialty as well as with pediatric surgery and
surgical subspecialties.
Objectives:
– How cutting edge informatics
can be used for collaborative pediatric research.
– Understand the Network structure, vision and function, in
a multidisciplinary field.
– Learn about new approaches to nosocomial infection during
critical illness.
Format: An introductory
presentation of Network structure and function, with emphasis
on innovation in collaborative research will begin the
workshop. A series of pediatric critical care scientists will
present some of the newer work being undertaken in the Network
in informatics, sedation, immunology, infectious disease, and
outcomes after pediatric critical illness and injury.
Here is a specific overview, with
each speaker being available for questions, and audience
interaction.
-
The CPCCRN: Overview
Douglas Willson, MD
-
Functional Disability Outcomes
in Pediatric Critical Care
Murray Pollack, MD
-
Toward Science-Based
Guidelines for Sedation and Mechanical Ventilation in
Pediatric Critical Care
Christopher Newth and/or Sunny Anand
-
Nosocomial Sepsis and
Lymphocytic Apoptosis: GI Prophylaxis, Glutamine and Zinc
in Pediatric Critical Illness
Joseph Carcillo, MD
3:00pm–5:00pm
4656—Newer Mouse Technologies Targeted at
Dissecting Mechanisms
PAS Educational Workshop
Yerba Buena Gardens Salon 5, SF Marriott
Leader: Parviz Minoo, Los Angeles, CA; Co-leaders: Francesco
J. DeMayo, Ed Morrisey
Target Audience: Clinician and
basic scientists, including new investigators, fellows and
postdoctoral fellows.
The use of transgenic mice has
become commonplace in analysis of developmental and
physiological pathways. This technology has contributed
significantly to and continues to provide critical information
for understanding fundamental biological processes and their
clinical implications in health and disease. The objective of
this workshop is to discuss and review state-of-the-art
approaches using transgenic technologies in mice, including
genetically engineering the airways for the regulated ablation
and expression of genes, as well as distinct examples from the
speakers’ works on lung development and gene regulation.
3:00pm–5:00pm
4690—Sepsis: Pathogenesis and Outcomes
PAS Platform Session
Room 3000, Moscone West
Chairs: John H. Arnold and Joseph A. Carcillo
Tuesday, May 2
8:00am–10:00am
5110A—Inflammation in Uremic
Pathophysiology
ASPN Symposium
Room 3010-3012, Moscone West
Chairs: H. William Schnaper, Northwestern University Feinberg
School of Medicine, Chicago, IL; and Robert H.K. Mak, Oregon
Health and Science University, Portland, OR
Target Audience: Pediatric
nephrologists and fellows, basic scientists, pathologists and
immunologists.
Recent evidence has strongly
suggested that the manifestations of uremia are caused in
large part by activation of inflammatory pathways. This
symposium will review the syndromic events that can be
attributed to uremic inflammation and include oxidant injury,
cytokine production and its end-organ effects on the body
tissues.
-
Oxidant Injury in ESRD
Jonathan Himmelfarb, Maine Medical Center, Portland, ME
-
MIA (Malnutrition, Inflammation,
Atherosclerosis) Syndrome in ESRD
Joel D. Kopple, Harbor-UCLA Medical Center, David Geffen School of
Medicine at UCLA, UCLA School of Public Health, Torrance,
CA
-
Leptin and Melanocortin Signaling
in Chronic Kidney Disease
Robert H.K. Mak, Oregon Health and Science University, Portland, OR
-
Molecular Pathophysiology of
Muscle Catabolism in Uremia: Effect of Acidosis and
Inflammation
William E. Mitch, Baylor College of Medicine, Houston, TX
Supported by an unrestricted educational grant from Abbott
8:00am–10:00am
5150—Cardiology—Genetics and Development
PAS Platform Session
Room 3000, Moscone West
Chairs: H. Scott Baldwin and Marlene Rabinovitch
Includes
-
SPR Student Research Award:
Mutations in JPH2-Encoded Junctophilin 2 as a Novel
Pathogenic Mechanism in Hypertrophic Cardiomyopathy
Karin Batalden, Mayo Medical School, College of Medicine, Rochester, MN
8:00am–10:00am
5168—Oxidants, Antioxidants and the Battles
They Wage
PAS Poster Symposium
Room 3014, Moscone West
Chairs: Jonathan M. Davis and Charles V. Smith
10:15am–11:45am
5405—Newborn Screening: The Coming
Revolution
PAS State of the Art Plenary
Room 3001, Moscone West
Chair: Alex R. Kemper, University of Michigan, Ann Arbor, MI
Target Audience: General
pediatricians, subspecialists involved with newborn screening,
for including neonatologists, endocrinologists, hematologists
and geneticists.
Newborn screening has resulted in
dramatic improvements in the morbidity and mortality of
inherited disorders. Recent laboratory developments have
dramatically increased the number of conditions that can be
detected in early infancy. Expanding the list of conditions
has lead to unique challenges for pediatric practices and
public health systems. This symposium will explore these new
and emerging challenges.
-
Overview
Alex R. Kemper, University of Michigan, Ann Arbor, MI
-
New Technologies for Newborn
Screening
Edward R.B. McCabe, David Geffen School of Medicine at UCLA, Mattel
Children's Hospital, Los Angeles, CA
-
Meeting the Needs for
Confirmation, Counseling and Treatment
R. Rodney Howell, Miller School of Medicine at the University of Miami,
Miami, FL
-
"Treatment" Versus
"Benefit" in Evaluating the Desirability of
Expanded Newborn Screening
Don Bailey, University of North Carolina at Chapel Hill, Chapel Hill,
NC
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Ethical Issues That Must Be
Addressed in an Expanded Newborn Screening Program
Ellen Wright Clayton, Vanderbilt University, Nashville, TN
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Summary Comments
Michele Puryear, Maternal and Child Health Bureau, Health Resources
& Services Administration, Rockville, MD
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Discussion
10:15am–12:15pm
5435—Endocrinology and Diabetes—Basic
Research
PAS/LWPES Platform Session
Room 3007-3011, Moscone West
Chairs: Stephen E. Gitelman and Anna Spagnoli
12:45pm–3:45pm
5650—Recent Advances in Understanding and
Treating Neonatal Chronic Lung Disease
PAS Hot Topic
Room 3002-3008, Moscone West
Chairs: Richard D. Bland, Stanford University School of
Medicine, Stanford, CA; and Bernard Thebaud, University of
Alberta, Edmonton, AB, Canada
Target Audience: Neonatologists
involved in the care of premature infants, pulmonologists and
general pediatricians who care for children suffering the ill
effects of neonatal chronic lung disease and clinician
scientists with a research interest in normal and disordered
development of the lung and its circulation.
A symposium to honor Dr. William
Northway and Dr. Jacqueline Coalson for their seminal
discoveries of bronchopulmonary dysplasia (BPD) in human
babies and premature baboons, as we approach the 40th
anniversary of Dr. Northway’s initial description of BPD and
the 25th anniversary of Dr. Coalson’s initial papers on the
Southwest Foundation’s authentic model of BPD in non-human
primates. The program will focus on recent advances in the
basic biology of lung development, its dysregulation in BPD
and implications for novel treatment strategies. The intent is
to improve understanding of the mechanisms that regulate the
formation of alveoli, pulmonary capillaries and extracellular
matrix components in the developing lung, with consideration
of some of the adverse conditions that may contribute to
impaired lung growth and development in BPD. This knowledge
will provide rationale for introducing novel strategies to
help treat or prevent neonatal chronic lung disease.
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Introduction
Alan H. Jobe, Cincinnati Children's Hospital Medical Center,
Cincinnati, OH
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BPD in Babies: An Historical
Perspective
William H. Northway, Lucile Salter Packard Hospital, Palo Alto, CA
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BPD in Baby Baboons: An Evolving
Saga
Jacqueline J. Coalson, UT Health Sciences Center at San Antonio, San
Antonio, TX
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Lung Septation and Its
Dysregulation in BPD
Jacques R. Bourbon, Universite Paris XII - Faculte de Medecine, Creteil,
France
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Lung Angiogenesis and Its
Dysregulation in BPD
Steven H. Abman, University of Colorado School of Medicine, The
Children’s Hospital, Denver, CO
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Lung Elastin and Its
Dysregulation in BPD
Richard D. Bland, Stanford University School of Medicine, Stanford, CA
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Novel Ways To Treat or Prevent
BPD
Bernard Thebaud, University of Alberta, Edmonton, Alberta, Canada
1:45pm–3:45pm
5720—Autosomal Recessive Polycystic Kidney
Disease (ARPKD): New Insights and Clinical Perspectives
PAS/ASPN/NASPGHAN Topic Symposium
Room 3010-3012, Moscone West
Chairs: Philip Rosenthal, University of California, San
Francisco, San Francisco, CA; and Lisa M. Guay-Woodford,
University of Alabama at Birmingham, Birmingham, AL
Target Audience: Pediatricians,
pediatric nephrologists, pediatric gastroenterologists,
neonatalogists and developmental biologists.
ARPKD is a developmental disorder
of the kidneys and liver caused by mutations in the PKHD1
gene. Fibrocystin/polyductin, the protein encoded by PKHD1, is
expressed on the primary cilia of renal and bile duct
epithelial cells. Several lines of evidence indicate that the
PKHD1 transcriptional profile is complex with extensive splice
variants. While the function of these transcripts and the
polypeptides that they encode is not well understood, these
proteins seem to play critical roles in establishing and
maintaining the tubular architecture. This symposium will
discuss the complex transcriptional profile of PKHD1 and the
role of these gene products in renal as well as biliary
epithelia. Given that ARPKD has a high perinatal mortality due
to oligohydramnios and resultant respiratory insufficiency,
current concepts regarding the interplay between the
developing kidney, the placenta and the developing lung will
be discussed. Finally, a clinical perspective based on the
on-going NHGRI-sponsored natural history study will focus on
ARPKD-associated morbidities and disease progression.
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Transcriptional Complexity of
PKHD1: Implications for Development and Disease
Pathogenesis
Gregory G. Germino, Johns Hopkins University, Baltimore, MD
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Pathobiology of Biliary Epithelia
in ARPKD
Tatyana Masyuk, Mayo Clinic College of Medicine, Rochester, MN
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Oligohydramnios: Current Concepts
and Implications for Pulmonary Development
F. Sessions Cole, Washington University School of Medicine, St. Louis
Children's Hospital, St. Louis, MO
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Report on the NIH ARPKD/CHF
Natural History Study
Meral Gunay-Aygun, National Human Genome Research Institute (NHGRI),
Bethesda, MD
Sponsored jointly by
the American Society of Pediatric Nephrology; the North
American Society for Pediatric Gastroenterology, Hepatology
and Nutrition; and the Pediatric Academic Societies
1:45pm–3:45pm
5755—Neonatal Brain Injury: How Can We Do
More Good Than Harm?
PAS Platform Session
Room 3003-3005, Moscone West
Chairs: Sylvain Chemtob and Augusto Sola
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