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Last
updated February 10, 2005
Friday, May 13
10:30am–4:00pm
3200A—ASPHO
Board of Trustees Meeting
ASPHO Executive Board Meeting
2:00pm–5:00pm
3400A—ASPHO
Committee Meeting #1
ASPHO Committee
2:00pm–5:00pm
3401A—ASPHO
Committee Meeting #2
ASPHO Committee
5:00pm–6:00pm
3500A—Hemorrhagic
Disorders
ASPHO Workshop
Co-chairs:
Pedro A. de Alarcon, St. Jude’s Research Hospital,
Memphis, TN; and Marilyn Manco-Johnson, Mountain States
Hemophilia Treatment Center, Aurora, CO
Information not yet available.
5:00pm–6:00pm
3501A—Pediatric
Cancer
ASPHO Workshop
Co-chairs:
Peter C. Adamson, Children’s Hospital of Philadelphia,
Philadelphia, PA; and Jeffrey A. Moscow, University of
Kentucky Medical Center, Lexington, KY
Information not yet available.
5:00pm–6:00pm
3502A—ASPHO
Workshop
ASPHO Workshop
5:00pm–6:00pm
3510A—ASPHO
Committee Meeting #3
ASPHO Committee
5:00pm–6:00pm
3511A—ASPHO
Committee Meeting #4
ASPHO Committee
6:00pm–7:00pm
3600A—ASPHO
Leadership Reception
ASPHO Reception
7:00pm–9:00pm
3700A—ASPHO
Corporate Forum Dinner — Enzon Pharmaceuticals
ASPHO Dinner
7:00pm–9:00pm
3701A—ASPHO
Corporate Forum Dinner — ZLB Behring
ASPHO Dinner
7:00pm–9:00pm
3750A—ASPHO
Training Program Director's Dinner Meeting
ASPHO Alliance Society
Saturday, MAY 14
7:00am–8:00am
4040A—Practice
Management: Survival Strategies for Pediatric
Hematology/Oncology Programs: Tapping Sources of
Non-Clinical Revenue
ASPHO Workshop
Co-chairs:
Timothy C. Griffin, MD, Cook Children’s Medical Center,
Fort Worth, TX; and Eric J. Werner, Children’s Hospital
of the Kings Daughters, Norfolk, VA
Most pediatric hematology/oncology programs in the
U.S. face ongoing economic pressures. In order to survive,
program directors must be creative in developing sources
of revenue outside of traditional reimbursement
structures. This workshop will provide an opportunity to
learn more about two potential approaches to develop new
sources of revenue. One program director’s recent
experiences with an expanded philanthropic development
effort will be discussed, and participants will gain
insights into the role of hematologist/oncologists in an
institution’s philanthropic development team.
Participants will also learn how participation in a 340b
program can help provide resources for hemophilia care.
7:00am–8:00am
4041A—Thrombosis:
Cancer and Thrombosis in Children
ASPHO Workshop
Co-chairs:
Patricia Massicotte, Edmonton, Alberta, Canada; and Lori
Luchtman-Jones, Washington University, St. Louis, MO
Children are surviving cancer due to improved
diagnostic, treatment and follow-up regimens.
Unfortunately, thrombosis continues to impact the
morbidity and mortality of pediatric cancer. This session
will highlight the developmental hemostatic differences
between children and adults and review the known
mechanisms of thrombosis in malignancy. A discussion about
the diagnosis and management of thrombosis in malignanancy
in adults and children will conclude the session.
8:00am–11:00am
4103—Neonatal
Immunology—Relevance to the Clinician
PAS Mini Course
Chair:
E. Richard Stiehm, Mattel Children's Hospital at UCLA, Los
Angeles, CA
Developmental immunology, immunotherapy for the
neonate with infection, diagnosis of immunodeficiency and
relevance to the development of allergy will be discussed.
Target Audience: Pediatricians who care for newborns,
including neonatologists dealing with premature, high-risk
newborns.
Overview
E.
Richard Stiehm, Mattel Children's Hospital at UCLA, Los
Angeles, CA
Transient and Congenital Immunodeficiencies of the
Newborn: Recognition and Management
David
B. Lewis, Stanford University Medical Center, Stanford, CA
Newborn Immunity as a Predictor for the Development
of Wheezing and Allergy
James
E. Gern, University of Wisconsin, Madison, WI
Immunologic Intervention in the Newborn: Relevance to
Newborn Infections
Harry
R. Hill, University of Utah School of Medicine, Salt Lake
City, UT
8:15am–10:15am
4200A—Recent
Advances in Radiotherapy for Pediatric Cancers
ASPHO Symposium
Chair:
Thomas E. Merchant, St. Jude Children's Research Hospital,
Memphis, TN
New technological advances have expanded the role of
radiotherapy and choices available for treating pediatric
tumors. The promise of safer and more effective therapy is
being realized through the use of such techniques as
intensity modulated radiotherapy and proton beams.
Appropriate scenarios for these different modalities are
not well understood by most pediatric oncologists. This
symposium will review recent advances in radiotherapy,
provide insight into their use and describe up-to-date
clinical trial data. After attending this session,
attendees will be better prepared to discuss radiotherapy
options with patients and make appropriate referrals.
Advances in Radiotherapy for Pediatric CNS Tumors
Thomas
E. Merchant
IMRT for Pediatric Solid/Musculoskeletal Tumors
Matthew
J. Krasin
The Use of Proton Beam Therapy in Pediatric Cancer
Torunn
Yock
10:30am–12:30pm
4401—Hematology/Oncology
I
PAS/ASPHO Original Science
Abstracts - Platform Session
10:30am–12:30pm
4450A—ASPHO
Practice Manager's Forum
ASPHO Alliance Society
New this year—the Practice Manager’s Forum is for
pediatric hematology/oncology practice managers. This
two-hour session presented by the ASPHO Practice Committee
will focus on issues of common interest, including
billing, coding and reimbursement; and contracting with
payers. Physician leaders and their practice managers are
encouraged to attend this session together to partner in
developing and implementing practice management policies,
procedures and strategic planning.
12:30pm–2:30pm
4590A—ASPHO
Corporate Forum Luncheon—Genzyme Oncology
ASPHO Luncheon
12:30pm–2:30pm
4591A—ASPHO
Corporate Forum Luncheon—Novartis Oncology
ASPHO Luncheon
1:00pm–2:00pm
4595A—ASPHO
Committee Meeting #5
ASPHO Committee.
1:00pm–2:00pm
4596A—ASPHO
Committee Meeting #6
ASPHO Committee
.
1:00pm–2:00pm
4597A—ASPHO
Committee Meeting #7
ASPHO Committee
1:00pm–2:00pm
4598A—ASPHO
Committee Meeting #8
ASPHO Committee
2:45pm–4:45pm
4700A—Ewing's
Sarcoma: Update on Biology, Therapy
ASPHO Symposium
Chair:
Jeffrey A. Toretsky, Lombardi Comprehensive Cancer Center
and Pediatrics, Georgetown University, Washington, DC
Therapy for the Ewing’s sarcoma family of tumors (ESFT)
including Ewing’s sarcoma, peripheral primitive
neuroectodermal tumor, Askin’s tumor and
neuroepithelioma has improved significantly since 1921
when the diffuse endothelioma of bone was described by Dr.
James Ewing. The development of “5-drug” therapy in
the 1980s raised the 5-year survival rate of patients with
localized disease to 70%. Survival for metastatic patients
remains poor despite continuing to intensify therapy to
the level of bone marrow transplantation.
Bone marrow transplantation has become widely
utilized in treating patients with metastatic ESFT,
despite controversy about the added benefits of high-dose
therapy for this subset of patients. A debate between
experts representing both supporting and opposing views of
transplant will be held. Questions and lively discussion
from conference participants is anticipated.
To improve overall survival for all ESFT patients,
new agents are needed. The final section of the symposia
will present unique opportunities to develop highly
specific therapy for ESFT patients. The biology of ESFT
has advanced significantly in the past 5 years. These
biologic advances could lead to highly specific therapies
that would potentially improve survival and reduce
treatment morbidity. These recent gains as well as future
experimental directions will be discussed.
Historical Background on James Ewing and the Naming
of the Tumor
Speaker
To Be Determined
The Role of Transplant/Megatherapy in Megastatic EFST:
A Debate
Paul
A. Meyers
The Role of Transplant/Megatherapy in Megastatic EFST:
A Debate
Stefan
Burdach
Central Biologic Role of EW-FLI1 in ESFT: What Is an
EWS-FLI1 and How will it Help My Patients?
Jeffrey
A. Toretsky
Central Biologic Role of EW-FLI1 in ESFT: Molecular
Targets for Therapeutic Development in ESFT
Speaker
To Be Determined.
3:15pm–5:15pm
4848—Neonatal
Hematology–Immunology
PAS Original Science Abstracts -
Poster Symposium
5:15pm–7:15pm
Poster
Session I
PAS Original Science Abstracts -
Poster Session
Hematology and Oncology:
4920—Neonatal Hematology
4921—Coagulation and Hemophilia
4922—Miscellaneous Bone Marrow
4923—Molecular Mechanisms/Changes
4924—CNS Tumors
4925—Osteogenic Sarcoma and Rare Tumors
4926—Neuroblastoma and Ewings
Sunday, MAY 15
7:00am–8:00am
5070A—Bone
Marrow Failure
ASPHO Workshop
Chair:
Adriana Vlachos, Schneider Children’s Hospital, New Hyde
Park, NY
Information not yet available.
7:00am–8:00am
5071A—Hemoglobinopathies
ASPHO Workshop
Co-chairs:
Russell E. Ware, St. Jude’s Research Hospital, Memphis,
TN; and Matthew M. Heeney, Boston Children’s Hospital,
Boston, MA
Information not yet available.
7:00am–8:00am
5072A—Young
Investigators
ASPHO Workshop
Co-chairs:
Judith Margolin, Baylor College of Medicine, Houston, TX;
and Kathleen Sakamoto, University of California Los
Angeles, Los Angeles, CA
This workshop will provide an opportunity for
hematology/oncology fellows and junior faculty to learn
about: (1) NIH grant review and submission process; (2)
preparation and submission of manuscripts to scientific
journals; and (3) important factors in making career
decisions. There will be time for informal discussion
related to issues of mutual interest.
8:15am–9:30am
5290—Hematology/Oncology
II
PAS/ASPHO Original Science
Abstracts - Platform Session
9:45am–11:45am
5300A—Autoimmune
Cytopenias
ASPHO Symposium
Chair:
Naynesh Kamani, Children's National Medical Center,
Washington, DC
Over the past decade, a number of genetic syndromes
have been characterized where autoimmune cytopenias
constitute a frequent clinical presentation. The
identification of the gene defects in these syndromes has
lead to a better understanding of the pathogenesis of
autoimmune manifestations in these children and will
eventually lead to the development of more specific
therapies for these cytopenias. While corticosteroids
continue to be the mainstay of therapy for autoimmune
cytopenias, a number of new immune suppressive agents have
been increasingly used in recent years in refractory
patients. Monoclonal antibodies (rituximab) are
increasingly being used to avoid the need for splenectomy
in these patients. This symposium will address the new
genetic syndromes, the pathogenesis of the autoimmune
cytopenias in these syndromes, the interpretation of
diagnostic tests in the autoimmune cytopenias and the
results achieved with novel therapies, the rationale for
their use and adverse effects of these therapies.
Introduction
Naynesh
R. Kamani
Diagnostic Tests and Pitfalls in the Interpretation
of Diagnostic Testing in Autoimmune Cytopenias
Naomi
L. C. Luban
Novel Therapies for Refractory Autoimmune Cytopenias:
Results, Adverse Effects and Mode of Action
James
B. Bussel
New Genetic Syndromes Associated with Autoimmune
Cytopenias
Naynesh
R. Kamani
11:45am–1:45pm
Poster
Session II
PAS Original Science Abstracts -
Poster Session
Hematology and Oncology:
5440—Thalassemia
5441—Sickle Cell Diseases
5442—RBC
5443—Leukemia/Lymphoma
5444—Infections
5445—Supportive Care
5446—Late Effects of Cancer Treatment
5447—Fertility/Supportive Care
5448—Palliative Care
1:45pm–3:45pm
5500—What's
New in Pediatric Thrombosis
PAS/ASPHO Topic Symposium
Chairs:
Guy Young, Children's Hospital of Orange County and Mattel
Children's Hospital at UCLA, Orange, CA; and Marilyn Manco-Johnson,
Mountain States Hemophilia Treatment Center and University
of Colorado, Aurora, CO
Thrombosis in children is an increasingly recognized
phenomenon in pediatrics largely due to major advances in
the care of critically ill patients and the increased use
of intravascular catheters. As a result of the increased
frequency of thrombosis and increased collaboration among
clinical researchers, there is a significant amount of new
data emerging in the field of pediatric thrombosis. These
data include new knowledge regarding genetic risk factors,
outcome predictors and the use of novel anticoagulants.
This session will include a detailed discussion of genetic
risk factors for thrombosis, including which risk factors
to test for in which patients and how to apply the results
of testing into decision making. A discussion on
traditional versus new anticoagulants will assess the
current and future role of these novel agents in the care
of pediatric patients. Finally, there will be a discussion
on outcome predictors for deep vein thrombosis and how
they influence initial treatment choices such as
anticoagulation and thrombolysis.
Target Audience: Physicians involved in the diagnosis
and management of patients with thromboembolic
complications.
Introduction
Marilyn
J. Manco-Johnson
Genetic Risk Factors for Thrombosis
Ulrike
Nowak-Gottl, University of Muenster, Muenster, Germany
Novel Anticoagulants
Guy
Young, Children's Hospital of Orange County, Mattel
Children's Hospital at UCLA, Orange County, CA
Outcome Predictors in Deep Vein Thrombosis
Marilyn
J. Manco-Johnson
Sponsored jointly by the American Society
of Pediatric Hematology/Oncology and the Pediatric
Academic Societies
4:00pm–6:00pm
5600A—4th
Annual Frank A. Oski Memorial Lecture and ASPHO Awards
ASPHO Award
Young Investigator Awards and Oral Abstract
Presentations
Frank A. Oski Memorial Lecture:
The Emerging Genetics of T-Cell ALL in Children and
Adolescents
A.
Thomas Look, Awardee
Distinguished Career Award
J.
Bruce Beckwith, Awardee
4:15pm–5:45pm
5703—Stem
Cell Therapies: What's on the Horizon for Pediatrics and
Pediatric Diseases
PAS State of the Art Plenary
Chair:
Stuart Orkin, Dana Farber Cancer Institute, Boston, MA
Stem cell-based approaches hold great promise for
treating many of the tissue degenerative disorders that
afflict our aging population. This symposium will explore
the role of stem cell therapies in pediatric disorders due
to inborn errors of metabolism and other single-gene
defects. Furthermore, the symposium will discuss the
implications of new data indicating that fetal cells in
the maternal circulation can participate in maternal wound
repair, implying that the fetus may be able to “treat”
its mother.
Target Audience: Broad appeal for scientists and
clinicians interested in new therapeutic approaches based
on stem cell biology.
Stem Cell Therapy in Lysosomal Storage Disorders
Susan
L. Staba, University of Florida, Gainesville, FL
Liver Repopulation with Stem Cells
Markus
Grompe, Oregon Health & Science University, Portland,
OR
Pregnancy-Associated Stem Cells: Does the Fetus
"Treat" Its Mother?
Diana
W. Bianchi, Tufts University School of Medicine, Floating
Hospital for Children, Boston, MA
Summation and Perspectives
Stuart
H. Orkin, Dana Farber Cancer Institute, Boston, MA
6:00pm–6:30pm
5850A—ASPHO
Business Meeting
ASPHO Business Meeting
6:30pm–7:00pm
5900A—ASPHO
Distinguished Career Awards Reception
ASPHO Reception
7:00pm–9:00pm
5950A—ASPHO
Corporate Forum Dinner—Wyeth
ASPHO Dinner
Monday, MAY 16
7:00am–8:00am
6080A—Cancer
Survivorship
ASPHO Workshop
Chair:
Debra Friedman, Children's Hospital and Medical Center,
Seattle, WA
Information not yet available.
7:00am–8:00am
6081A—Histiocytosis
ASPHO Workshop
Co-chairs:
James A. Whitlock, Vanderbilt University Medical Center,
Nashville, TN; and Alexandra Filipovitch, Cincinnati
Children’s Hospital Medical Center, Cincinnati, OH
This session will provide state of the art
information regarding the biology, diagnosis, evaluation
and management of children with Langerhans cell
histiocytosis and hemophagocytic lymphohistiocytosis,
including updates from recently completed and ongoing
international clinical trials for these disorders.
7:00am–8:00am
6082A—Immune
Thrombocytopenia
ASPHO Workshop
Co-chairs:
Victor S. Blanchette, Hospital for Sick Children, Toronto,
Canada; and Thomas Kuehne, University Children’s
Hospital, Basel, Switzerland
Information not yet available.
8:15am–10:15am
6150A—Gene
Therapy—A Primer for the Clinician
ASPHO Symposium
Chair:
Jakub Tolar, University of Minnesota, Minneapolis, MN
Viral and non-viral vectors have been utilized to
ferry novel DNA into target cells and raise the potential
of altering congenital gene lesions in a therapeutic
manner. Work in this intensely exciting field must come
with caution, however, as these approaches carry some
nontrivial dangers for the patients, such as insertional
mutagenesis and immune deregulation. The program will
begin with a review of gene therapy approaches and a
summary of gene therapy clinical trials to date, including
the severe combined immunodeficiency (SCID) clinical
trial. The program will follow with a presentation of the
most recent data on gene therapy for hemophilia. The
symposium will conclude with an overview of immune
implications when inserting foreign genes in the human
genome and rationale for the upcoming clinical trial of
gene therapy for Fanconi Anemia. While gene therapy has
yielded some notable successes and holds considerable
promise, one should walk away from the session with a
realistic overview of the possibilities and limitations of
gene therapy for childhood diseases.
Introduction
Jakub
Tolar
Review of Gene Therapy Clinical Trials, Including
SCID
Christof
von Kalle
Gene Therapy for Hemophilia
Katherine
A. High
Immunology of Gene Transfer and Gene Therapy for
Fanconi Anemia
Patrick
F. Kelly
10:30am–12:30pm
6375—Hematology/Oncology
III
PAS/ASPHO Original Science
Abstracts - Platform Session
12:00pm–1:00pm
6400A—XXII
Annual Audrey K. Brown Kernicterus Symposium
Club
Chairs:
David K. Stevenson, Stanford University School of
Medicine, Stanford, CA; William J. Cashore, Women and
Infants Hospital of Rhode Island, Providence, RI; and
Vinod K. Bhutani, Stanford University School of Medicine,
Stanford, CA
Dynamics of Neuronal and Glial Cell Response to
Unconjugated Bilirubin
Dora
Brites, Centro de Patogenese Molecular, University of
Lisbon, Lisbon, Portugal
G-6-PD Deficiency-Associated Neonatal
Hyperbilirubinemia: A Hidden Risk for Kernicterus in North
America
(This talk is dedicated to the memory of Marguerite
Herschel, MD)
Michael
Kaplan, Shaare Zedek Medical Center, Associate Professor
of Pediatrics, Faculty of Medicine, The Hebrew University,
Jerusalem, Israel
Contact:
David K. Stevenson, M.D.
Stanford University School of Medicine
Phone: 650-724-6966
Email: dstevenson@stanford.edu
This talk is dedicated to the memory of
Marguerite Herschel, MD.
Supported by an educational grant from
Natus Medical, Inc.
12:30pm–2:30pm
6430A—ASPHO
Corporate Forum Luncheon—Novo Nordisk Pharmaceuticals
ASPHO Luncheon
12:30pm–2:30pm
6431A—ASPHO
Corporate Forum Luncheon—TBD
ASPHO Luncheon
1:00pm–2:45pm
6500—The
March of Dimes Prize in Developmental Biology Lectures
PAS Award
Mario Capecchi, University of Utah, Salt Lake City
Oliver Smithies, University of North Carolina at Chapel
Hill.
Dr. Capecchi and Dr. Smithies were chosen to receive
the Prize for pioneering the development of gene targeting
in mice as a means of determining how genes function.
Their seminal work on "knockouts" revolutionized
not only the use of the mouse as a model system, but the
study of human disease and development as well. Gene
targeting is now practiced routinely by thousands of
scientists all over the world, enabling them to address
the most complex and critical biological problems,
including the causes and treatment of birth defects and
many other disorders.
Presented by the March of Dimes Birth
Defects Foundation
2:00pm–4:00pm
6600—Virus–Host
Interactions: Mechanisms Underlying Persistent Viral
Infections
PAS/PIDS Topic Symposium
Chairs:
Kenneth A. Alexander, Duke University Medical Center,
Durham, NC; and John Vanchiere, Baylor College of
Medicine, Houston, TX
In recent years it has become clear that traditional
concepts about immune response to and clearance of
pathogenic viruses are only part of the whole story.
Increasing numbers of viruses are now recognized to cause
persistent, low-level replication in the host, with
long-term adverse health consequences in both normal and
immune compromised hosts. These include viruses known to
establish latency, such as the herpes viruses, and viruses
that can cause persistent infection without a latent
state, such as hepatitis C virus and polyomaviruses. This
symposium will focus on virus–host interactions that
allow for establishment of latent or persistent infection
and the opportunities to exploit these interactions to
facilitate gene therapy.
Target Audience: Scientists and clinicians from the
following disciplines: pediatric infectious diseases,
general pediatricians, pediatric gastroenterologists,
pediatric hematology/oncology physicians.
Viral Persistence: Surveillance of the Iceberg from
Its Surface
John
A. Vanchiere, Baylor College of Medicine, Houston, TX
Unraveling the Molecular Mechanisms of Herpes Simplex
Virus Latency and Reactivation in the Nervous System
Nancy
M. Sawtell, Cincinnati Children's Hospital Medical Center,
Cincinnati, OH
Hepatitis C: Mechanisms Contributing to Chronic
Infection and Immune Evasion
Stanley
Lemon, University of Texas Medical Branch, Galveston, TX
Adenovirus Based Vectors as Tools to Understand Viral
Persistence
Andrea
Amalfitano, Duke University Medical Center, Durham, NC
Sponsored jointly by the Pediatric
Infectious Diseases Society and the Pediatric Academic
Societies
3:00pm–5:00pm
6700—Disorders
of Leukocyte Movement
PAS Topic Symposium
Chair:
Richard E. Stiehm, Mattel Children's Hospital at UCLA, Los
Angeles, CA
This symposium will focus on the importance of
leukocyte movement in infection and inflammation,
including basic mechanisms and abnormalities in several
rheumatic and immunodeficiency syndromes, including the
WHIM syndrome, the first described disorder of a chemokine
receptor mutation.
Target Audience: Immunologists, hematologists,
rheumatologists and basic scientists.
Introduction
E.
Richard Stiehm, Mattel Children's Hospital at UCLA, Los
Angeles, CA
Introduction to Cell Movement and Abnormalities in
Rheumatic Syndromes
Anna
Huttenlocher, University of Wisconsin, Madison, WI
Chemokines, Chemokine Receptors and the Defect in the
Warts-Hypogammaglobulinemia-Infection-Myelokathexis (WHIM)
Syndrome
Virginia
Gulino, National Cancer Institute/NIH, Bethesda, MD
Leukocyte Adhesion Defects: Clinical and Laboratory
Correlates
Steven
M. Holland, National Institute of Allergy and Infectious
Disease/NIH, Bethesda, MD
6701—Endocrine Complications of Cancer Therapy
PAS/ASPHO/LWPES Topic Symposium
Chairs:
H. Stacy Nicholson, Oregon Health & Science
University, Portland, OR; and Charles A. Sklar, Memorial
Sloan-Kettering Cancer Center, New York, NY
The primary objective of this session is to review
common endocrine sequelae of anti-cancer therapies,
focusing particularly on fertility outcomes. In addition
to discussing sequelae, interventions will also be a
particular focus, in particular assisted fertility
(present and future options).
Target Audience: Oncologists and endocrinologists who
must counsel patients and families regarding fertility
outcomes and options following treatment for childhood
cancer.
Reproductive Challenges After Childhood Cancer
Henry
Stacy Nicholson,
Fertility Deficits in Survivors of Childhood Cancer
Charles
A. Sklar, Memorial Sloan-Kettering Cancer Center, New
York, NY
The Promise of Ovarian Cryopreservation
David
Lee, Oregon Health & Science University, Portland, OR
Fertility Preservation Options for Males
Paul
Turek, University of California, San Francisco, CA
Sponsored jointly by the American Society
of Pediatric Hematology/Oncology, the Lawson Wilkins
Pediatric Endocrine Society and the Pediatric Academic
Societies
5:15pm–6:45pm
Poster
Session III
PAS Original Science Abstracts -
Poster Session
Neonatology
6874—Neonatal Hematology
Tuesday, MAY 17
8:00am–10:00am
7102—Transitioning
Complex Pediatric Patients to Adult Care
PAS/ASPN/LWPES Hot Topic
Chair:
Sandra L. Watkins, University of Washington, Seattle, WA
Transitions are a part of everyone's life experience.
Most young people with special health care needs and
disabilities (SHCN/D) become independent members of adult
society, but some need deliberate guidance and support.
With increasing success in reducing the mortality of once
devastating pediatric diseases, the latter group is
growing in number. A new consensus statement from the
American Academy of Pediatrics and the U.S. Federal
Government (Healthy People 2010) has focused attention on
the need to assist young people with SHCN/D in attaining
their potential in adulthood. This symposium will discuss
the growing number of young people with SHCN/D and present
approaches for effecting these transitions. Specific disease-related examples will be used to highlight
the issues, the barriers and the key elements of
successful programs that transition patients from
pediatric care to the adult system.
All Grown Up and Wondering What To Do: Transitioning
Complex Pediatric Patients to Adult Care
Patience
H. White, George Washington University School of Medicine,
Washington, DC
Transition Best Practices
Cecily
L. Betz, University Center For Excellence in Development
Disabilities, Children's Hospital Los Angeles, CA
Transition to Adult Care in the Nephrology
Population–Renal Failure or Success
Maria
Ferris, University of North Carolina-Chapel Hill, NC
Training and Workforce Issues for Successful
Transition
Roberta
G. Williams, University of Southern California, Los
Angeles, CA
Discussion
Sponsored jointly by the American Society of Pediatric Nephrology,
Lawson Wilkins Pediatric Endocrine Society and the
Pediatric Academic Societies
8:00am–10:00am
7152—Clinical
Trials in Perinatal and Neonatal Medicine II
PAS Original Science Abstracts -
Platform Session
1:45pm–3:45pm
7601—New
Therapeutic Strategies for Classical Pediatric Diseases
PAS Hot Topic
Chairs:
David Cornfield, University of Minnesota School of
Medicine, Minneapolis, MN; and Nina F. Schor, Children's
Hospital of Pittsburgh, Pittsburgh, PA
The pathogenesis of numerous single-gene disorders
has been effectively delineated. However, the application
of this knowledge to patient care has lagged far behind.
This symposium will present recent progress made in the
development of therapeutic strategies for four classical
pediatric disorders. First, novel genetic therapies for
hematologic diseases will be discussed. Second,
interventions that reverse the key abnormalities in signal
transduction underlying autosomal dominant polycystic
kidney disease, a leading cause of end-stage renal
disease, will be presented. Third, we will discuss a
treatment strategy that normalizes the intracellular
processing and function of the mutated cystic fibrosis
transmembrane conductance regulator (CFTR), which
underlies the majority of cases of CF. Fourth,
pharmacologic strategies against muscular dystrophy will
be presented. These four innovative approaches provide
great hope for patients suffering from these disorders,
and they serve as exciting examples of potential means to
combat other devastating pediatric conditions.
Target Audience: Scientists and clinicians involved
with the development of new therapeutic strategies for a
variety of childhood disorders.
Embryonic Globins as Therapeutic Agents for
Hemoglobinopathies and Thalassemias
J.
Eric Russell, The Children's Hospital of Philadelphia,
Philadelphia, PA
Novel Therapies for Renal Cystic Diseases
Vicente
E. Torres, Mayo Clinic, Rochester, MN
Treatment of Cystic Fibrosis with Curcumin
Marie
E. Egan, Yale University School of Medicine, New Haven, CT
Pharmacologic Strategies Against Muscular Dystrophy
Tejvir
S. Khurana, University of Pennsylvania School of Medicine,
Philadelphia, PA
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