Pediatric Academic Societies'
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Telephone:  281-419-0052

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2006 PAS Annual Meeting

April 29–May 2 
San Francisco, California

ASPN Daily Schedule (as of February 1, 2006)


Saturday, April 29

8:00am–11:00am
2120—Management of Childhood Hypertension: Guidelines and Controversies
PAS/ASPN/IPHA Mini Course
Chairs: Steven R. Daniels, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; and Ronald J. Portman, University of Texas Medical School, Houston, TX

Target Audience: General pediatricians, emergency medicine physicians, hospitalists, intensivists, nephrologists and cardiologists.

The 2004 NHLBI guidelines for childhood hypertension answered many questions about how to approach hypertensive children, but left others unanswered. This mini course is designed to address some of the more controversial aspects of managing hypertensive children, with the hope of stimulating further discussion on the optimal approach to these patients. Practical approaches to clinical management will be emphasized.

  • Overview
    Stephen R. Daniels, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

  • Overview of Treatment Guidelines from the 4th Report
    Bonita E. Falkner, Thomas Jefferson University, Philadelphia, PA

  • Management of Pre-hypertension: Lifestyle Changes or Pharmacologic Treatment?
    Shawna D. Nesbitt, University of Texas Southwestern Medical Center, Dallas, TX

  • Choice of Agent for Children with Primary Hypertension
    Joseph T. Flynn, Children's Hospital at Montefiore, Bronx, NY

  • Treatment of Severe Hypertension in Ambulatory and Inpatient Settings
    Joshua Samuels, University of Texas, Houston, TX

  • Treatment of Hypertension in Special Populations
    Donald L. Batisky, Columbus Children's Hospital/The Ohio State University College of Medicine, Columbus, OH

  • Discussion

Sponsored jointly by the American Society of Pediatric Nephrology, the International Pediatric Hypertension Association and the Pediatric Academic Societies

9:45am–11:45am
2200A—Clinical Trials and Observational Studies
ASPN Workshop
Chairs: Susan L. Furth, Johns Hopkins University, Baltimore, MD; and Craig Wong, Children's Hospital of New Mexico, Albuquerque, NM

Target Audience: Clinical investigators and pediatric nephrologists.

This workshop will address statistical, measurement, ethical and regulatory issues in clinical research. We will discuss methodological issues in randomized clinical trials when the sample size is limited, as often occurs in pediatric studies. We will also address the measurement of kidney function in large cohort studies. Finally, we will have an extended discussion on the evolution of the current regulatory system of clinical research in the United States. This has evolved from concerns about ethical issues and protection of subjects to concerns about protection of the institution through compliance with inflexible requirements. The session will end with suggestions on what changes are needed and how to achieve them in the current regulatory environment.

  • Introduction
    Catherine Stehman-Breen, Amgen, Thousand Oaks, CA

  • Methodologic Issues in Clinical Trials When Sample Size Is Limited
    Tom Greene, The Cleveland Clinic, Cleveland, OH

  • Measurement of Glomerular Filtration Rate in Large Cohort Studies: Design, Conduct and Analysis
    Alvaro Munoz, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD

  • The Dysregulation of Research
    Norman Fost, University of Wisconsin Medical School, Madison, WI

Supported by an unrestricted educational grant from the Kidney and Urology Foundation of America, Inc. (KUFA)

11:45am–2:45pm
2424—Treatment of the Metabolic Syndrome in Children and Adolescents
PAS Educational Workshop
Leader: J. Darrell Nesmith, Little Rock, AR; Co-leaders: Alba Morales, Mohammad Ilyas, Lisa Lubsch

Target Audience: Trainees, fellows, junior faculty, mid-level faculty, senior faculty, and community practitioners.

The rise in pediatric obesity and metabolic syndrome is well established. Less understood for the pediatrician is the treatment of the metabolic syndrome. In this workshop, we aim 1) to briefly discuss the epidemiology of the metabolic syndrome in children and adolescents, 2) to discuss non-pharmacologic and pharmacologic treatment of the metabolic syndrome, and 3) to review a stepped approach in treating adolescents with the metabolic syndrome.

This workshop will be largely case-based. Come prepared to devise treatment plans in a small group setting. Participants are invited to bring their own cases for discussion.

Objectives:

– Participants will learn the epidemiology of the metabolic syndrome in children and adolescents.
– Participants will become familiar with existing treatment guidelines for components of the metabolic syndrome in children and adolescents.
– Participants will identify gaps in the literature regarding treatment guidelines of the metabolic syndrome in children and adolescents.
– Participants will consider pharmacologic treatment options of metabolic syndrome treatment based on the available evidence.

12:00pm–1:00pm
2450A—Renal Pathology—Battle of the Brains
ASPN Luncheon
Chairs: Patrick D. Walker, Nephropathology Associates, Little Rock, AR; and Victoria F. Norwood, University of Virginia Children's Medical Center, Charlottesville, VA

Target Audience: American Society of Pediatric Nephrology members and trainees.

Interactive clinical renal pathology conference for fellows and selected pediatric nephrology and pathology faculty during which cases will be presented for evaluation and spirited discussion. The objectives are to stimulate interactions between fellows and members of the society; encourage broad discussions of renal pathology, pathophysiology and treatment conundrums; and stimulate the development of potential research questions. Space is limited.

  • Welcome on Behalf of ASPN
    Victoria F. Norwood, University of Virginia Children's Medical Center, Charlottesville, VA

  • Overview and Keys to the Game
    Patrick D. Walker, Nephropathology Associates, Little Rock, AR

1:00pm–3:00pm
2625—Nephrology I
PAS/ASPN Platform Session

3:15pm–5:15pm
2730—Mechanisms of Hypertension in the Molecular Era
PAS/ASPN/IPHA/LWPES Topic Symposium
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 biology, most notably the identification of several single-gene defects that cause hypertension. This session will update the audience on the latest advances in our knowledge of molecular mechanisms of a variety of forms of hypertension.

  • Role of Dopamine Receptors
    Pedro A. Jose, Georgetown University Medical Center, Washington, DC

  • Perinatal Programming and the Development of Hypertension
    Lori Woods, Oregon Health & Science University, Portland, OR

  • Low Renin Hypertension in Childhood
    Maria I. New, Mount Sinai School of Medicine, New York, NY

  • 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

5:15pm–6:00pm
2800—Clinical Pediatric Hypertension
PAS/ASPN/IPHA Poster Symposium

8:00pm–10:00pm
2980A—ASPN Member Reception
ASPN Dinner

  • Presenter—Research Trainee Awards
    Susan L. Furth, Johns Hopkins University, Baltimore, MD

  • ASPN Trainee Basic Research Award Recipients
    Janis Dionne, British Columbia Children's Hospital, Vancouver, CA
    David Sullivan Hains, Children's Hospital, Columbus, OH

  • ASPN Trainee Clinical Research Award Recipients
    Karen L McNiece, University of Texas-Houston Medical School, Houston, TX
    Mai Thanh-Thuy Nguyen, Cincinnati Children's Hospital, Cincinnati, OH


Sunday, April 30

7:00am–8:00am
3050—Life as a Pediatric Nephrologist
PAS Meet the Professor

This session is intended to provide trainees and junior faculty with insights about career opportunities in pediatric nephrology and appropriate preparation for these careers. Career opportunities, both within and outside of academic departments, will be discussed. Faculty tracks and the perspective of department chairs about these tracks also will be addressed. Topics will include how to choose the appropriate academic position for one’s interests and talents, as well as balancing career objectives with personal and family goals.

Thomas R. Welch, Professor and Chair, Department of Pediatrics, SUNY Upstate Medical University, Syracuse, NY

8:00am–10:00am
3115A—Renal Pathology—It's Still Not Just Little Adults
ASPN Symposium
Chairs: Sharon P. Andreoli, James Whitcomb Riley Hospital for Children, Indiana University Medical Center, Indianapolis, IN; and Patrick Walker, Nephropathology Associates

Target Audience: Nephrologists and pathologists.

The pathologic features of the kidney in pediatric kidney disease have unique features compared to adult patients and, some kidney diseases are solely observed in pediatric patients. This symposia will address the unique pathologic features of congenital nephrotic syndrome, MPGN, renal pathology in pediatric transplant patients and will also propose a new taxonomy for the podocytopathies.

  • Congenital Nephrotic Syndrome—An Update
    Stephen M. Bonsib, James Whitcomb Riley Hospital for Children, Indiana University Medical Center, Indianapolis, IN

  • MPGN and Dense Deposit Disease
    Patrick D. Walker, Nephropathology Associates, Little Rock, AR

  • Renal Pathology in Pediatric Transplant Patients
    Carole A. Vogler, Saint Louis University, St. Louis, MO

  • Toward a New Taxonomy for the Podocytopathies
    Laura Barisoni, New York University School of Medicine, New York, NY

10:15am–12:00pm
3350—APS Presidential Plenary and Awards
APS Presidential Plenary
Chair: David K. Stevenson, Stanford University School of Medicine, Palo Alto, CA

*The Joseph W. St. Geme, Jr. Leadership Award is presented by the Federation of Pediatric Organizations on behalf of the Ambulatory Pediatric Association, American Academy of Pediatrics, American Board of Pediatrics, American Pediatric Society, Association of Medical School Pediatric Department Chairmen, Association of Pediatric Program Directors and Society for Pediatric Research.

  • 2006 APS Presidential Address
    David K. Stevenson, Harold K. Faber Professor of Pediatrics, Senior Associate Dean for Academic Affairs, Stanford University School of Medicine; Director, Charles B. and Ann L. Johnson Center for Pregnancy and Newborn Services; Chief, Division of Neonatal and Developmental Medicine, Lucile Packard Children's Hospital at Stanford, Palo Alto, CA

  • New Member Outstanding Science Award

  • Epithelial Branching and the Origins of Kidney Malformation
    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

  • 54th Annual John Howland Award
    Kurt Hirschhorn, Professor of Pediatrics, Human Genetics and Medicine, Chairman Emeritus, Department of Pediatrics, Mount Sinai School of Medicine, New York, NY

    • Introduction, John Howland Awardee
      Frederick J. Suchy, Herbert H. Lehman Professor of Pediatrics and Chair, Department of Pediatrics, Mount Sinai School of Medicine, New York, NY

  • Joseph W. St. Geme, Jr. Leadership Award
    Carol D. Berkowitz, Professor of Clinical Pediatrics David Geffen School of Medicine at UCLA

    • Introduction, Joseph W. St. Geme, Jr. Leadership Award

12:00pm–1:30pm
3430A—ASPN Business Meeting, Luncheon and Presidential Address
ASPN Luncheon

  • ASPN Presidential Address
    Sandra L. Watkins, Children's Hospital and Regional Medical Center, Seattle, WA

2:00pm–4:00pm
3728—Nephrology II
PAS/ASPN Original Science Abstracts - Platform Session

4:15pm–6:15pm
3825A—Systemic Lupus: Implications of Recent Developments for Management of Children with Lupus Nephritis
ASPN Symposium
Chairs: Joseph T. Flynn, Montefiore Medical Center, Bronx, NY; and James Jarvis, University of Oklahoma Health Sciences Center, Oklahoma City, OK

Target Audience: Nephrologists and rheumatologists.

Glomerulonephritis remains a significant source of morbidity in children with SLE. However, recent changes in renal pathology and immunosuppressive regimens offer the potential for improved outcomes in affected children. This session will highlight some of the recent advances in the diagnosis and treatment of children with lupus nephritis.

  • Overview
    Joseph T. Flynn, Children's Hospital at Montefiore, Bronx, NY

  • Lupus Nephritis: The Rheumatologist's View
    James N. Jarvis, University of Oklahoma Health Sciences Center, Oklahoma City, OK

  • Updated WHO Classification System: Are There Implications for Therapy?
    Glen S. Markowitz, Columbia University Medical Center, New York, NY

  • Application of Monoclonal Antibodies in Therapy: Rituximab and Beyond
    Sangeeta Sule, Johns Hopkins University, Baltimore, MD

  • Cyclophosphamide Versus Mycophenolate as Initial Therapy for Class III and IV Lupus Nephritis
    Ana L. Paredes, Miami Children's Hospital, Miami, FL

Sponsored jointly by the AAP Section on Rheumatology and the American Society of Pediatric Nephrology


Monday, May 1

8:00am–10:00am
4110—Pediatric Fluids and Hyponatremia: Are We Giving Too Much Water?
PAS/ASPN/LWPES Topic Symposium
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, hospitalits, 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

10:15am–12:10pm
4300    SPR Presidential Plenary and Awards
Chair: Philip W. Shaul, University of Texas Southwestern Medical Center, Dallas, TX

  • Introduction
    Philip W. Shaul, University of Texas Southwestern Medical Center, Dallas, TX

  • Award Presentations 
    Student Research Awards
    House Officer Awards
    Fellow’s Basic Research Awards
    Fellow’s Clinical Research Awards
    David G. Nathan Award in Hematology/Oncology
    Japan Pediatric Society Fellow Awardees
    Douglas K. Richardson Award for Perinatal and Pediatric Healthcare Research
    Richard D. Rowe Award in Perinatal Cardiology

  • Young Investigator Award and Lecture

  • Maureen Andrew Mentor Award and Lecture

  • E. Mead Johnson Awards for Research in Pediatrics and Lectures*

  • Presidential Address
    Philip W. Shaul, University of Texas Southwestern Medical Center, Dallas, TX

*The E. Mead Johnson Awards are supported by an educational grant from Mead Johnson Nutritionals

12:00pm–1:00pm
4400A—ASPN Awards Luncheon
ASPN Presidential Plenary

  • American Academy of Pediatrics Section on Nephrology—Henry L. Barnett Award

    • Introducer:
      Steven J. Wassner, MD, Milton S. Hershey Medical Center, Hershey, PA

    • Presenter: 
      Richard N. Fine, MD, State University of New York at Stony Brook, Stony Brook, NY

    • Recipient:
      Jose Strauss, MD, Founder and Program Chairman, Pediatric Nephrology Seminar XXXIII, Division of Pediatric Nephrology, University of Miami Miller School of Medicine, Miami, FL

  • American Society of Pediatric Nephrology Founder’s Awards

    • Introduction:
      Eileen Brewer, MD, Professor and Chief, Pediatric Nephrology, Baylor College of Medicine/Texas Children’s Hospital, Houston, TX

    • Presenter:
      Sandra L. Watkins, MD, Children's Hospital and Regional Medical Center, Seattle, WA

    • Recipient:
      Barbara R. Cole, MD, Emeritus, Department of Pediatrics, Washington University School of Medicine, St. Louis, MO

    • Introduction:
      Lisa M. Satlin, MD, Chief, Division of Pediatric Nephrology, Mount Sinai School of Medicine, New York, NY

    • Presenter:
      Sandra L. Watkins, MD, Children's Hospital and Regional Medical Center, Seattle, WA

    • Recipient:
      Adrian Spitzer, MD, Professor of Pediatrics, Albert Einstein College of Medicine, Bronx, NY

1:00pm–2:45pm
4500   March of Dimes Prize in Developmental Biology Lecture

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

3:00pm–5:00pm
4630A—Molecular Control of the Formation of the Renal Collecting System
ASPN Symposium
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


Tuesday, May 2

8:00am–10:00am
5110A—Inflammation in Uremic Pathophysiology
ASPN Symposium
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

10:00am–12:00pm
5360A—Pay for Performance: The Pediatric Perspective—Hemodialysis
ASPN Workshop
Chairs: Sandra L. Watkins, Children's Hospital and Regional Medical Center, Seattle, WA; and Bradley A. Warady, Children's Mercy Hospital and Clinics, Kansas City, MO

Target Audience: Nephrologists.

Quality patient care is of utmost importance to pediatricians caring for children. Congress and the Centers for Medicare and Medicaid are exploring models of “Pay for Performance” in an effort to reward high quality patient care and encourage ongoing quality improvement. This workshop explores the latest research results that aid the clinician in improving patient outcomes in hemodialysis, reviews the data available linking performance measures and outcomes and discusses the mechanisms for reimbursement.

  • Overview
    Bradley A. Warady, Children's Mercy Hospital and Clinics, Kansas City, MO

  • New Insights into Improved Quality Care in Hemodialysis
    Stuart L. Goldstein, Baylor College of Medicine/Texas Children's Hospital, Houston, TX

  • Quality Measures for Pediatric Hemodialysis—What Should They Be?
    Barbara Fivush, Johns Hopkins University, Baltimore, MD

  • How Can a Pediatric Nephrologist be Appropriately Compensated for Providing Quality Hemodialysis Care?
    Linda Upchurch, Baxter Healthcare Corporation, Fletcher, NC

  • Discussion

Sponsored jointly by the North American Pediatric Renal Transplant Cooperative Study (NAPRTCS) and the American Society of Pediatric Nephrology

1:45pm–3:45pm
5720—Autosomal Recessive Polycystic Kidney Disease (ARPKD): New Insights and Clinical Perspectives
PAS/ASPN/NASPGHAN Topic Symposium
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.

  • Transcriptional Complexity of PKHD1: Implications for Development and Disease Pathogenesis
    Gregory G. Germino, Johns Hopkins University, Baltimore, MD

  • Pathobiology of Biliary Epithelia in ARPKD
    Tatyana Masyuk, Mayo Clinic College of Medicine, Rochester, MN

  • Oligohydramnios: Current Concepts and Implications for Pulmonary Development
    F. Sessions Cole, Washington University School of Medicine, St. Louis Children's Hospital, St. Louis, MO

  • 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

 

   
 

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Last Updated: September 26, 2006