Subject: [NATAP] NEW HEP C STUDY FOR CHILDREN (PEDS-C)
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NEW HEPATITIS C STUDY FOR CHILDREN (PEDS-C)
Jay H. Hoofnagle, MD, Liver Disease Research Branch
National Institute of Diabetes & Digestive & Kidney Diseases
National Institute of Health
Hepatitis C is a major cause of morbidity & mortality from liver disease in adults, but it is fortunately a rare cause of liver disease in children. In population-based surveys the prevalence of antibody to HCV in children was found to be 0.2% to 0.4%, from which it is estimated that 60,000 to 100,000 children in the US are chronically infected with HCV. As in adults, HCV in children occurs largely in high-risk groups. The major risk factor for HCV infection in young children is perinatal exposure from an infected mother. Among adolescents, risk factors also include injection drug use & receipt of blood transfusions or blood products before 1992.
HCV is often mild & minimally progressive during childhood; cases of cirrhosis from HCV have been reported but are rare. Chronic HCV is an uncommon reason for liver transplantation in the pediatric age group, and several prospective as well as cross-sectional studies of hepatitis C in children have documented the rarity of progressive liver disease in this population.
Nevertheless, HCV is a concern in pediatrics. While the disease tends to be mild, it is likely to be life-long, raising concerns about long-term survival & health. In addition, therapy for HCV is most likely to be successful in patients with early disease, before the onset of significant fibrosis or appearance of co-morbid conditions. A child with hepatitis C also must face concerns about infectivity & the possibility of spreading the infection to loved ones & future children.
Complicating the issues surrounding HCV in children is the lack of information on the safety & efficacy of current therapies for HCV in the pediatric age group. The combination of standard interferon alfa & ribavirin was recently approved for use in children with chronic HCV who are 3 yrs & older. However, studies of more current regimens of therapy using peginterferon & ribavirin have not been conducted in adequate numbers of children to make rational decisions regarding therapy.
In response to these needs, the National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK) in collaboration with the Food & Drug Administration's Office of Orphan Products Development have funded a multicenter, randomized, controlled trial of peginterferon with or without ribavirin in children with chronic HCV (PEDS-C).
Major funding & support have also been provided by Hoffman-LaRoche, Inc. (Nutley, NJ), The study is being conducted at 11 medical centers throughout the USA. The principal investigator is Dr. Kathleen Schwarz of the Johns Hopkins University School of Medicine. The other principal investigators & clinical centers are:
William Balistreri, Children's Hospital Medical Center, Cincinnati, OH
Regino Gonzalez-Peralta, University of Florida, Gainesville, FL
Barbara Haber, Children's Hospital of Philadelphia, Philadelphia, PA
Maureen Jonas, Children's Hospital, Boston, MA
Parvathi Mohan, George Washington University, Wash DC
Jean P. Molleston, Indiana University, Indianapolis, IN
Karen F. Murray, Children's Hospital & Regional Medicam Center, Seattle, WA
Michael R. Narkewicz, Children's Hospital, Denver, CO
Philip Rosenthal, University of California, San Francisco, SF
Lesley Smith, Columbia University Medical Center, New York, NY
Zachary Goodman (Pathologist), Armed Forces Institute of Pathology, Wash DC
Patrick Robuck (Project Scientist), NIDDK, Bethesda, MD
The design of the study is for 112 children with chronic HCV (ages 5-18) to be randomized to receive a 48-week course of peginterferon alfa-2a with either ribavirin or placebo. The dose of peginterferon will be 180 ug/1.73 m2 & that of ribavirin will be 15 mg/kg/day in two divided doses. Therapy will be discontinued at 24 weeks in nonresponders, defined as the persistence of HCV RNA. The primary endpoint is the absence of detectable HCV RNA in serum at least 24 weeks after completion of therapy. Children will have a liver biopsy before treatment, but follow-up biopsies are not planned. Children will be closely monitored. Major ancillary studies in this trial will focus on health-related quality of life, cognitive function, and psychological functioning before, during, and after therapy, with plans for long-term follow-up. Also included are measures of growth & development, diet, physical activity, and body composition. Thus, this trial will allow for a careful analysis of both short-
and long-term safety & efficacy of peginterferon and ribavirin in children with hepatitis C. referral of patients fpr enrollment is encouraged.
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