Sokol, Ronald J. M.D.
Section Editor(s): Baker, Robert D. Jr. M.D., Ph.D.; Rosenthal, Philip M.D.; Sherman, Philip M. M.D., F.R.C.P.C.; Büller, Hans M.D.
Chairman, Children's Liver Council, American Liver Foundation
Dr. Rosenthal is a Principal Investigator for a bioartificial liver support device (Circe Biomedical) currently undergoing Phase II/III clinical trial.
In November, 2000 the American Liver Foundation released the “Pediatric Liver Research Agenda 2000: A Blueprint for the Future”. This group of documents, written by twenty pediatric experts in hepatology, has identified research needs and priorities in eleven important areas of pediatric hepatology that demand increased attention by the scientific community and by research funding agencies. The major concerns that led to the creation of the Pediatric Liver Research Agenda were a need to establish a focused direction in pediatric liver research efforts, a lack of manpower to carry out needed research, a lack of effective therapies for many of the most common hepatic diseases of childhood, and the inadequate funding to support important basic and clinical research. It was hoped that publishing this Agenda would begin to address these needs. Investigators could use this document as a blueprint for directing and prioritizing basic and applied research in pediatric liver disease in order to gain a better grasp of etiology and pathogenesis of these conditions and make better progress in developing effective therapies. Moreover, it was anticipated that the Agenda would serve as an impetus to private and federal agencies to increase support of this research by educating them about the scope of liver disease in childhood, identifying the unique and critical research needs in pediatric hepatology, and encouraging them to place pediatric liver diseases higher on their priority lists for funding in the immediate future.
The authors of the Agenda believed strongly that there is an insufficient appreciation of the serious impact of liver diseases in infancy and childhood. There is a wide spectrum of disorders that present at birth, in infancy, or later in childhood, many of which are unique to children. The research efforts of colleagues in adult hepatology have not had a significant impact upon the disease-related morbidities of pediatric patients, which include impaired growth and development, diarrhea, pruritus, gastrointestinal hemorrhage and the complications of cirrhosis. Although some notable advances have been made recently in the treatment of several metabolic liver diseases (e.g., hereditary tyrosinemia), treatment for most pediatric liver disorders is inadequate and does not significantly alter the natural history of the disease. Therefore, liver transplantation all too frequently has been the only option available to children with end-stage liver disease. In the United States alone, in 1998, 573 liver transplants were performed in children. Over 15,000 children are hospitalized in the United States for liver diseases each year at a cost of over $350 million per year. The responsibility of caring for an infant or child with liver disease has profound and uncounted emotional and financial effects on families involved as well. Despite this considerable impact on the health of children, on the family unit, and on health care expenditures, there has been insufficient research funding for investigations into the causes and treatments of pediatric hepatobiliary disorders.
In response to these pressing needs, the Pediatric Liver Research Agenda identified four critical actions that should be taken. These include:
setting research priorities, which is the purpose of this Research Agenda
increasing financial support from federal and private funding agencies for basic and applied research in these areas
establishing and supporting a national network of specialized centers with expertise in the care of children with liver disorders and in the conduct of clinical investigations and trials
supporting the training of present, and increasing the number of future, pediatric gastroenterologist/hepatologists with expertise in hepatobiliary disorders and liver transplantation and promoting the training of other scientists investigating important issues related to the Research Agenda.
The specific areas of emphasis covered by the Agenda are development of the liver and bile ducts, liver injury and fibrosis, biliary atresia, intrahepatic cholestatic liver diseases, metabolic liver disease, viral hepatitis in childhood, fulminant hepatic failure in children, immune and autoimmune disorders of the liver and biliary tract, liver transplantation in children, gene therapy in pediatric liver disease, and the impact of liver disease on growth and nutrition.
The Agenda will be distributed widely to governmental agencies, research funding sources, pediatric, hepatology and research societies, appropriate foundations and the NASPGN membership. Copies of the Agenda are currently available from the American Liver Foundation website (http://www.liverfoundation. org) and the entire Agenda will be published as a supplement to a pediatric journal this year.
© 2001 Lippincott Williams & Wilkins, Inc.