A PROSPECTIVE DATABASE OF INFANTS WITH CHOLESTASIS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objectives of this research study are to establish (1) a database containing clinical information and (2) a repository of blood and tissue samples from children with neonatal liver disease such as biliary atresia and neonatal hepatitis to facilitate research in these important liver problems in children. Examples of the use of this database and repository are to study the pathogenesis and natural history of biliary atresia and neonatal hepatitis or to evaluate patterns of cellular gene and protein expression in tissue specimens and plasma by viral, genomic and proteomic techniques. The study population will consist of infants, both male and female, with cholestasis who are less than or equal to 180 days old at the time of diagnosis at a Biliary Atresia Research Consortium (BARC) clinical site. In order to study the natural history, subjects will be followed until 10 years of age, liver transplantation or, for children without biliary atresia, until complete recovery off of all therapy. The multi-center network of investigators will address issues of etiology, pathogenesis, natural history, diagnosis, and novel treatments for one of the most devastating pediatric illnesses that occur in the first few months of life, biliary atresia, and related disorders such as idiopathis neonatal hepatitis. Although these disorders are not common, the effects on children, siblings, and their families and the frequent need for Liver transplantation, mandate a greater degree of attention by the scientific community than in the past. Moreover, knowledge gained from these investiagtions has a high likelihood of affecting diagnosis and treatment of adults with cholestatic liver disease and improving our understanding of fundamental processes regulating bile flow, cytoprotection, and liver and biliary development. The development of a serum and tissue bank of specimens from children with various neonatal cholestatic disorders will be invaluable tool for current and future investigations into the etiology and pathogenesis of hepatobiliary injury to the infant.