A Retrospective Study of Biliary Atresia Patients up to 2 Years of Age (P001)

General Description

The Biliary Atresia Research Consortium (BARC) is an NIH-funded network of 9 clinical sites and a data coordinating center whose goal is to study biliary atresia, neonatal hepatitis and other cholestatic diseases in the infant. BARC is preparing a prospective observational study of infants with cholestatic disease and clinical trials related to modalities of treatment.

Biliary atresia is the leading cause of morbidity and mortality from pediatric liver disease, and is the most common indication for pediatric liver transplantation. Despite its clinical importance, little is known of a wide range of fundamental issues regarding biliary atresia and optimal approaches to the medical management are unknown. No prospective randomized trials of important aspects of the care of children with biliary atresia have been performed. The majority of published data regarding biliary atresia is anecdotal and based upon a single center experience. Comprehensive registries of the management and outcome of biliary atresia do not exist.

This retrospective study will be unique and powerful because it analyzes the results of a large cohort of children in the United States from a diverse group of institutions with expertise in the management of children with liver disease. The proposed retrospective study may provide novel information and may lead to insights that can be used in the design of a subsequent 5 year prospective study.

Objectives

The primary aim of the retrospective study of biliary atresia is to collect and correlate data about the presentation, diagnostic interventions, medical and surgical management of infants with this disease in order to identify risk factors that affect outcomes at two years of age. The database is anticipated to provide comprehensive information which can lead to disease management standards and insights that might be used in the design of future BARC studies such as the creation of a prospective data base and the design of a randomized clinical trial to study the effect of using corticosteroids post-surgery.

Outcome Measure

As the objective of this study is to collect and correlate data, there are no outcome measures.

Criteria

Inclusion Criteria:

• Patients with a date of birth between 01/01/1997 and 12/31/2000, who had a hepatopotoenterostomy performed for the diagnosis of biliary atresia as defined by BARC will be considered for this study.

Exclusion Criteria:

• Patients with a diagnosis of chelodochal cyst or other disease manifesting in cholestatic liver disease, and for whom there is lack of documentation of follow-up with labs, sentinel events, etc. (not secondary to death) extending to the child’s 2nd birthday will not be considered for this study.