Current Studies

Follow these links for information regarding each study:

• A Retrospective Study of Biliary Atresia Patients up to 2 Years of Age

• Assessment of Inter-Observer Variability in the Histological Diagnosis of Infantile Cholestasis

• PROBE: A Prospective Database of Infants with Cholestasis

• START: A randomized double-blinded, placebo-controlled trial of corticosteroid therapy following portoenterostomy in infants with biliary atresia

• A Database of Older Children with Biliary Atresia

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

• Case Report Form
• Disease Definitions

This was a retrospective study of 104 infants in the BARC centers who underwent a hepatoportoenterostomy (HPE) for biliary atresia between January 1997 and December 2000 to determine clinical and/or laboratory factors predicting patient outcome at 24 months. The following abstracts have been presented:

Shneider, B et al. (2004). A multi-center analysis of outcome at 24 months of age in children with biliary atresia in the United States. Presented at the American Association for the Study of Liver Diseases, 55th Annual Meeting 29, Boston, MA, 2004. Hepatology 2004 Oct; 40(1): 201A
There was markedly improved survival in children with a total serum bilirubin less than 2 mg/dl at 3 months after hepatoportoenterostomy (HPE) compared to those whose bilirubin levels were greater than 6 mg/dl. Negative outcome (NO) (as defined by death or transplant within the first 24 months of age) was more common in children with polysplenia syndrome than in those without anomalies (82 vs. 49%). The age at presentation to the BARC center was higher in children with NO (58 vs. 45 days) as was the age at HPE (66 vs. 54 days).

Haber, B. et al. (2004). Patient demographics and clinical practices in a US multi-center study of biliary atresia. Presented at the American Association for the Study of Liver Diseases, 55th Annual Meeting 29, Boston, MA, 2004. Hepatology 2004 Oct; 40(1): 472A
Regardless of ethnicity or gender, the prevalence of associated anomalies (24% total) was higher than previously reported. There was also variability between patients in terms of tests performed as well as antibiotic, ursodeoxycholic acid and steroid use. The age at referral and at hepatoportoenterostomy (HPE) continued to be unacceptably high. Lastly, outcome continued to be poor with less than half of patients surviving with their native liver at 2 years of age.

DeRusso P, Ye W, Haber B, Shneider B, Sokol R, Whitington P, Squires R, Bezerra J, Shepherd R, Rosenthal P, Robuck P. Early Growth Failure following Hepatoportoenterostomy is Associated with Liver Transplantation or Death in Infants with Biliary Atresia. Presented at the American Association for the Study of Liver Diseases, 56th Annual Meeting, San Francisco, CA 2005. Hepatology 2005 Oct; 42(S1): 64A.

The following paper has been accepted for publication:

Shneider BL, MB Brown, B Haber, PF Whitington, K Schwarz, R Squires, R Shepherd, J Bezerra, P Rosenthal, J Hoofnagle and RJ Sokol (2006). A multi-center study of biliary atresia outcome in the United States (1997-2000). Journal of Pediatrics, 2006 Apr; 148(4):467-74.

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Assessment of Inter-Observer Variability in the Histological Diagnosis of Infantile Cholestasis

This is a retrospective study of histopathology of biliary atresia among the BARC pathologists to refine diagnostic criteria and develop a scoring system. This study is ongoing.

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PROBE: A Prospective Database of Infants with Cholestasis

This is a prospective, comprehensive database study of all infants in the BARC centers fulfilling the inclusion criteria of 0 – 180 days of age with cholestasis (serum direct bilirubin of 2 mg/dl or greater and >20% of the total bilirubin). The factors which might predict clinical outcomes, such as age at diagnosis and age of the portoenterostomy, are being investigated, as are long term assessments of health related quality of life, school performance, and neurodevelopmental outcome. A serum/plasma/tissue bank and immortalized cell lines are also being created to facilitate scientific studies of these diseases. SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT THE SITE NEAREST YOU FOR FURTHER DETAILS.

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START: STeroids in biliary Atresia Randomized Trial: A randomized, double-blinded, placebo-controlled trial of corticosteroid therapy following portoenterostomy in infants with biliary atresia

This is a randomized controlled trial designed to study the efficacy and safety of high-dose perioperative corticosteroids for 2 months following portoenterostomy. The primary outcome is serum bilirubin at 6 months after surgery and the secondary outcomes include survival without liver transplantation, complications, and PELD score. Toxicity measures being investigated include adverse events, vaccine responses, infections, neurodevelopmental outcome, and GI bleeding. SUBJECTS ARE ACTIVELY BEING ENROLLED. PLEASE CONTACT THE SITE NEAREST YOU FOR FURTHER DETAILS.

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A Database of Older Children with Biliary Atresia

This is a long-term follow-up of biliary atresia patients in BARC centers. The study is currently under development.

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