Pentoxifylline Therapy in Biliary Atresia

Biliary atresia (BA) is a devastating liver disease of infancy of unknown etiology,
characterized by bile duct obstruction, live fibrosis, and cirrhosis. BA has no known
medical treatments. The only proven treatment is a surgical portoenterostomy (the Kasai
procedure, or KP) which can achieve bile drainage and improve outcomes in some cases. The
KPs success is variable depending on several factors including age of the infant, experience
of the surgeon, and extent of liver fibrosis at the time of KP.

In this study, the investigators conduct a phase II trial of a potential new medical therapy
for BA: pentoxifylline (PTX). PTX is a methylxanthine derivative closely related to caffeine
that has been used safely in infants with other diseases such as sepsis. In adults, PTX has
been shown to have a number of properties beneficial to the liver, including preventing
liver fibrosis, improving liver regeneration, and reducing cirrhosis-related complications.

The trial's objective is to determine whether PTX has sufficient biological activity against
BA to warrant further study. PTX will be administered orally for 90 days as an adjunct to
standard therapy (i.e. KP if appropriate). The primary outcome will measure the change in
serum conjugated bilirubin levels after 90 days. Secondary outcomes include changes in body
weight, serum markers, liver imaging, and time to liver transplant in infants with BA.

Inclusion Criteria:

- 0-180 days old

- Diagnosed with biliary atresia through liver biopsy and/or intra-operative
cholangiogram

- No previous Kasai portoenterostomy performed at another institution

- Able to take medications orally

- Legal guardian signs consent after understanding risks and investigational nature of
study

Exclusion Criteria:

- Infants greater than 180 days old

- Infants receiving a Kasai portoenterostomy at another institution

- Infants unable to take medications orally