Digestive Diseases News
Fall 2006

Consortium of Clinical Centers Collaborates on Biliary Atresia

Pooling knowledge, resources, and patients, scientists in a consortium of 10 pediatric liver disease and transplant centers are recruiting participants for studies aimed at finding the cause and improving treatment for biliary atresia, a devastating disease affecting about one in 10,000 newborns in the United States.

The Biliary Atresia Research Consortium (BARC) is charged with

• finding the causes of, and practical diagnosis methods for, biliary atresia

• identifying the natural history, disease progression, and complication risk factors for the disease

• recommending better management and treatment methods

Though rare, biliary atresia is a severe disease most responsible for liver transplantation in children—at least 40 percent of the cases in the United States. Without intervention, children with the illness die of end-stage liver disease within the first few years of life.

Because of both its rarity and severity, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) has made ongoing studies of biliary atresia a research priority. The NIDDK singled out biliary atresia for study because of the dearth of research on the disease, according to Patricia Robuck, Ph.D., BARC project scientist for the NIDDK. “We don’t even know why or how children develop it.”

Since no single clinical center handles a large enough number of infants with the illness to conduct studies of new therapies, the NIDDK created the BARC network to collaborate on studies of biliary atresia and other infant liver diseases related to the suppression of biliary flow. See the sidebar for a list of participating clinical sites.

BARC Projects

The consortium has designed several research projects to achieve its goals:

PROBE: The outcome of the PROspective Study of Biliary Atresia Epidemiology is a database of clinical information and repository of blood, DNA, and tissue samples from children with neonatal cholestatic liver diseases such as biliary atresia and neonatal hepatitis. The study population includes infants 6 months old or younger upon diagnosis at a BARC clinical site. The study will track participants up to age 10, liver transplantation, or, for children without biliary atresia, until 12 months of age or complete recovery, whichever is later.

START: STeroids in Biliary Atresia Randomized Trial is a randomized, double-blinded, placebo-controlled trial to test the effectiveness of corticosteroids following portoenterostomy— a procedure to improve bile drainage in infants with biliary atresia.

BASIC: Biliary Atresia Study in Infants and Children Database of Older Children with Biliary Atresia is a database of clinical information, genetic material, and body-fluid samples from children at least 1 year old that will allow investigators to

• identify genes that could be responsible for biliary atresia

• identify polymorphisms that might be important in disease progression

• characterize the natural history of older children with biliary atresia who have not had a transplant

• provide data and biological samples for future studies

Biliary atresia is generally discovered within the first 6 months of life, according to Robuck. “Eighty to 90 percent of these babies are born at term and look pretty healthy, but then their stool becomes very light in color, their urine dark, and they look yellow. These changes generally happen within the first few weeks of life.”

For more information about BARC and the ongoing studies, go to www.barcnetwork.org. To view or download a copy of an NIDDK fact sheet about biliary atresia, go to www.digestive.niddk.nih.gov/ddiseases/pubs/atresia.

[Top]

NIH Publication No. 07–4552
November 2006