Designated Biliary Atresia Center
Biliary atresia remains the number one indication for liver transplant in children. One of the major contributing factors to a newborn’s outcome when diagnosed with this form of liver disease is a timely diagnosis, due to the rapidity with which the liver becomes damaged in the first few weeks of life. Studies have shown that patients have a much better chance of long-term survival if a child is under the medical and surgical direction of a specialized pediatric liver program.
Due to its extensive experience in treating this pediatric liver disease, St. Louis Children’s Hospital has become a designated biliary atresia center of excellence. “Most patients with biliary atresia will require transplantation,” states Dr. Turmelle, medical director of liver transplantation. “Expert medical management of chronic liver disease is necessary while awaiting transplant, since the overall health status at time of transplant influences outcome after transplant. These include maximizing the patient’s nutritional status and managing complications such as ascites, electrolyte disturbances, increased risk of bleeding and infections.”
Biliary Atresia Research
For seven years, St. Louis Children’s Hospital has been one of 10 leading pediatric research centers participating in the Biliary Atresia Research Consortium (BARC), a nationwide, multi-center network that receives funding from the National Institutes of Health to study biliary atresia. “No single center has enough experience or sees enough patients, because biliary atresia is so rare,” explains Dr. Turmelle, WUSM instructor in pediatrics. “The consortium was first created to study biliary atresia, but now we’re looking at other cholestatic liver diseases along with the Childhood Liver Disease Research and Education Network (ChiLDREN)—a group of doctors, nurses, research coordinators and medical facilities in the United States working together to study infants with rare liver diseases, including alpha-1 antitrypsin deficiency, progressive familiar intrahepatic cholestasis, bile acid synthetic defects, Alagille syndrome, mitochondrial hepatopathies, and cystic fibrosis liver disease.”
This study is what first brought 2-year-old Emery Patterson to St. Louis Children’s Hospital. Emery was enrolled in the study in New York following her biliary atresia diagnosis. Knowing a job relocation was imminent, her parents moved to a Southern Illinois town to ensure that Emery could be near a participating study hospital. Emery was eventually listed for transplant. Six weeks later, she underwent a whole liver transplant at St. Louis Children’s Hospital.
Patient Success Story: Baby Anthony, diagnosed with biliary atresia, gets a liver transplant
Research in the Long Term
Dr. Turmelle is working on an algorithm to help pediatricians and other physicians better understand clinical features and laboratory levels that would suggest biliary atresia or other types of liver diseases. Additionally, as part of the ChiLDREN study group, they are involved in a blind, randomized study to determine the efficacy of steroid use after the Kasai procedure, the initial corrective surgery that most children with biliary atresia undergo. A prospective field of future research includes the type and duration of immunosuppression following liver transplant. “We are very aggressive in trying to minimize immune suppression, and by six months to one year post-transplant, most of our patients are on immunosuppressive therapy with a single drug,” notes Dr. Turmelle. “Since our program started, our longest-surviving patient is 23-years-old. There’s really no limitation to their survival.”