High-dose steroids following initial surgery for pediatric biliary atresia do not help prevent the need for liver transplantation, researchers reported here.
Of the 70 children assigned to receive corticosteroids following hepatoportoenterostomy, 58.6% achieved a total bilirubin of less than 1.5 mg/dL at 6 months with his or her native liver, compared with 48.6% of the 70 children who were treated with placebo, for an adjusted relative risk of 1.14 in both the intention-to-treat analysis (95% CI 0.83-1.57, P=0.43) and in the per-protocol analysis (95% CI 0.81-1.61, P=0.44), said Jorge Bezerra, MD, of the University of Cincinnati Children's Hospital.
In the secondary endpoint, survival with the native liver at 24 months was 58.7% with steroids and 59.4% with placebo (P=0.99), Bezerra said at the annual meeting of the American Association for the Study of Liver Diseases.
"Therefore, the addition of high-dose steroids as an adjuvant treatment after hepatoportoenterostomy cannot be recommended," he said in his plenary presentation.
Bezerra noted that biliary atresia, a rapidly fibrosing cholangiopathy that obstructs the extrahepatic bile duct, is the most common cause of end-stage cirrhosis in children and the most frequent indication for pediatric liver transplantation. The primary treatment is the surgical excision of the biliary remnant followed by roux-en-y hepatoportoenterostomy, which has a reported success rate for restoring bile drainage of about 50% in the U.S.
"Despite treatment," Bezerra said, "the intrahepatic cholangiopathy progresses and more than 70% of patients ultimately require liver transplantation. Therefore, there is a need for adjunct therapies to improve survival with the native liver."
Over the years groups in the United States, and notably in Japan, have suggested use of corticosteroids as an adjuvant therapy based on their anti-inflammatory effects, he explained. Several studies have reported mixed results.
Hence, Bezerra said, "we conducted the Steroids in Biliary Atresia Randomized Trial (START) as a double-blind and placebo-controlled study, designed to determine whether the treatment with high-dose steroids after portoenterostomy is superior to the surgical procedure alone."