Adjuvant Medical Therapies After Portoenterostomy for Biliary Atresia: Time for a Randomized Controlled Trial

This retrospective chart review from the Primary Children’s Medical Center, University of Utah describes the outcome of 28 consecutive infants during an 8-year period with biliary atresia after portoenterostomy (Kasai operation), comparing those who received postoperative steroids with those who received standard therapy. There were 14 patients in each group assigned to protocol in a nonrandom fashion by the preference of the attending pediatric surgeon. In the steroid group, intravenous methylprednisolone was given for 7 days, at tapering daily doses of 10, 8, 6, 5, 4, 3, and 2 mg/kg/d. Oral prednisolone at 2 mg/kg/d was then given for the following 8 to 12 weeks. The steroid group also received oral ursodeoxycholic acid indefinitely at 20 mg/kg/d. Intravenous antibiotics were given for 8 to 12 weeks after surgery, followed by oral trimethoprim/sulfamethoxazole at 10 mg/kg/d. The intravenous antibiotic therapies used were either piperacillin/tazobactam plus gentamicin or cefoperazone. The comparison group received no steroids, with the exception of 2 patients who received oral prednisone at 2 mg/kg/d for 4 days at an unspecified time. A regimen of 3 to 4 days of ampicillin plus gentamicin was used perioperatively, followed by the same oral antibiotic regimen used in the steroid group. Standard therapy did not include ursodeoxycholic acid, except that the two patients who received the short courses of prednisone also received variable courses of ursodeoxycholic acid. Surgery in most patients in both groups included a 40-cm retrocolic Roux-en-Y limb. Six patients in the standard therapy group received a Roux-en-Y limb with an intussuscepting valve. Conjugated bilirubin values 3 to 4 months after surgery were significantly lower (< 1.0 mg/dL) in the steroid group (79% vs. 21%) than in the standard therapy group (P < 0.001). In the steroid group, 3 of 14 (21%) patients had a liver transplant at <1 year of age, compared with 8 of 13 (61%) in the standard group (P < 0.01). Fewer patients in the steroid group (7% vs. 53%) died during the first year of life (P < 0.001). More patients in the steroid group (71% vs. 8%) were jaundice free with native liver (P < 0.001). The mean follow-up of survivors was 3.8 years in the steroid group, compared with 6.9 years in the standard group. One child in the standard therapy group was lost to follow-up at 6 months of age.