Efficacy of ursodeoxycholic acid in the treatment of primary sclerosing cholangitis in children.

Background: Ursodeoxycholic acid (UDCA) has been shown to be beneficial in reducing disease activity in adult patients with primary sclerosing cholangitis (PSC). However, there has been little published regarding PSC in children and no studies investigating the efficacy of UDCA as a treatment for PSC.

Methods: This retrospective study included 10 children who were found to have the diagnosis of PSC during the past 15 years at the Texas Children's Hospital and Herman Hospital, both in Houston, Texas. The male:female ratio was 8:2, the median age of onset was 12 years (range, 1-17 years), and eight patients had coexistent inflammatory bowel disease (IBD; six ulcerative colitis, one Crohn's disease, one unspecified). At the time of diagnosis, five patients were asymptomatic, all of whom had IBD with elevated liver enzymes and three of whom had hepatomegaly. Nine patients were treated with UDCA. The one patient who did not receive UDCA was lost to follow-up soon after diagnosis. The mean dose of UDCA was 17 mg/kg with the doses ranging from 9 to 37 mg/kg.

Results: There were no side effects from the medication recorded for any of the patients. These patients showed a significant reduction in alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, and gamma-glutamyl transferase at 1, 3, 6, 15, and 20 months after treatment.

Conclusions: This study demonstrates that children with PSC treated with UDCA have significant improvements in liver biochemical indices. However, the long-term effect of UDCA on clinical outcome is unknown.