Budesonide or prednisone in combination with ursodeoxycholic acid in primary sclerosing cholangitis: A randomized double-blind pilot study

OBJECTIVE: PSC has characteristics of an (auto)immune-mediated disease; how ever, few studies have evaluated corticosteroid therapy for this disorder. METHODS: We performed an 8-wk double-blind randomized pilot study to assess the effects of additional treatment with 9 mg budesonide (n = 6) versus 3 mg budesonide (n = 6) versus 10 mg prednisone (n = 6) in patients who had b een treated with UDCA (mean dose, 12 mg/kg/day) for at least 5 months witho ut achieving biochemical remission. Pruritus and fatigue were evaluated usi ng visual analog scales. Serum Liver biochemistry was measured every 4 wk. At entry and at the end of the trial, adrenocorticotrophic hormone (ACTH) a nd dehydroepiandrosterone (DHEA) were measured to assess effects on the pit uitary-adrenal axis. Duodenal bile was collected for assessment of biliary corticosteroid activity. RESULTS: Pruritus decreased significantly more in the prednisone group comp ared to both the 3-mg and the 9-mg budesonide groups (p < 0.05). Alkaline p hosphatase (mean: -23.4%; p = 0.03) and IgG (mean: -16.2%; p = 0.04) decrea sed in the prednisone group, whereas bilirubin, gamma-glutamyl transferase, aspartate aminotransferase, and alanine aminotransferase did not change si gnificantly. No significant clinical or liver biochemical changes were obse rved in the 3-mg and 9-mg budesonide groups. Significantly larger drops in serum ACTH were found in the 10-mg prednisone group (-40.7%, p = 0.04) and 9-mg budesonide group (-36.6%; p = 0.02) compared to the 3-mg budesonide gr oup (+19.0%). No significant differences in percentage change in baseline v alues for DHEA between the three treatment arms were found. Mononuclear cel l proliferation assays did not demonstrate corticosteroid activity in bile. Autoimmune hepatitis was observed in one case (9 mg budesonide) when corti costeroids were tapered off. CONCLUSION: The results of this pilot study suggest only minor beneficial s hort-term effects of prednisone but not budesonide on symptoms and serum li ver tests in UDCA-treated PSC patients. (C) 2000 by Am. Cell. of Gastroente rology.