Ursodeoxycholic acid as adjunctive therapy for problematic type 1 autoimmune hepatitis: A randomized placebo-controlled treatment trial

To evaluate the efficacy of ursodeoxycholic acid as adjunctive therapy in t ype 1 autoimmune hepatitis, 37 patients who had experienced treatment failu re, repeated relapse, or incomplete response were randomized to ursodeoxych olic acid (13-15 mg/kg daily) or placebo for 6 months in addition to their usual corticosteroid schedule. Serum aspartate transaminase (70% vs. 31%, P = .04) and alkaline phosphatase (47% vs. 7%, P = .02) levels improved more commonly in the 21 patients randomized to ursodeoxycholic acid. Mean serum levels, however, were similar before and after the treatment period. The f requency of dose reduction or corticosteroid withdrawal was comparable in b oth groups (29% versus 31%, P > .9), and clinical improvement (48% vs. 44%, P > .9) or its absence (52% vs. 56%, P > .9) occurred as commonly in patie nts receiving ursodeoxycholic acid or placebo. The modifed histological act ivity score (3.5 +/- 0.8 vs. 3.5 +/- 0.9) and the modified fibrosis score ( 2.4 +/- 0.4 vs. 2.4 +/- 0.4) were similar before and after treatment with u rsodeoxycholic acid and no different than after placebo therapy. We conclud e that ursodeoxycholic acid can improve certain laboratory tests in problem atic patients with type 1 autoimmune hepatitis when administered adjunctive ly for 6 months. Short-term therapy, however, does not facilitate reduction in the dose of corticosteroids or its withdrawal, affect clinical outcome, or reduce histological activity.