Oral budesonide in the treatment of primary sclerosing cholangitis

OBJECTIVE: This study was designed to evaluate the safety and estimate the efficacy of oral budesonide in patients with primary sclerosing cholangitis (PSC). METHODS: Twenty-one patients with PSC were treated with 9 mg daily of oral budesonide for 1 yr. RESULTS: Significant, but marginally important, improvement in serum alkali ne phosphatase (1235 +/- 190 vs 951 +/- 206 U/L, p = 0.003) and AST levels (119 +/- 14 vs 1.03 +/- 19 U/L, p = 0.02) was noted at the end of the treat ment period. Serum bilirubin levels increased significantly in the 18 patie nts who completed 1 yr of treatment (1.1 +/- 10.1 vs 1.4 +/- 0.3, p = 0.01) and no significant changes in liver tests were noted 3 months after budeso nide was discontinued. The Mayo risk score did not change significantly, an d although a significant improvement in the degree of portal inflammation w as noted at the end of the treatment period, the degree of fibrosis and sta ge of disease were not significantly affected. There was a marked loss of b one mass of the femoral neck (0.851 +/- 0.02 vs 0.826 +/- 0.02 g/cm(2), p = 0.002) and lumbar spine (1.042 +/- 0.02 vs 1.029 +/- 0.02 g/cm(2), p = 0.0 9) at 1 yr of treatment with budesonide. Two patients required evaluation f or liver transplantation during treatment, and two patients developed cosme tic side effects. CONCLUSIONS: Oral budesonide appears to be of minimal, if any, benefit and it is associated with a significant worsening of osteoporosis in patients w ith PSC. (Am J Gastroenterol 2000;95:2333-2337. (C) 2000 by Am. Coll. of Ga stroenterology).