Ursodiol for the Long-Term Treatment of Primary Biliary Cirrhosis

作者：Renée E. Poupon, Raoul Poupon, Beverley Balkau · 发表于：New England Journal of Medicine · 年份：1994 · DOI：10.1056/nejm199405123301903 · 被引用次数：499 · 研究领域：Liver Diseases and Immunity、Gallbladder and Bile Duct Disorders、Drug Transport and Resistance Mechanisms

BACKGROUND: Ursodiol (ursodeoxycholic acid) therapy leads to major improvements in patients with primary biliary cirrhosis. The benefit of long-term treatment is uncertain. METHODS: We randomly assigned 145 patients with biopsy-proved primary biliary cirrhosis to receive ursodiol (13 to 15 mg per kilogram of body weight per day) (72 patients) or placebo (73 patients). After two years of follow-up, because of the benefit from ursodiol, all patients completing the study received ursodiol in an open trial and were monitored for two more years. The end points in the assessment of efficacy were as follows: progression of disease, as defined by the presence of hyperbilirubinemia, variceal bleeding, ascites, or encephalopathy; liver transplantation or a referral for that procedure; and liver transplantation (or a referral) or death. RESULTS: Disease progressed significantly less frequently in the ursodiol group than in the placebo group (P < 0.002; relative risk, 0.28; 95 percent confidence interval, 0.12 to 0.63). The probability of liver transplantation or a referral for that procedure and the probability of transplantation or death were significantly lower in the group assigned to ursodiol than in the group assigned to placebo (for transplantation alone, P = 0.003; relative risk, 0.21; 95 percent confidence interval, 0.07 to 0.66; for transplantation or death, P = 0.005; relative risk, 0.32; 95 percent confidence interval, 0.14 to 0.74). High bilirubin levels and, to a lesser ext...