Ursodeoxycholic acid for primary biliary cirrhosis: Final results of a 12-year, prospective, randomized, controlled trial

OBJECTIVES: The aim of this study was to evaluate whether ursodeoxycholic acid (UDCA) has any effect on the development of liver decompensation and on survival of patients with primary biliary cirrhosis (PBC). METHODS: A total of 86 patients with compensated PBC were randomly assigned to receive UDCA (n = 43) or to remain untreated (controls, n = 43). There was no significant difference in the baseline characteristics between the two groups. Mean follow-up was 7.3 +/- 3.0 yr in the UDCA and 8.1 +/- 3.1 yr in the control group. Fourteen control patients were crossed-over to UDCA therapy after a median of 3.5 yr (range 2-8 yr), at their own request. RESULTS: Liver decompensation developed in 41 patients (22 in the UDCA and 19 in the control group) and liver death or transplantation in 33 (19 in the UDCA and 14 in the control group) patients. There was no significant difference in the probability of development of liver decompensation, liver death, or transplantation (by log-rank test) between UDCA-treated and control patients, whether by an intention-to-treat or by treatment-as-received analysis. CONCLUSIONS: UDCA was not found to have any demonstrable effect on the long-term outcome of PBC and did not improve the survival of PBC patients.