BACKGROUND & AIMS:Ursodeoxycholic acid (UDCA) has been shown to be a safe and effective treatment for patients with primary biliary cirrhosis; however, its effect on patient survival is less certain. To study this issue, the survival of patients receiving long-termUDCA treatment was compared with that of a control group, adjusting for their risk scores based on the Mayo model. METHODS:One hundred eighty patients were randomized to receive either 13-15 mg.kg-1.day-1 UDCA (n = 89) of placebo (n = 91). After the study closure, the patients originally receiving placebo were switched to active drug, and prospective follow-up was continued for 3 years. Patients were censored at the time of transplantation, voluntary withdrawal, of crossover of the placebo group (efficacy analysis). The survival of the two groups was adjusted for risk scores at the time of entry to the study. A secondary analysis was an intent-to-treat analysis, whereby patients were followed up regardless of their voluntary withdrawal or crossover. RESULTS: At the time of analysis, the patients receiving placebo had a significantly increased risk of death and/or requiring transplantation (relative risk, 2.6; P = 0.04) compared with the UDCA-treated patients CONCLUSIONS:UDCA should be considered as a safe, effective, and life-extending treatment for patients with primary biliary cirrhosis.
RCT Entities:
BACKGROUND & AIMS:Ursodeoxycholic acid (UDCA) has been shown to be a safe and effective treatment for patients with primary biliary cirrhosis; however, its effect on patient survival is less certain. To study this issue, the survival of patients receiving long-term UDCA treatment was compared with that of a control group, adjusting for their risk scores based on the Mayo model. METHODS: One hundred eighty patients were randomized to receive either 13-15 mg.kg-1.day-1 UDCA (n = 89) of placebo (n = 91). After the study closure, the patients originally receiving placebo were switched to active drug, and prospective follow-up was continued for 3 years. Patients were censored at the time of transplantation, voluntary withdrawal, of crossover of the placebo group (efficacy analysis). The survival of the two groups was adjusted for risk scores at the time of entry to the study. A secondary analysis was an intent-to-treat analysis, whereby patients were followed up regardless of their voluntary withdrawal or crossover. RESULTS: At the time of analysis, the patients receiving placebo had a significantly increased risk of death and/or requiring transplantation (relative risk, 2.6; P = 0.04) compared with the UDCA-treated patients CONCLUSIONS: UDCA should be considered as a safe, effective, and life-extending treatment for patients with primary biliary cirrhosis.
Authors: Burton Combes; Velimir A Luketic; Marion G Peters; Rowen K Zetterman; Guadalupe Garcia-Tsao; Santiago J Munoz; Danyu Lin; Nancy Flye; Robert L Carithers Journal: Am J Gastroenterol Date: 2004-02 Impact factor: 10.864