Background: Azathioprine is used for patients with primary biliary cirrhosis, but the therapeutic responses in randomised clinical trials have been conflicting.
Objectives: To assess the benefits and harms of azathioprine for patients with primary biliary cirrhosis.
Search strategy: Randomised clinical trials were identified by searching The Cochrane Hepato-Biliary Group Controlled Trials Register, the Cochrane Central Register of Controlled Trials (CENTRAL) in The Cochrane Library, MEDLINE, EMBASE, Science Citation Index Expanded, The Chinese Biomedical Database, and LILACS, and manual searches of bibliographies to September 2005.
Selection criteria: Randomised clinical trials comparing azathioprine versus placebo, no intervention, or another drug were included irrespective of blinding, language, year of publication, and publication status.
Data collection and analysis: Our primary outcomes were mortality, and mortality or liver transplantation. Dichotomous outcomes were reported as relative risk (RR) with 95% confidence interval (CI). Continuous outcomes were reported as weighted mean difference (WMD) or standardised mean difference (SMD). We examined the intervention effects by random-effects and fixed-effect models.
Main results: We identified two randomised clinical trials with 293 patients. Only one of the trials was regarded as having low bias risk. Azathioprine did not significantly decrease mortality (RR 0.80, 95% CI 0.49 to 1.31, 2 trials). Azathioprine did not improve pruritus at one-year intervention (RR 0.71, 95% CI 0.28 to 1.84, 1 trial), cirrhosis development, or quality of life. Patients given azathioprine experienced significantly more adverse events than patients given no intervention or placebo (RR 2.44, 95% CI 1.14 to 5.20, 2 trials). The common adverse events were rash, severe diarrhoea, and bone marrow depression.
Authors' conclusions: There is no evidence to support the use of azathioprine for patients with primary biliary cirrhosis. Researchers who are interested in performing further randomised clinical trials should be aware of the risks of adverse events.