Obeticholic acid (OCA) is approved for the treatment of patients with
primary biliary cholangitis (PBC) who are partial responders or intolerant to
ursodeoxycholic acid. Reports of serious liver injury have raised concerns about its safety in
cirrhosis. We investigated the effects of treatment with OCA on hepatic decompensation and liver-related mortality or
transplantation in a cohort with compensated PBC
cirrhosis. This was a retrospective cohort study using national data of US veterans with PBC and
cirrhosis. We performed a propensity score model using variables associated with OCA prescription to control for baseline risk of decompensation. New OCA users were matched to nonusers. We identified 509 subjects with compensated PBC
cirrhosis. We developed a propensity score model using variables associated with OCA prescription; 21 OCA users were matched with 84 nonusers. Over 569 and 3,847 person-months, respectively, of follow-up, 5 (23.8%) OCA users and 22 (26.2%) OCA nonusers decompensated. The C-statistic of the propensity score model was 0.87. On multivariable analysis, after adjusting for potential confounders, OCA use was associated with an increased risk of hepatic decompensation (adjusted hazard ratio, 3.9; 95% confidence interval, 1.33-11.57; P = 0.01). There was no association between OCA use and liver-related mortality or
transplantation (adjusted hazard ratio, 1.35; 95% confidence interval, 0.35-5.21; P = 0.66). Conclusion: OCA use was associated with an increase in hepatic decompensation but not liver-related mortality or
transplantation in patients with compensated PBC
cirrhosis. Additional studies are recommended to prospectively investigate these findings.