Fenofibrate add-on therapy improves transplant-free survival in primary biliary cholangitis patients.

BACKGROUND

Fenofibrate has shown biochemical benefit in primary biliary cholangitis (PBC) patients with a suboptimal response to ursodeoxycholic acid (UDCA), but its long-term efficacy on survival remains unknown.

METHODS

In this retrospective-prospective cohort study, we enrolled 160 PBC patients with a suboptimal response to UDCA and followed up on these patients to obtain laboratory results and adverse events. Finally, we evaluated long-term survivals analyzed with Kaplan-Meier plotting and log-rank test.

RESULTS

The fenofibrate add-on therapy group showed more significant improvements in alkaline phosphatase (ALP) and gamma-glutamyl transferase levels compared with UDCA monotherapy group after 1 year of treatment, resulting in a normalization rate of 60.9% for ALP and 45.3% for both ALP and total bilirubin. Importantly, compared with UDCA monotherapy group, the fenofibrate add-on therapy group had a better transplant-free survivals of 5 (89.7 vs 75.3%) and 10 years (87.0 vs 47.6%), with a hazard ratio of 0.3282 (95% confidence interval: 0.1334-0.8073, P < 0.05). Twenty-one cases (25.6%) developed adverse events, with liver injury being the most frequent one (17.1%).

CONCLUSION

Fenofibrate add-on therapy improved not only biochemical responses but also long-term transplant-free survival in PBC patients with suboptimal response to UDCA. However, liver injury needs to be closely monitored and properly managed.