Ursodeoxycholic acid is associated with improved long-term outcome in patients with primary sclerosing cholangitis

Background It remains unclear whether ursodeoxycholic acid (UDCA) treatment improves long-term outcomes in patients with primary sclerosing cholangitis (PSC). In this study, we investigated whether UDCA treatment is associated with improved liver transplantation (LT)-free survival in a cohort of Japanese patients with PSC.Journal instruction requires a city and country for affiliations; however, these are missing in affiliation [6]. Please verify if the provided city and country are correct and amend if necessary.'Tokyo, Japan' is correct. Methods We used retrospective data from the Japanese PSC registry that included 435 patients with PSC. In this study, we enrolled patients with a complete dataset at diagnosis, along with the diagnosis year, treatment protocol, follow-up period, and outcome data. The association between UDCA treatment and all-cause death or LT was analyzed using Cox regression and inverse probability of UDCA treatment weighting (IPTW)-adjusted Cox regression models adjusted for covariates. Results Among 435 patients with PSC, 110 were excluded due to insufficient or missing data, and the remaining 325 patients (male, 187 (58%); mean age at diagnosis, 45.8 years) were enrolled. The mean follow-up period was 5.1 years, and 57 deaths and 24 LTs occurred during observation. UDCA was administered to 278 patients (86%). The Cox regression model demonstrated that UDCA treatment was associated with an improvement in LT-free survival [adjusted hazard ratio (aHR) 0.47, 95% confidence interval (CI) 0.28-0.78, p = 0.003]. In addition, the IPTW-adjusted model indicated a significant association between UDCA and LT-free survival (aHR 0.43, 95% CI 0.25-0.75, p = 0.020). Sensitivity analysis excluding patients treated with bezafibrate indicated a similarly significant association between UDCA treatment and LT-free survival. Conclusion In this Japanese PSC cohort, UDCA treatment was significantly associated with improved LT-free survival.

Automated summary

This study investigated the association between UDCA treatment and improved LT-free survival in a cohort of Japanese PSC patients.