The longstanding issue of the benefit of ursodeoxycholic acid in primary sclerosing cholangitis: a long-term population-based study

Background Although ursodeoxycholic acid (UDCA) is commonly prescribed for primary sclerosing cholangitis (PSC), a beneficial long-term effect on solid clinical endpoints has not yet been established. This study evaluated the efficacy of UDCA on long-term transplant-free survival in a longitudinal population-based cohort. Methods We conducted a retro/prospective study between January 2008 and August 2020, using data from the Dutch population-based EpiPSC2 cohort. Medication use was collected through medical chart review, periodic questionnaires, and the national pharmacy prescription database. The effect of UDCA use was assessed as time-dependent variable using a Cox proportional hazards model. Sex, age at diagnosis, PSC type, IBD status, transplant center inclusion, and PSC diagnosis year were considered as covariates. Results Medication data were available for 739 cases. Individuals with uncertain use of UDCA were excluded, resulting in a study population of 527. No discernable effect of UDCA use was observed on the endpoints liver transplantation (LT)/all-cause mortality [adjusted HR 1.13 (95%CI 0.76–1.67)] nor LT/PSC-related death/occurrence of hepatobiliary malignancy [adjusted HR 1.07 (95%CI 0.71–1.60)]. The HR of UDCA use was numerically reduced for hepatobiliary malignancy [0.68 (95%CI 0.34–1.37)]. Conclusion Our study failed to demonstrate that UDCA use impacts long-term outcomes in patients with PSC.