Liver and Bile
Clin Gastroenterol Hepatol. 2023;21(6):1561–70.e13
UK-wide multicenter evaluation of second-line therapies in primary biliary cholangitis
Background and aims: 30–40% of patients with primary biliary cholangitis inadequately respond to ursodeoxycholic acid (UDCA). Aim of the present study was to assemble national, real-world data on the effectiveness of obeticholic acid (OCA) as a second-line treatment, alongside non-licensed therapy with fibric acid derivatives (bezafibrate or fenofibrate).
Methods: This was a nationwide observational cohort study conducted from August 2017 until June 2021.
Results: The authors accrued data from 457 patients; 349 treated with OCA and 108 with fibric acid derivatives. At baseline/pretreatment, individuals in the OCA group manifest higher risk features compared with those taking fibric acid derivatives, evidenced by more elevated alkaline phosphatase (ALP) values, and a larger proportion of individuals with cirrhosis, abnormal bilirubin, prior non-response to UDCA acid, and elastography readings > 9.6 kPa (p < 0.05 for all). Overall, 259 patients (OCA) and 80 patients (fibric acid derivatives) completed 12 months of second-line therapy, yielding a dropout rate of 25.7% and 25.9%, respectively. At 12 months, the magnitude of ALP reduction was 29.5% and 56.7% in OCA and fibric acid groups (p < 0.001). Conversely, 55.9% and 36.4% of patients normalized serum alanine transaminase (ALT) and bilirubin in the OCA group (p < 0.001). The proportion with normal ALT or bilirubin values in the fibric acid group was no different at 12 months compared with baseline. 12-month biochemical response rates were 70.6% with OCA and 80% under fibric acid treatment (p = 0.121). Response rates between treatment groups were no different on propensity-score matching or on subanalysis of high-risk groups defined at baseline.
Conclusion: Across the population of patients with primary biliary cholangitis in the United Kingdom, rates of biochemical response and drug discontinuation appear similar under fibric acid and obeticholic acid treatment.