Dose Reduction of Ursodeoxycholic Acid in Primary Biliary Cholangitis Patients With Complete Biochemical Response: An Open-Label Randomized Clinical Trial.

BACKGROUND AND

aimUrsodeoxycholic acid (UDCA) is first-line therapy for primary biliary cholangitis (PBC), evidence for dose reduction after biochemical remission remains limited. This study aimed to evaluate the feasibility and safety of UDCA reduction in PBC.

methodsIn this open-label randomized controlled clinical trial, PBC patients who reached complete biochemical response (Paris-II criteria) after ≥ 6 months of standard 13-15 mg/kg/day UDCA therapy were prospectively enrolled. Eligible patients were assigned in a 1:1:1 ratio to receive either (1) continued 13-15 mg/kg/day, (2) 10 mg/kg/day, or (3) 5-mg/kg/day UDCA. The primary endpoint was biochemical relapse rate within 12 months. Secondary endpoints included changes in liver biochemistry, changes in patient-reported symptoms, and changes in GLOBE and UK-PBC scores, as well as adverse events.

resultsFrom January 2020 to March 2024, 96 patients who complied with the protocol after randomization were analyzed. Within the 12-month follow-up period, the relapse rates in the three groups were 0%, 2.9%, and 19.4%, respectively (overall p = 0.008). Secondary outcomes showed no significant intergroup differences. In patients with a disease relapse, prompt retreatment with a standard dose of UDCA could help liver function recover. No severe adverse events were observed.

conclusionsThe relapse rate in the 5-mg/kg/day group was significantly higher than that in the standard-dose group, necessitating close clinical monitoring, and no significant difference in relapse was observed between the 10 mg/kg/day and standard-dose group, suggesting it may be a potential dose-reduction strategy, though further studies are needed for confirmation.