Obeticholic Acid in the Treatment of Patients with Primary Biliary Cholangitis with Inadequate Response to Ursodeoxycholic Acid in Real Practice

OCARELIFE Study 

OCARELIFE is a French observational non-interventional multicenter phase IV study that included adult patients with PBC who had an inadequate response to ursodeoxycholic acid (UDCA) (ALP and AST > 1.5× upper limit of normal range /ULN/) and who started treatment with obeticholic acid (OCA) at doses of 5 or 10 mg. The primary parameter observed was the proportion of patients meeting the Paris II criteria (ALP and AST ≤ 1.5× ULN and normal bilirubin level) after 12 months of treatment.

Interim Evaluated Population and Data

The interim analysis involved data collected up to April 2, 2019, when 12-month treatment data were available for 50 patients. The average age of these patients was 56 years, with women making up 87%. Before entering the study, 96% of participants were taking UDCA at an average dose of 15.6 mg/kg. The median duration of PBC among them was 10 years.

Findings

Efficacy

At the study entry, 17.1% of patients met the primary observed parameter, while after 12 months of OCA treatment, this increased to 40%. The proportion of patients with ALP ≤ 1.5× ULN rose from 40.0 to 48.9%, the proportion with AST ≤ 1.5× ULN from 69.4 to 84.8%, and the proportion of patients with normal bilirubin levels from 64.4 to 81.8%. Significant improvements in liver test results were observed.

Safety

The most common adverse events were pruritus and arthralgia, with the incidence of pruritus decreasing from 41.3 to 25.0% during OCA treatment. The incidence of fatigue remained unchanged during treatment. Serious adverse events occurred in 3 patients. 

Conclusion

This real-life clinical practice study, which included patients with PBC with an inadequate response to UDCA, demonstrated that OCA treatment leads to improvements in liver parameters. After 12 months, 40% of evaluated patients met the Paris II criteria. Additionally, a reduction in the incidence of pruritus was observed. The study concluded in February 2020, and the final analysis of results for all 128 participants is currently underway.

(zza)

Source: Leroy V., Corpechot C., Dumortier J. et al. Efficacy and tolerance of obeticholic acid in patients with primary biliary cholangitis and inadequate response to ursodeoxycholic acid in real life: interim analysis of the OCARELIFE study. Poster FRI-180. Digital International Liver Congress, European Association for the Study of the Liver (EASL), 2020 Aug 28.