4CPS-084 Efficacy of obeticholic acid in patients with primary biliary cholangitis: a multicentre study in real-life

<h3>Background and Importance</h3> The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the revocation of the marketing authorisation for obeticholic acid (OA). Efficacy in primary biliary cholangitis (PBC) could not be confirmed with available evidence. <h3>Aim and Objectives</h3> To assess the efficacy of OA treatment in patients diagnosed with PBC in our region. <h3>Material and Methods</h3> A multicentric, retrospective and descriptive study was carried out from June 2017 to September 2024. Demographic and clinical variables colleted were age, sex, previous treatment with ursodeoxycholic acid (UDCA), treatment duration and analytical data of liver markers [alkaline phosphatase (ALP), total bilirubin (TB), gamma-glutamyl transferase (GGT), aspartate aminotransferase (AST) and alanine aminotransferase (ALT)] at the beginning of treatment with OA and at 12 months. The primary endpoint, based on the pivotal trial, was a composite variable defined as the percentage of patients who achieve ALP&lt;1.67 times upper limit of normal (ULN), normal total bilirubin level, and a decrease in ALP≥15% at 12 months. Secondary variables included the percentage of patients who had plasma levels ULN of GGT, AST and ALT after 12 months. <h3>Results</h3> Sixty-two patients were included with median age 61(35–88) years. There were 83.9% women and 16.1% men. All patients were treated with UDCA previously. The median treatment time with OA was 36 months. The median of basal liver markers values were: 233(108–813) UI/L ALP, 0.5(0.2–1.5) mg/dL TB, 173(19–981) UI/L GGT, 40(17–103) UI/L AST and 38(11–118) UI/L ALT. The median of liver markers values at 12 months were: 180(56–815) UI/L ALP, 0.49(0.3–1.5) mg/dL TB, 61(12–790) UI/L GGT, 30(12–97) UI/L AST and 26(9–137) UI/L ALT. At 12 months: ALP&lt;1.67 times ULN was achieve by 58.1% of patients, 66.1% of cases showed a normal total bilirubin level and a reduction in ALP≥15% was reached by 54.8% of the population. After 12 months, 20.9% of patients presented plasma levels ULN of GGT, AST and ALT. <h3>Conclusion and Relevance</h3> According to clinical trials, OA improved the analytical parameters of patients in our region after 12 months. However, a longer follow-up period is needed in our study to detect the impact on robust variables such as death or liver transplantation. <h3>References and/or Acknowledgements</h3> <h3>Conflict of Interest</h3> No conflict of interest