S2793 Real-World Clinical Outcomes of Obeticholic Acid Plus Ursodeoxycholic Acid Versus Ursodeoxycholic Acid Alone in Non-Cirrhotic Patients With Primary Biliary Cholangitis

Introduction: Clinical trials have indicated that obeticholic acid (OCA) increases pruritus in patients with primary biliary cholangitis (PBC), and guidelines advise against its use in patients with cirrhosis due to potential hepatic safety concerns. Real-world data regarding OCA-associated pruritus and liver-related adverse outcomes remain limited. Thus, we aimed to examine the association of OCA use with pruritus and adverse liver-related outcomes in a real-world cohort of non-cirrhotic PBC patients. Methods: We performed a retrospective, propensity-score matched cohort study using the TriNetX database, comparing non-cirrhotic PBC patients treated with OCA plus ursodeoxycholic acid (UDCA, n = 666) versus UDCA alone (n = 666). Primary outcomes assessed over a 1-year period included the incidence of pruritus, jaundice, all-cause hospitalizations, and all-cause mortality. Outcomes were reported using risk ratios (RR) with 95% confidence intervals (CI). Results: Patients receiving OCA + UDCA had a significantly higher incidence of pruritus compared to those on UDCA monotherapy (8.4% vs 4.2%; RR 2.00; 95% CI, 1.29–3.11; P = 0.002). No significant difference was observed in the incidence of jaundice between groups (1.5% each; RR 1.00; 95% CI, 0.42–2.39; P = 1.000). Additionally, OCA + UDCA therapy was associated with significantly fewer hospitalizations than UDCA alone (4.1% vs 11.0%; RR 0.37; 95% CI, 0.24–0.57; P < 0.001). All-cause mortality rates did not differ significantly between groups (1.5% vs 2.4%; RR 0.63; 95% CI, 0.29–1.37; P = 0.235). Conclusion: This real-world analysis of non-cirrhotic PBC patients adds observational data indicating an increased association of pruritus with OCA use, aligning with previous clinical trial findings. No differences in jaundice or mortality were identified; however, OCA was associated with fewer hospitalizations. These results highlight the need for careful monitoring of pruritus and consideration of individual patient tolerability when prescribing OCA. Further prospective studies are necessary to better understand the hepatic safety profile of OCA in routine clinical practice.