Management of primary biliary cholangitis prior to obeticholic acid availability

BACKGROUND: Primary biliary cholangitis (PBC) is an autoimmune cholestatic liver disease with unknown etiology. The prognosis of patients affected by PBCis heterogeneous, with a relevant improvement achieved after the introduction of ursodeoxycolic acid (UDCA). Since in the last years obeticholic acid (OCA) has been approved for the combined treatment of PBC, in patient non-responders to UDCAor as monotherapy in those intolerant to UDCA, we evaluated the response to UDCAin a cohort of patients with PBCmanaged in a specialistic setting. METHODS: We included 38 UCDA-treated non-cirrhotic, early-PBCpatients. Data were retrieved from documents compiled during the annual follow-up. The response to therapy was assessed comparing the parameters of our cohort with the inclusion criteria of the POISETrial and the Paris Iand Paris IIcriteria. RESULTS: The cohort included 34/38 female patients and the average age was 65.34±10.69 years. Over 50% of the patients were affected by at least one disease associated to PBC. Using the POISEcriteria and the Paris Iand Paris IIcriteria, we identified 5, 2 and 5 non-responders, respectively. All patients with severe fibrosis had a biochemical response to UDCAaccording to the three different criteria applied. No side effect was reported. CONCLUSIONS: We confirm that UDCA is a safe and effective treatment in patients with PBC. Non-responder patients represent 13% of our population, with high risk of disease progression and complications. In this context, further therapy using OCAshould be considered.