O24\u2005Durability of obeticholic acid response in PBC patients who did not achieve poise trial criteria

Abstract

Introduction In clinical studies and in clinical practice, response to primary biliary cholangitis (PBC) treatment has been assessed using dichotomous biochemical response criteria. Although achieving these criteria may be associated with improved clinical outcomes, the benefit in patients (pts) with an incomplete response to treatment may be underestimated. This analysis assessed the extent and durability of obeticholic acid (OCA) response in pts with PBC not achieving the dichotomous primary endpoint in the phase 3 POISE study through 72 months of OCA treatment. Methods Key inclusion criteria included PBC diagnosis, alkaline phosphatase (ALP) ≥1.67× upper limit of normal (ULN) and/or total bilirubin >ULN to Results Of the 193 pts enrolled in the OLE, 107 (55%) did not achieve the POISE criteria after 12 months of OCA treatment. Pts were 93% female, 91% Caucasian, 56 (10) years old at BL, and 91% received UDCA (15 [4] mg/kg/day). At BL, ALP was 356 (138) U/L and total bilirubin was 13 (8) µmol/L (>ULN in 18 pts [17%]). Despite not achieving the POISE criteria after 12 months of OCA, a significant and durable reduction was observed in ALP (p Conclusions Despite not achieving the POISE primary endpoint, these pts showed significant and sustained biochemical improvements.