P214 Predicted risk of end stage liver disease utilizing the UK-PBC risk score in PBC patients

Abstract

Introduction The UK-PBC Study group developed and validated a long-term prognostic model of primary biliary cholangitis (PBC) based on data from ~3000 patients (pts) with PBC. The model uses albumin, platelets, alanine aminotransferase (ALT), aspartate aminotransferase (AST) or alkaline phosphatase (ALP), and total bilirubin before and after 12 months of treatment to predict the risk of end-stage liver disease (ESLD), liver-related death or liver transplant. POISE, a randomised, double-blind (DB), placebo-controlled 12-month Phase 3 trial, investigated daily 5 mg to 10 mg obeticholic acid (OCA) for the treatment of PBC. After the DB phase, 97% of pts enrolled in an open label extension (OLE) wherein all pts received OCA. This analysis assessed the change in predicted risk of ESLD with the UK-PBC model in pts who had received placebo during the DB phase and transitioned to OCA during the OLE. Methods POISE inclusion criteria: PBC diagnosis, ALP ≥1.67x upper limit of normal (ULN) and/or total bilirubin >ULN to Results At BL, the placebo group was median (interquartile range) 55 (14) years old, 93% female, 90% white, and 93% received daily UDCA at a median (interquartile range) dose of 15 (4) mg/kg. After 1 year of continued standard-of-care treatment, placebo pts demonstrated a slight increase in predicted risk of ESLD (table 1), due to worsening liver biochemistry. However, after 1 year of OCA treatment, predicted risk of ESLD at 5, 10, and 15 years was reduced to below BL levels. Furthermore, through the 60-month OLE the median risk of ESLD was sustained below BL levels. Conclusions In POISE, the UK-PBC risk score predicted a trend for increased risk of ESLD in pts with PBC treated with placebo for 12 months in addition to standard of care. Addition of OCA led to sustained improvements in serum biochemistry and reductions in predicted risk of ESLD for up to 60 months of OCA treatment.