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BACKGROUND & AIMS: The Primary Biliary Cholangitis (PBC) Obeticholic Acid (OCA) International Study of Efficacy (POISE) randomized, double-blind, placebo-controlled trial demonstrated that OCA reduced biomarkers associated with adverse clinical outcomes (ie, alkaline phosphatase, bilirubin, aspartate aminotransferase, and alanine aminotransferase) in patients with PBC. The objective of this study was to evaluate time to first occurrence of liver transplantation or death in patients with OCA in the POISE trial and open-label extension vs comparable non-OCA-treated external controls. METHODS: Propensity scores were generated for external control patients meeting POISE eligibility criteria from 2 registry studies (Global PBC and UK-PBC) using an index date selected randomly between the first and last date (inclusive) on which eligibility criteria were met. Cox proportional hazards models weighted by inverse probability of treatment assessed time to death or liver transplantation. Additional analyses (Global PBC only) added hepatic decompensation to the composite end point and assessed efficacy in patients with or without cirrhosis. RESULTS: During the 6-year follow-up, there were 5 deaths or liver transplantations in 209 subjects in the POISE cohort (2.4%), 135 of 1381 patients in the Global PBC control (10.0%), and 281 of 2135 patients in the UK-PBC control (13.2%). The hazard ratios (HRs) for the primary outcome were 0.29 (95% CI, 0.10-0.83) for POISE vs Global PBC and 0.30 (95% CI, 0.12-0.75) for POISE vs UK-PBC. In the Global PBC study, HR was 0.20 (95% CI, 0.03-1.22) for patients with cirrhosis and 0.31 (95% CI, 0.09-1.04) for those without cirrhosis; HR was 0.42 (95% CI, 0.21-0.85) including hepatic decompensation. CONCLUSIONS: Patients treated with OCA in a trial setting had significantly greater transplant-free survival than comparable external control patients. ispartof: Gastroenterology vol:163 issue:6 pages:1630-1642.e3 ispartof: location:United States status: published

Anaemia is a common reason for referral to any gastroenterological service. Iron-deficiency anaemia is the most frequent, making up to 13% of referrals. The type of anaemia and any clinical symptoms often delineate the most appropriate investigations. However, iron-deficiency anaemia (IDA) can be the sole reason for referral and, depending upon patient’s age, necessitates endoscopic investigation of the gastrointestinal tract (GIT). The functional definition of anaemia is a circulating red cell mass insufficient to meet tissue oxygen requirements. However, arbitrary criteria are often used to define anaemia, the most frequently used being the WHO criteria of 13 g/dl for males, 12 g/dl for non-pregnant females and 11 g/dl for pregnant women. Anaemia results from either defective production or an increased destruction or loss of cells and can be classified by red cell indices, particularly the mean cell volume. It can be divided into three main categories: microcytic, normocytic and macrocytic. In this article we will give a brief overview of the most common presentations of anaemia relevant to the GI system.