Fibrosis stage is an independent predictor of outcome in primary biliary cholangitis despite biochemical treatment response

• DOI: 10.1111/apt.15533
• Publication Date: 2019-10-17T09:19:05Z
• Authors: Carla F. Murillo ...; Gideon M. Hirschf...; Christophe Corpechot; Annarosa Floreani; Marlyn J. Mayo; Adriaan van der Meer; Cyriel Y. Ponsioen; Willem J. Lammers; Albert Parés; Pietro Invernizzi; Marco Carbone; Pier Maria Battez...; Frederik Nevens; Kris V. Kowdley; Douglas Thorburn; Andrew L. Mason; Palak J. Trivedi; Keith D. Lindor; Tony Bruns; George N. Dalekos; Nikolaos K. Gatselis; Xavier Verhelst; Harry L.A. Janssen; Bettina E. Hansen; Aliya Gulamhusein
• Keywords: Adult; Liver Cirrhosis; Male; EMC MM-04-20-02-A; Liver Cirrhosis, Biliary; Biopsy; Ursodeoxycholic Acid; Middle Aged; Prognosis; Severity of Illness Index; Biomarkers, Pharmacological; 3. Good health; Cohort Studies; 03 medical and health sciences; Treatment Outcome; 0302 clinical medicine; Liver; Liver Function Tests; Predictive Value of Tests; Disease Progression; Humans; Female

SummaryBackgroundFibrosis stage predicts prognosis in patients with chronic liver disease independent of aetiology, although its precise role in risk stratification in patients with primary biliary cholangitis (PBC) remains undefined.AimTo assess the utility of baseline fibrosis stage in predicting long‐term outcomes in the context of biochemical risk stratificationMethodsIn a large and globally representative cohort of patients with PBC, liver biopsies performed from 1980 to 2014 were evaluated. The predictive ability of histologic fibrosis stage in addition to treatment response at 1 year (Toronto/Paris‐II criteria), as well as non‐invasive markers of fibrosis (AST/ALT ratio [AAR], AST to platelet ratio index [APRI], FIB‐4), for transplant‐free survival was assessed with Cox proportional‐hazards models.ResultsThere were 1828 patients with baseline liver biopsy. Advanced histologic fibrosis (stage 3/4) was an independent predictor of survival in addition to non‐invasive measures of fibrosis with the hazard ratios ranging from 1.59 to 2.73 (P < .001). Patients with advanced histologic fibrosis stage had worse survival despite biochemical treatment response, with a 10‐year survival of 76.0%‐86.6% compared to 94.5%‐95.1% depending on the treatment response criteria used. Poor correlations were observed between non‐invasive measures of fibrosis and histologic fibrosis stage.ConclusionAssessment of fibrosis stage grants prognostic value beyond biochemical treatment response at 1 year. This highlights the need to incorporate fibrosis stage in individual risk stratification in patients with PBC, partly to identify those that may derive benefit from novel therapies.