Milder disease stage in patients with primary biliary cholangitis over a 44-year period: A changing natural history

Fiorella Murillo Perez, Jorn C. Goet, Willem J. Lammers, Aliya Gulamhusein, Henk R. van Buuren, Cyriel Y. Ponsioen, Marco Carbone, Andrew Mason, Christophe Corpechot, Pietro Invernizzi, Marlyn J. Mayo, Pier Maria Battezzati, Annarosa Floreani, Albert Pares, Frederik Nevens, Kris V. Kowdley, Tony Bruns, George N. Dalekos, Douglas Thorburn, Gideon HirschfieldNicholas F. LaRusso, Keith D. Lindor, Kalliopi Zachou, Raoul Poupon, Palak J. Trivedi, Xavier Verhelst, Harry L. A. Janssen, Bettina E. Hansen

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Abstract

Changes over time in the presenting features and clinical course of patients with primary biliary cholangitis (PBC) are poorly described. We sought to describe temporal trends in patient and disease characteristics over a 44-year period across a large international PBC cohort of 4805 patients diagnosed between 1970 and 2014, from 17 centers across Europe and North America. Patients were divided into five cohorts according to their year of diagnosis: 1970-1979 (n=143), 1980-1989 (n=858), 1990-1999 (n=1754), 2000-2009 (n=1815), ≥2010 (n=235). Age at diagnosis, disease stage, response to ursodeoxycholic acid (UDCA), and clinical outcomes were compared. Mean age at diagnosis increased incrementally by 2-3 years per decade from 46.9±10.1 years in the 1970s to 57.0±12.1 years from 2010 onward (p <0.001). The female to male ratio (9:1) and anti-mitochondrial antibody positivity (90%) were not significantly variable. The proportion of patients presenting with mild biochemical disease (according to Rotterdam staging) increased from 41.3% in the 1970s to 72.2% in the 1990s (p <0.001), and remained relatively stable thereafter. Patients with a mild histological stage at diagnosis increased from 60.4% (1970-1989) to 76.5% (1990-2014) (p <0.001). Correspondingly, response to UDCA according to Paris-I criteria increased; 51.7% in the 1970s and 70.5% in the 1990s (p <0.001). Recent decades were also characterized by lower decompensation rates (18.5% in the 1970s to 5.8% in the 2000s, p <0.001) and higher 10-year transplant-free survival (48.4%, 68.7%, 79.7%, and 80.1% for each respective cohort, p <0.001). Over the last decades, there is a pattern of PBC presentation consistent with an older age at diagnosis alongside reduced disease severity. The observed trends may be explained by an increase in routine testing of liver function and/or a changing environmental trigger. This article is protected by copyright. All rights reserved
Original languageEnglish
JournalHepatology (Baltimore, Md.)
Volume67
Issue number5
Early online date6 Apr 2017
DOIs
Publication statusPublished - 2018

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