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

the GLOBAL PBC Study Group

Research output: Contribution to journalArticle

2 Citations (Scopus)

Abstract

Background: Fibrosis 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. Aim: To assess the utility of baseline fibrosis stage in predicting long-term outcomes in the context of biochemical risk stratification. Methods: In 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. Results: There 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. Conclusion: Assessment 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.

Original languageEnglish (US)
Pages (from-to)1127-1136
Number of pages10
JournalAlimentary Pharmacology and Therapeutics
Volume50
Issue number10
DOIs
StatePublished - Nov 1 2019

Fingerprint

Cholangitis
Fibrosis
Therapeutics
Survival
Patient Advocacy
Biopsy
Organized Financing
Liver
Paris
Proportional Hazards Models
Liver Diseases
Chronic Disease
Blood Platelets
Transplants

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology
  • Pharmacology (medical)

Cite this

Fibrosis stage is an independent predictor of outcome in primary biliary cholangitis despite biochemical treatment response. / the GLOBAL PBC Study Group.

In: Alimentary Pharmacology and Therapeutics, Vol. 50, No. 10, 01.11.2019, p. 1127-1136.

Research output: Contribution to journalArticle

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title = "Fibrosis stage is an independent predictor of outcome in primary biliary cholangitis despite biochemical treatment response",
abstract = "Background: Fibrosis 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. Aim: To assess the utility of baseline fibrosis stage in predicting long-term outcomes in the context of biochemical risk stratification. Methods: In 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. Results: There 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. Conclusion: Assessment 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.",
author = "{the GLOBAL PBC Study Group} and {Murillo Perez}, {Carla F.} and Hirschfield, {Gideon M.} and Christophe Corpechot and Annarosa Floreani and Mayo, {Marlyn J.} and {van der Meer}, Adriaan and Ponsioen, {Cyriel Y.} and Lammers, {Willem J.} and Albert Par{\'e}s and Pietro Invernizzi and Marco Carbone and {Maria Battezzati}, Pier and Frederik Nevens and Kowdley, {Kris V.} and Douglas Thorburn and Mason, {Andrew L.} and Trivedi, {Palak J.} and Lindor, {Keith D.} and Tony Bruns and Dalekos, {George N.} and Gatselis, {Nikolaos K.} and Xavier Verhelst and Janssen, {Harry L.A.} and Hansen, {Bettina E.} and Aliya Gulamhusein",
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language = "English (US)",
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T1 - Fibrosis stage is an independent predictor of outcome in primary biliary cholangitis despite biochemical treatment response

AU - the GLOBAL PBC Study Group

AU - Murillo Perez, Carla F.

AU - Hirschfield, Gideon M.

AU - Corpechot, Christophe

AU - Floreani, Annarosa

AU - Mayo, Marlyn J.

AU - van der Meer, Adriaan

AU - Ponsioen, Cyriel Y.

AU - Lammers, Willem J.

AU - Parés, Albert

AU - Invernizzi, Pietro

AU - Carbone, Marco

AU - Maria Battezzati, Pier

AU - Nevens, Frederik

AU - Kowdley, Kris V.

AU - Thorburn, Douglas

AU - Mason, Andrew L.

AU - Trivedi, Palak J.

AU - Lindor, Keith D.

AU - Bruns, Tony

AU - Dalekos, George N.

AU - Gatselis, Nikolaos K.

AU - Verhelst, Xavier

AU - Janssen, Harry L.A.

AU - Hansen, Bettina E.

AU - Gulamhusein, Aliya

PY - 2019/11/1

Y1 - 2019/11/1

N2 - Background: Fibrosis 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. Aim: To assess the utility of baseline fibrosis stage in predicting long-term outcomes in the context of biochemical risk stratification. Methods: In 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. Results: There 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. Conclusion: Assessment 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.

AB - Background: Fibrosis 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. Aim: To assess the utility of baseline fibrosis stage in predicting long-term outcomes in the context of biochemical risk stratification. Methods: In 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. Results: There 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. Conclusion: Assessment 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.

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