Validation of Risk Scoring Systems in Ursodeoxycholic Acid-Treated Patients with Primary Biliary Cholangitis

Cumali Efe, Koray Taşçilar, Ida Henriksson, Ellina Lytvyak, Fatema Alalkim, Hirsh Trivedi, Fatih Eren, Johanna Eliasson, Benedetta Terziroli Beretta-Piccoli, Janett Fischer, Ali Riza Calişkan, Maneerat Chayanupatkul, Claudia Coppo, Henriette Ytting, Tugrul Purnak, Luigi Muratori, Mårten Werner, Paolo Muratori, Fredrik Rorsman, Kristina ÖnnerhagFulya Günşar, Emma Nilsson, Alexandra Heurgué-Berlot, Fatih Güzelbulut, Nurhan Demir, Can Gönen, David Semela, Murat Aladaǧ, Murat Kiyici, Thomas D. Schiano, Aldo J. Montano-Loza, Thomas Berg, Ersan Ozaslan, Eric M. Yoshida, Alan Bonder, Hanns Ulrich Marschall, Staffan Wahlin

Research output: Contribution to journalArticlepeer-review

21 Scopus citations

Abstract

INTRODUCTION:Risk stratification based on biochemical variables is a useful tool for monitoring ursodeoxycholic acid (UDCA)-treated patients with primary biliary cholangitis (PBC). Several UDCA response criteria and scoring systems have been proposed for risk prediction in PBC, but these have not been validated in large external cohorts.METHODS:We performed a study on data of 1746 UDCA-treated patients with PBC from 25 centers in Europe, United States, and Canada. The prognostic performance of the risk scoring systems (GLOBE and UK-PBC) and the UDCA response criteria (Barcelona, Paris I, Paris II, Rotterdam, and Toronto) were evaluated. We regarded cirrhosis-related complications (ascites, variceal bleeding, and/or hepatic encephalopathy) as clinical end points.RESULTS:A total of 171 patients reached a clinical end point during a median 7 years (range 1-16 years) of follow-up. The 5-, 10- and 15-year adverse outcome-free survivals were 95%, 85%, and 77%. The GLOBE and UK-PBC scores predicted cirrhosis-related complications better than the UDCA response criteria. The hazard ratio (HR) for a 1 standard deviation increase was HR 5.05 (95% confidence interval (CI): 4.43-5.74, P < 0.001) for the GLOBE score and HR 3.39 (95% CI: 3.10-3.72, P < 0.001) for the UK-PBC score. Overall, the GLOBE and UK-PBC risk scores showed similar and excellent prognostic performance (C-statistic, 0.93; 95% CI: 0.91%-95% vs 0.94; 95% CI: 0.91%-0.96%).DISCUSSION:In our international, multicenter PBC cohort, the GLOBE and UK-PBC risk scoring systems were good predictors of future cirrhosis-related complications.

Original languageEnglish
Pages (from-to)1101-1108
Number of pages8
JournalAmerican Journal of Gastroenterology
Volume114
Issue number7
DOIs
StatePublished - Jul 2019

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