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

Publication

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

Journal Paper/Review - Jul 1, 2019

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

Citation

Efe C, Günşar F, Nilsson E, Heurgué-Berlot A, Güzelbulut F, Demir N, Gönen C, Semela D, Aladağ M, Kiyici M, Schiano T, Montano-Loza A, Berg T, Ozaslan E, Yoshida E, Bonder A, Marschall H, Önnerhag K, Rorsman F, Muratori P, Taşçilar K, Henriksson I, Lytvyak E, Alalkim F, Trivedi H, Eren F, Eliasson J, Beretta-Piccoli B, Fischer J, Calişkan A, Chayanupatkul M, Coppo C, Ytting H, Purnak T, Muratori L, Werner M, Wahlin S. Validation of Risk Scoring Systems in Ursodeoxycholic Acid-Treated Patients With Primary Biliary Cholangitis. Am J Gastroenterol 2019; 114:1101-1108.

Type

Journal Paper/Review (English)

Journal

Am J Gastroenterol 2019; 114

Publication Date

Jul 1, 2019

Issn Electronic

1572-0241

Pages

1101-1108

Brief description/objective

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.