Cumali Efe1, Koray Taşçilar2, Ida Henriksson3, Ellina Lytvyak4, Fatema Alalkim5, Hirsh Trivedi6, Fatih Eren7, Johanna Eliasson8, Benedetta Terziroli Beretta-Piccoli9, Janett Fischer10, Ali Riza Calişkan11, Maneerat Chayanupatkul12,13, Claudia Coppo14, Henriette Ytting8, Tugrul Purnak15, Luigi Muratori14, Mårten Werner16, Paolo Muratori14, Fredrik Rorsman17, Kristina Önnerhag18, Fulya Günşar19, Emma Nilsson20, Alexandra Heurgué-Berlot21, Fatih Güzelbulut22, Nurhan Demir1, Can Gönen22, David Semela23, Murat Aladağ11, Murat Kiyici7, Thomas D Schiano12, Aldo J Montano-Loza4, Thomas Berg10, Ersan Ozaslan24, Eric M Yoshida5, Alan Bonder6, Hanns-Ulrich Marschall25, Staffan Wahlin26. 1. Department of Gastroenterology, Gazi Yaşargil Education and Research Hospital, Diyarbakir, Turkey. 2. Department of Rheumatology, Istanbul Okmeydani Education and Research Hospital, Istanbul, Turkey. 3. Department of Gastroenterology, Faculty of Medicine and Health, Örebro University, Örebro, Sweden. 4. Division of Gastroenterology and Liver Unit, University of Alberta, Alberta, Canada. 5. Division of Gastroenterology, University of British Columbia and Vancouver General Hospital, Vancouver, Canada. 6. Division of GI and Hepatology, Beth Israel Medical Center, Harvard Medical School, Boston, MA,USA. 7. Department of Gastroenterology, Medical Faculty, Uludag University, Bursa, Turkey. 8. Department of Hepatology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark. 9. Department of Internal Medicine, Epatocentro Ticino, Lugano, Switzerland. 10. Section of Hepatology, Clinic for Gastroenterology, University Clinic Leipzig, Leipzig, Germany. 11. Department of Gastroenterology, Inönü University School of Medicine, Malatya, Turkey. 12. Division of Liver Diseases, the Mount Sinai Medical Center, New York, NY, USA. 13. Faculty of Medicine, Chulalongkorn University, Bangkok, Thailand. 14. Center for the Study and Treatment of Autoimmune Diseases of the Liver and Biliary System, University of Bologna, Bologna, Italy. 15. Department of Gastroenterology, Hacettepe University, Ankara, Turkey. 16. Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden. 17. Department of Gastroenterology and Hepatology, Uppsala University Hospital, Uppsala, Sweden. 18. Department of Gastroenterology and Hepatology, Skåne University Hospital, Malmö, Sweden. 19. Department of Gastroenterology, Ege University, Bornova, Izmir, Turkey. 20. Department of Clinical Sciences, Gastroenterology Division, Lund University, University Hospital Skane, Lund, Sweden. 21. Department of Hepato-Gastroenterology, CHU Reims, Reims, France. 22. Department of Gastroenterology, Haydarpaşa Numune Education and Research Hospital, İstanbul, Turkey. 23. Division of Gastroenterology and Hepatology, Kantonsspital St. Gallen, St. Gallen, Switzerland. 24. Department of Gastroenterology, Numune Research and Education Hospital, Ankara, Turkey. 25. Department of Molecular and Clinical Medicine, Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden. 26. Hepatology Division, Centre for Digestive Diseases, Karolinska Institute and Karolinska University Hospital, Stockholm, Sweden.
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.
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.
Authors: Eric M Yoshida; Mark Gordon Swain; Cynthia Tsien; Edward Tam; Robert James Bailey; Dusanka Grbic; Hin Hin Ko; Alnoor Ramji; Nir Hilzenrat; Magdy Elkhashab; Euiseok Kim; Meaghan O'Brien; Marco Amedeo Puglia; Kevork M Peltekian Journal: Can Liver J Date: 2022-08-16