학술논문

The UK-PBC risk scores: Derivation and validation of a scoring system for long-term prediction of end-stage liver disease in primary biliary cholangitis.
Document Type
Academic Journal
Author
Carbone M; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Division of Gastroenterology and Hepatology, Addenbrooke's Hospital, Cambridge, United Kingdom.; Sharp SJ; MRC Epidemiology Unit, University of Cambridge, Cambridge, United Kingdom.; Flack S; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Paximadas D; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Spiess K; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Adgey C; Liver Unit, Royal Victoria Hospital, Belfast, United Kingdom.; Griffiths L; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Lim R; Liver Unit, Queen Elizabeth Hospital, Birmingham, United Kingdom.; Trembling P; Liver Unit, Barts and the London NHS Trust, London, United Kingdom.; Williamson K; Translational Gastroenterology Unit, John Radcliffe Hospital, Oxford, United Kingdom.; Wareham NJ; MRC Epidemiology Unit, University of Cambridge, Cambridge, United Kingdom.; Aldersley M; Liver Unit, St James's University Hospital, Leeds, United Kingdom.; Bathgate A; Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh, Edinburgh, United Kingdom.; Burroughs AK; Sheila Sherlock Liver Center, The Royal Free London NHS Foundation Trust, London, United Kingdom.; Heneghan MA; Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, United Kingdom.; Neuberger JM; Liver Unit, Queen Elizabeth Hospital, Birmingham, United Kingdom.; Thorburn D; Sheila Sherlock Liver Center, The Royal Free London NHS Foundation Trust, London, United Kingdom.; Hirschfield GM; Center for Liver Research and NIHR Biomedical Research Unit, University of Birmingham, Birmingham, United Kingdom.; Cordell HJ; Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Alexander GJ; Division of Gastroenterology and Hepatology, Addenbrooke's Hospital, Cambridge, United Kingdom.; Jones DE; Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.; Sandford RN; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Mells GF; Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.; Division of Gastroenterology and Hepatology, Addenbrooke's Hospital, Cambridge, United Kingdom.
Source
Publisher: Wolters Kluwer Health, Inc Country of Publication: United States NLM ID: 8302946 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1527-3350 (Electronic) Linking ISSN: 02709139 NLM ISO Abbreviation: Hepatology Subsets: MEDLINE
Subject
Language
English
Abstract
Unlabelled: The biochemical response to ursodeoxycholic acid (UDCA)--so-called "treatment response"--strongly predicts long-term outcome in primary biliary cholangitis (PBC). Several long-term prognostic models based solely on the treatment response have been developed that are widely used to risk stratify PBC patients and guide their management. However, they do not take other prognostic variables into account, such as the stage of the liver disease. We sought to improve existing long-term prognostic models of PBC using data from the UK-PBC Research Cohort. We performed Cox's proportional hazards regression analysis of diverse explanatory variables in a derivation cohort of 1,916 UDCA-treated participants. We used nonautomatic backward selection to derive the best-fitting Cox model, from which we derived a multivariable fractional polynomial model. We combined linear predictors and baseline survivor functions in equations to score the risk of a liver transplant or liver-related death occurring within 5, 10, or 15 years. We validated these risk scores in an independent cohort of 1,249 UDCA-treated participants. The best-fitting model consisted of the baseline albumin and platelet count, as well as the bilirubin, transaminases, and alkaline phosphatase, after 12 months of UDCA. In the validation cohort, the 5-, 10-, and 15-year risk scores were highly accurate (areas under the curve: >0.90).
Conclusions: The prognosis of PBC patients can be accurately evaluated using the UK-PBC risk scores. They may be used to identify high-risk patients for closer monitoring and second-line therapies, as well as low-risk patients who could potentially be followed up in primary care.
(© 2015 by the American Association for the Study of Liver Diseases.)