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
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- Marco Carbone
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
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- Stephen J. Sharp
- MRC Epidemiology Unit, University of Cambridge,Cambridge,United Kingdom
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- Steve Flack
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
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- Dimitrios Paximadas
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
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- Kelly Spiess
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
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- Carolyn Adgey
- Liver Unit, Royal Victoria Hospital,Belfast,United Kingdom
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- Laura Griffiths
- Institute of Cellular Medicine, Newcastle University,Newcastle upon Tyne,United Kingdom
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- Reyna Lim
- Liver Unit, Queen Elizabeth Hospital,Birmingham,United Kingdom
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- Paul Trembling
- Liver Unit, Barts and the London NHS Trust,London,United Kingdom
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- Kate Williamson
- Translational Gastroenterology Unit, John Radcliffe Hospital,Oxford,United Kingdom
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- Nick J. Wareham
- MRC Epidemiology Unit, University of Cambridge,Cambridge,United Kingdom
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- Mark Aldersley
- Liver Unit, St James's University Hospital,Leeds,United Kingdom
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- Andrew Bathgate
- Scottish Liver Transplant Unit, Royal Infirmary of Edinburgh,Edinburgh,United Kingdom
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- Andrew K. Burroughs
- Sheila Sherlock Liver Center, The Royal Free London NHS Foundation Trust,London,United Kingdom
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- Michael A. Heneghan
- Institute of Liver Studies, King's College Hospital NHS Foundation Trust,London,United Kingdom
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- James M. Neuberger
- Liver Unit, Queen Elizabeth Hospital,Birmingham,United Kingdom
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- Douglas Thorburn
- Sheila Sherlock Liver Center, The Royal Free London NHS Foundation Trust,London,United Kingdom
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- Gideon M. Hirschfield
- Center for Liver Research and NIHR Biomedical Research Unit, University of Birmingham,Birmingham,United Kingdom
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- Heather J. Cordell
- Institute of Genetic Medicine, Newcastle University,Newcastle upon Tyne,United Kingdom
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- Graeme J. Alexander
- Division of Gastroenterology and Hepatology,Addenbrooke's Hospital,Cambridge,United Kingdom
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- David E.J. Jones
- Institute of Cellular Medicine, Newcastle University,Newcastle upon Tyne,United Kingdom
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- Richard N. Sandford
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
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- George F. Mells
- Academic Department of Medical Genetics, University of Cambridge,Cambridge,United Kingdom
抄録
<jats:p>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). <jats:italic toggle="yes">Conclusions</jats:italic>: 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. (H<jats:sc>epatology</jats:sc> 2016;63:930–950)</jats:p>
収録刊行物
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- Hepatology
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Hepatology 63 (3), 930-950, 2015-10-20
Ovid Technologies (Wolters Kluwer Health)