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UK-Wide Multicenter Evaluation of Second-line Therapies in Primary Biliary Cholangitis.

Authors
  • Abbas, Nadir1
  • Culver, Emma L2
  • Thorburn, Douglas3
  • Halliday, Neil3
  • Crothers, Hannah4
  • Dyson, Jessica K5
  • Phaw, April5
  • Aspinall, Richard6
  • Khakoo, Salim I7
  • Kallis, Yiannis8
  • Smith, Belinda9
  • Patanwala, Imran10
  • McCune, Anne11
  • Chimakurthi, Chenchu R12
  • Hegade, Vinod12
  • Orrell, Michael2
  • Jones, Rebecca12
  • Mells, George13
  • Thain, Colette14
  • Thain, Robert-Mitchell14
  • And 3 more
  • 1 National Institute for Health and Care Research Birmingham Biomedical Research Centre, Centre for Liver and Gastroenterology Research, University of Birmingham, United Kingdom; Liver Unit, University Hospitals Birmingham Queen Elizabeth. Birmingham, United Kingdom; Institute of Immunology and Immunotherapy, University of Birmingham, United Kingdom. , (United Kingdom)
  • 2 Oxford Liver Unit, John Radcliffe Hospital, Oxford, United Kingdom. , (United Kingdom)
  • 3 Institute of Liver and Digestive Health, University College London, London, United Kingdom. , (United Kingdom)
  • 4 Department of Informatics, University Hospitals Birmingham, United Kingdom. , (United Kingdom)
  • 5 Department of Hepatology, Newcastle upon Tyne Hospital National Health Service Foundation Trust, Newcastle, United Kingdom; National Institute for Health and Care Research, Newcastle Biomedical Research Centre, Newcastle University, Newcastle, United Kingdom. , (United Kingdom)
  • 6 Department of Gastroenterology and Hepatology, Queen Alexandra Hospital, Portsmouth, United Kingdom. , (United Kingdom)
  • 7 Clinical and Experimental Sciences, Faculty of Medicine, University of Southampton, Southampton, United Kingdom. , (United Kingdom)
  • 8 Department of Hepatology, Barts Health National Health Service Trust and Blizard Institute, Queen Mary University of London, London, United Kingdom. , (United Kingdom)
  • 9 Department of Digestive Diseases, St Mary's Hospital, Imperial College London, London, United Kingdom. , (United Kingdom)
  • 10 Department of Gastroenterology and Hepatology, Royal Liverpool and Broadgreen University Hospitals National Health Service Trust, Liverpool, United Kingdom. , (United Kingdom)
  • 11 Department of Liver Medicine, University Hospitals Bristol National Health Service Foundation Trust, Bristol, United Kingdom. , (United Kingdom)
  • 12 Department of Hepatology, Leeds Teaching Hospital National Health Service Trust, Leeds, United Kingdom. , (United Kingdom)
  • 13 Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom. , (United Kingdom)
  • 14 PBC Foundation, United Kingdom. , (United Kingdom)
  • 15 Toronto Centre for Liver Disease, University of Toronto and University Health Network, Toronto, Ontario, Canada. , (Canada)
  • 16 National Institute for Health and Care Research Birmingham Biomedical Research Centre, Centre for Liver and Gastroenterology Research, University of Birmingham, United Kingdom; Liver Unit, University Hospitals Birmingham Queen Elizabeth. Birmingham, United Kingdom; Institute of Immunology and Immunotherapy, University of Birmingham, United Kingdom; Institute of Applied Health Research, University of Birmingham, United Kingdom. Electronic address: [email protected]. , (United Kingdom)
Type
Published Article
Journal
Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association
Publication Date
Jun 01, 2023
Volume
21
Issue
6
Identifiers
DOI: 10.1016/j.cgh.2022.07.038
PMID: 35961518
Source
Medline
Keywords
Language
English
License
Unknown

Abstract

Thirty-to-forty percent of patients with primary biliary cholangitis inadequately respond to ursodeoxycholic acid. Our aim was to assemble national, real-world data on the effectiveness of obeticholic acid (OCA) as a second-line treatment, alongside non-licensed therapy with fibric acid derivatives (bezafibrate or fenofibrate). This was a nationwide observational cohort study conducted from August 2017 until June 2021. We accrued data from 457 patients; 349 treated with OCA and 108 with fibric acid derivatives. At baseline/pre-treatment, individuals in the OCA group manifest higher risk features compared with those taking fibric acid derivatives, evidenced by more elevated alkaline phosphatase values, and a larger proportion of individuals with cirrhosis, abnormal bilirubin, prior non-response to ursodeoxycholic acid, and elastography readings >9.6kPa (P < .05 for all). Overall, 259 patients (OCA) and 80 patients (fibric acid derivatives) completed 12 months of second-line therapy, yielding a dropout rate of 25.7% and 25.9%, respectively. At 12 months, the magnitude of alkaline phosphatase reduction was 29.5% and 56.7% in OCA and fibric acid groups (P < .001). Conversely, 55.9% and 36.4% of patients normalized serum alanine transaminase and bilirubin in the OCA group (P < .001). The proportion with normal alanine transaminase or bilirubin values in the fibric acid group was no different at 12 months compared with baseline. Twelve-month biochemical response rates were 70.6% with OCA and 80% under fibric acid treatment (P = .121). Response rates between treatment groups were no different on propensity-score matching or on sub-analysis of high-risk groups defined at baseline. Across the population of patients with primary biliary cholangitis in the United Kingdom, rates of biochemical response and drug discontinuation appear similar under fibric acid and OCA treatment. Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.

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