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Abstract Details
UK-wide Multicenter Evaluation of Second-line Therapies in Primary Biliary Cholangitis
Clin Gastroenterol Hepatol. 2022 Aug 9;S1542-3565(22)00738-8.doi: 10.1016/j.cgh.2022.07.038. Online ahead of print.
1National 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.
2Oxford Liver Unit, John Radcliffe Hospital, Oxford, United Kingdom.
3Institute of Liver and Digestive Health, University College London, London, United Kingdom.
4Department of Informatics, University Hospitals Birmingham, United Kingdom.
5Department 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.
6Department of Gastroenterology and Hepatology, Queen Alexandra Hospital, Portsmouth, United Kingdom.
7Clinical and Experimental Sciences, Faculty of Medicine, University of Southampton, Southampton, United Kingdom.
8Department of Hepatology, Barts Health National Health Service Trust and Blizard Institute, Queen Mary University of London, London, United Kingdom.
9Department of Digestive Diseases, St Mary's Hospital, Imperial College London, London, United Kingdom.
10Department of Gastroenterology and Hepatology, Royal Liverpool and Broadgreen University Hospitals National Health Service Trust, Liverpool, United Kingdom.
11Department of Liver Medicine, University Hospitals Bristol National Health Service Foundation Trust, Bristol, United Kingdom.
12Department of Hepatology, Leeds Teaching Hospital National Health Service Trust, Leeds, United Kingdom.
13Academic Department of Medical Genetics, University of Cambridge, Cambridge, United Kingdom.
14PBC Foundation, United Kingdom.
15Toronto Centre for Liver Disease, University of Toronto and University Health Network, Toronto, Ontario, Canada.
16National 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: p.j.trivedi@bham.ac.uk.
Abstract
Background & aims: One-third 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/fenofibrate).
Methods: This was a nationwide observational cohort study conducted from August 2017 until June 2021.
Results: 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.
Conclusion: 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.