Greater Transplant-Free Survival in Patients Receiving Obeticholic Acid for Primary Biliary Cholangitis in a Clinical Trial Setting Compared to Real-World External Controls
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BACKGROUND & AIMS: The Primary Biliary Cholangitis (PBC) Obeticholic Acid (OCA) International Study of Efficacy (POISE) randomized, double-blind, placebo-controlled trial demonstrated that OCA reduced biomarkers associated with adverse clinical outcomes (ie, alkaline phosphatase, bilirubin, aspartate aminotransferase, and alanine aminotransferase) in patients with PBC. The objective of this study was to evaluate time to first occurrence of liver transplantation or death in patients with OCA in the POISE trial and open-label extension vs comparable non-OCA-treated external controls. METHODS: Propensity scores were generated for external control patients meeting POISE eligibility criteria from 2 registry studies (Global PBC and UK-PBC) using an index date selected randomly between the first and last date (inclusive) on which eligibility criteria were met. Cox proportional hazards models weighted by inverse probability of treatment assessed time to death or liver transplantation. Additional analyses (Global PBC only) added hepatic decompensation to the composite end point and assessed efficacy in patients with or without cirrhosis. RESULTS: During the 6-year follow-up, there were 5 deaths or liver transplantations in 209 subjects in the POISE cohort (2.4%), 135 of 1381 patients in the Global PBC control (10.0%), and 281 of 2135 patients in the UK-PBC control (13.2%). The hazard ratios (HRs) for the primary outcome were 0.29 (95% CI, 0.10-0.83) for POISE vs Global PBC and 0.30 (95% CI, 0.12-0.75) for POISE vs UK-PBC. In the Global PBC study, HR was 0.20 (95% CI, 0.03-1.22) for patients with cirrhosis and 0.31 (95% CI, 0.09-1.04) for those without cirrhosis; HR was 0.42 (95% CI, 0.21-0.85) including hepatic decompensation. CONCLUSIONS: Patients treated with OCA in a trial setting had significantly greater transplant-free survival than comparable external control patients.
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Obeticholic acid treatment was significantly associated with longer liver transplant-free survival compared with external controls from the GLOBAL PBC and UK PBC registries.
“Last, obeticholic acid treatment was significantly associated with longer liver transplant-free survival, as shown in the comparison of obeticholic acid-treated patients in the POISE trial with external controls extracted from the registry data in the GLOBAL PBC and UK PBC.93”
From Primary biliary cholangitis. (2024) · cites this paper as [93]
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References 27 · Cited by 107
- Authors
- Murillo Perez, C. F., Fisher, H., Hiu, S., Kareithi, D., Adekunle, F., Mayne, T., Malecha, E., Ness, E., van der Meer, A. J., Lammers, W. J., Trivedi, P. J., Battezzati, P. M., Nevens, F., Kowdley, K. V., Bruns, T., Cazzagon, N., Floreani, A., Mason, A. L., Parés, A., Londoño, M.-C., Invernizzi, P., Carbone, M., Lleo, A., Mayo, M. J., Dalekos, G. N., Gatselis, N. K., Thorburn, D., Verhelst, X., Gulamhusein, A., Janssen, H. L. A., Smith, R., Flack, S., Mulcahy, V., Trauner, M., Bowlus, C. L., Lindor, K. D., Corpechot, C., Jones, D., Mells, G., Hirschfield, G. M., Wason, J., Hansen, B. E., Sturgess, R., Healey, C., Gunasekera, A., Kallis, Y., Wright, G., Mathialahan, T., Evans, R., Gasem, J., Ramanaden, D., Ward, E., Bhalme, M., Southern, P., Maggs, J., Yousif, M., Mells, G., Srivastava, B., Foxton, M., Collins, C., Prasad, Y., Porras-Perez, F., Yapp, T., Patel, M., Ede, R., Carte, M., Koss, K., Sattianayagam, P., Grimley, C., Tidbury, J., Mansour, D., Beckley, M., Hollywood, C., Ramag, J., Gordon, H., Ridpath, J., Grover, B., Abouda, G., Rees, I., Narain, M., Salam, I., Banim, P., Das, D., Matthews, H., Mohammed, F., Jones, R., Sen, S., Bird, G., Prince, M., Prasad, G., Kitchen, P., Hutchinson, J., Gupta, P., Jones, D., Shah, A., Saha, S., Pollock, K., Barclay, S., McDonald, N., Rushbrook, S., Przemioslo, R., Millar, A., Mitchell, S., Davis, A., Naqvi, A., Lee, T., Ryder, S., Collier, J., Cramp, M., Aspinal, R., Booth, J., Williams, E., Hussaini, H., Christie, J., Chaudhry, T., Thorburn, D., Mann, S., Ala, A., Maltby, J., Corbett, C., Singhal, S., Hoeroldt, B., Butterworth, J., Douglas, A., Sinha, R., Panter, S., Shearman, J., Bray, G., Roberts, M., Forton, D., Taylor, N., Jafar, W., Cowan, M., Ch'ng, C. L., Rahman, M., Wesley, E., Jain, S., Mandal, A., Wright, M., Trivedi, P., Gordon, F., Unitt, E., Austin, A., Palegwala, A., Vemala, V., Higham, A., Fraser, J., Li, A., Ramakrishnan, S., King, A., Whalley, S., Gee, I., Keld, R., Fellows, H., Gotto, J., & Millson, C.
- Affiliations
- Newcastle University (GB)
- Intercept Pharmaceuticals (United States) (US)
- Erasmus MC (NL)
- University of Milan (IT)
- KU Leuven (BE)
- Liver Institute Northwest (US)
- Universitätsklinikum Aachen (DE)
- University of Padua (IT)
- University of Alberta (CA)
- Hospital Clínic de Barcelona (ES)
- Azienda Ospedaliera San Gerardo (IT)
- University of Milano-Bicocca (IT)
- The University of Texas Southwestern Medical Center (US)
- University Hospital of Larissa (GR)
- Ghent University Hospital (BE)
- University of Toronto (CA)
- National Health Service (GB)
- University of Cambridge (GB)
- Medical University of Vienna (AT)
- University of California, Davis (US)
- Mayo Clinic in Arizona (US)
- Hôpital Saint-Antoine (FR)
- Humanitas University (IT)
- Addenbrooke's Hospital (GB)
- Corresponding author
- Hansen, Bettina E.
- ORCID
- Fisher, Holly
- Hiu, Shaun
- Kareithi, Dorcas
- Mayne, Tracy
- Lammers, Willem J.
- Battezzati, Pier Maria
- Nevens, Frederik
- Kowdley, Kris V.
- Bruns, Tony
- Cazzagon, Nora
- Floreani, Annarosa
- Mason, Andrew L.
- Parés, Albert
- Invernizzi, Pietro
- Carbone, Marco
- Mayo, Marlyn J.
- Gatselis, Nikolaos K.
- Verhelst, Xavier
- Gulamhusein, Aliya
- Janssen, Harry L.A.
- Smith, Rachel
- Mulcahy, Victoria
- Trauner, Michael
- Bowlus, Christopher L.
- Lindor, Keith D.
- Corpechot, Christophe
- Hirschfield, Gideon M.
- Wason, James
- Hansen, Bettina E.
- Kallis, Yiannis
- Wright, Gavin
- Collins, Carole
- Mansour, Dina
- Banim, Paul
- Das, Debasish
- Jones, Rebecca
- Barclay, Stephen
- McDonald, Natasha
- Rushbrook, Simon
- Przemioslo, Robert
- Millar, Andrew
- Lee, Tom
- Ryder, Stephen
- Collier, Jane
- Cramp, Matthew
- Ala, Aftab
- Hoeroldt, Barbara
- Douglas, Andrew
- Forton, Daniel
- Jafar, Wisam
- Cowan, Matthew
- Rahman, Mesbah
- Wright, Mark
- Gordon, Fiona
- Whalley, Simon
- Keld, Richard
- Millson, Charles
- Venue
- Gastroenterology
- Publisher
- Elsevier BV
- Published
- 2022-12
- Volume
- 163
- Issue
- 6
- Pages
- 1630-1642.e3
- ISSN
- 0016-5085 (ISSN-L)
- OpenAlex
- W4296777654
- Cited by
- 107
- References
- 27
- Citations by year
- 2026: 18 · 2025: 30 · 2024: 32 · 2023: 22 · 2022: 5
- FWCI
- 13.26
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Gastroenterology (publishedVersion, cc-by)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (publishedVersion, cc-by)
- Lirias (KU Leuven) (submittedVersion, cc-by)
- UEA Digital Repository (University of East Anglia) (submittedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion, other-oa)
- Archivio Istituzionale della Ricerca (Universita Degli Studi Di Milano) (submittedVersion, other-oa)
- BOA (University of Milano-Bicocca) (submittedVersion, cc-by)
- RWTH Publications (RWTH Aachen) (submittedVersion, other-oa)
- Padua Research Archive (University of Padova) (submittedVersion, cc-by)
- RWTH Publications (RWTH Aachen)
- Funders
- Newcastle upon Tyne Hospitals NHS Foundation Trust
- Oxford University Hospitals NHS Foundation Trust
- Betsi Cadwaladr University Health Board
- Intercept Pharmaceuticals
- Topics
- Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Health Sciences › Medicine › Oncology › Drug Transport and Resistance Mechanisms
- Keywords
- obeticholic acid, primary biliary cirrhosis, transplant-free survival, liver transplantation, mortality, POISE trial, external control, real-world evidence, liver cirrhosis, hepatic decompensation
- MeSH terms
- Chenodeoxycholic Acid, Humans, Liver Cirrhosis, Liver Cirrhosis, Biliary, Ursodeoxycholic Acid
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