Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis
Objective: The clinical benefit of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has never been reported in absolute measures. The aim of this study was to assess the number needed to treat (NNT) with UDCA to prevent liver transplantation (LT) or death among patients with PBC. Met...
| Autores: | , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Tipo de recurso: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2020 |
| País: | España |
| Institución: | Universidad de Barcelona |
| Repositorio: | Dipòsit Digital de la UB |
| OAI Identifier: | oai:diposit.ub.edu:2445/173021 |
| Acceso en línea: | https://hdl.handle.net/2445/173021 |
| Access Level: | acceso abierto |
| Palabra clave: | Cirrosi hepàtica Mortalitat Trasplantament hepàtic Hepatic cirrhosis Mortality Hepatic transplantation |
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Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitisHarms, Maren H.Veer, Rozanne C. deLammers, Willem J.Corpechot, ChristopherThorburn, DouglasJanssen, Harry L. A.Lindor, Keith D.Trivedi, Palak J.Hirschfield, Gideon M.Parés Darnaculleta, AlbertFloreani, AnnarosaMayo, Marlyn J.Invernizzi, PietroBattezzati, Pier MariaNevens, FrederickPonsioen, Cyriel Y.Mason, Andrew L.Kowdley, Kris V.Hansen, Bettina E.van Buuren, Henk R.van der Meer, Adriaan J.Cirrosi hepàticaMortalitatTrasplantament hepàticHepatic cirrhosisMortalityHepatic transplantationObjective: The clinical benefit of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has never been reported in absolute measures. The aim of this study was to assess the number needed to treat (NNT) with UDCA to prevent liver transplantation (LT) or death among patients with PBC. Methods: The NNT was calculated based on the untreated LT-free survival and HR of UDCA with respect to LT or death as derived from inverse probability of treatment weighting-adjusted Cox proportional hazard analyses within the Global PBC Study Group database. Results: We included 3902 patients with a median follow-up of 7.8 (4.1-12.1) years. The overall HR of UDCA was 0.46 (95% CI 0.40 to 0.52) and the 5-year LT-free survival without UDCA was 81% (95% CI 79 to 82). The NNT to prevent one LT or death within 5 years (NNT5y) was 11 (95% CI 9 to 13). Although the HR of UDCA was similar for patients with and without cirrhosis (0.33 vs 0.31), the NNT5y was 4 (95% CI 3 to 5) and 20 (95% CI 14 to 34), respectively. Among patients with low alkaline phosphatase (ALP) (≤2× the upper limit of normal (ULN)), intermediate ALP (2-4× ULN) and high ALP (>4× ULN), the NNT5y to prevent one LT or death was 26 (95% CI 15 to 70), 11 (95% CI 8 to 17) and 5 (95% CI 4 to 8), respectively. Conclusion: The absolute clinical efficacy of UDCA with respect to LT or death varied with baseline prognostic characteristics, but was high throughout. These findings strongly emphasise the incentive to promptly initiate UDCA treatment in all patients with PBC and may improve patient compliance.BMJ Publishing Group2020info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/2445/173021Articles publicats en revistes (Medicina)reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésReproducció del document publicat a: https://doi.org/10.1136/gutjnl-2019-319057Gut, 2020, vol. 69, num. 8, p. 1502-1509https://doi.org/10.1136/gutjnl-2019-319057cc by (c) Harms et al., 2020http://creativecommons.org/licenses/by/3.0/es/info:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/1730212026-05-27T06:46:51Z |
| dc.title.none.fl_str_mv |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| title |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| spellingShingle |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis Harms, Maren H. Cirrosi hepàtica Mortalitat Trasplantament hepàtic Hepatic cirrhosis Mortality Hepatic transplantation |
| title_short |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| title_full |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| title_fullStr |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| title_full_unstemmed |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| title_sort |
Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis |
| dc.creator.none.fl_str_mv |
Harms, Maren H. Veer, Rozanne C. de Lammers, Willem J. Corpechot, Christopher Thorburn, Douglas Janssen, Harry L. A. Lindor, Keith D. Trivedi, Palak J. Hirschfield, Gideon M. Parés Darnaculleta, Albert Floreani, Annarosa Mayo, Marlyn J. Invernizzi, Pietro Battezzati, Pier Maria Nevens, Frederick Ponsioen, Cyriel Y. Mason, Andrew L. Kowdley, Kris V. Hansen, Bettina E. van Buuren, Henk R. van der Meer, Adriaan J. |
| author |
Harms, Maren H. |
| author_facet |
Harms, Maren H. Veer, Rozanne C. de Lammers, Willem J. Corpechot, Christopher Thorburn, Douglas Janssen, Harry L. A. Lindor, Keith D. Trivedi, Palak J. Hirschfield, Gideon M. Parés Darnaculleta, Albert Floreani, Annarosa Mayo, Marlyn J. Invernizzi, Pietro Battezzati, Pier Maria Nevens, Frederick Ponsioen, Cyriel Y. Mason, Andrew L. Kowdley, Kris V. Hansen, Bettina E. van Buuren, Henk R. van der Meer, Adriaan J. |
| author_role |
author |
| author2 |
Veer, Rozanne C. de Lammers, Willem J. Corpechot, Christopher Thorburn, Douglas Janssen, Harry L. A. Lindor, Keith D. Trivedi, Palak J. Hirschfield, Gideon M. Parés Darnaculleta, Albert Floreani, Annarosa Mayo, Marlyn J. Invernizzi, Pietro Battezzati, Pier Maria Nevens, Frederick Ponsioen, Cyriel Y. Mason, Andrew L. Kowdley, Kris V. Hansen, Bettina E. van Buuren, Henk R. van der Meer, Adriaan J. |
| author2_role |
author author author author author author author author author author author author author author author author author author author author |
| dc.subject.none.fl_str_mv |
Cirrosi hepàtica Mortalitat Trasplantament hepàtic Hepatic cirrhosis Mortality Hepatic transplantation |
| topic |
Cirrosi hepàtica Mortalitat Trasplantament hepàtic Hepatic cirrhosis Mortality Hepatic transplantation |
| description |
Objective: The clinical benefit of ursodeoxycholic acid (UDCA) in primary biliary cholangitis (PBC) has never been reported in absolute measures. The aim of this study was to assess the number needed to treat (NNT) with UDCA to prevent liver transplantation (LT) or death among patients with PBC. Methods: The NNT was calculated based on the untreated LT-free survival and HR of UDCA with respect to LT or death as derived from inverse probability of treatment weighting-adjusted Cox proportional hazard analyses within the Global PBC Study Group database. Results: We included 3902 patients with a median follow-up of 7.8 (4.1-12.1) years. The overall HR of UDCA was 0.46 (95% CI 0.40 to 0.52) and the 5-year LT-free survival without UDCA was 81% (95% CI 79 to 82). The NNT to prevent one LT or death within 5 years (NNT5y) was 11 (95% CI 9 to 13). Although the HR of UDCA was similar for patients with and without cirrhosis (0.33 vs 0.31), the NNT5y was 4 (95% CI 3 to 5) and 20 (95% CI 14 to 34), respectively. Among patients with low alkaline phosphatase (ALP) (≤2× the upper limit of normal (ULN)), intermediate ALP (2-4× ULN) and high ALP (>4× ULN), the NNT5y to prevent one LT or death was 26 (95% CI 15 to 70), 11 (95% CI 8 to 17) and 5 (95% CI 4 to 8), respectively. Conclusion: The absolute clinical efficacy of UDCA with respect to LT or death varied with baseline prognostic characteristics, but was high throughout. These findings strongly emphasise the incentive to promptly initiate UDCA treatment in all patients with PBC and may improve patient compliance. |
| publishDate |
2020 |
| dc.date.none.fl_str_mv |
2020 |
| dc.type.none.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion |
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article |
| status_str |
publishedVersion |
| dc.identifier.none.fl_str_mv |
https://hdl.handle.net/2445/173021 |
| url |
https://hdl.handle.net/2445/173021 |
| dc.language.none.fl_str_mv |
Inglés |
| language_invalid_str_mv |
Inglés |
| dc.relation.none.fl_str_mv |
Reproducció del document publicat a: https://doi.org/10.1136/gutjnl-2019-319057 Gut, 2020, vol. 69, num. 8, p. 1502-1509 https://doi.org/10.1136/gutjnl-2019-319057 |
| dc.rights.none.fl_str_mv |
cc by (c) Harms et al., 2020 http://creativecommons.org/licenses/by/3.0/es/ info:eu-repo/semantics/openAccess |
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cc by (c) Harms et al., 2020 http://creativecommons.org/licenses/by/3.0/es/ |
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openAccess |
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application/pdf |
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BMJ Publishing Group |
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BMJ Publishing Group |
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Articles publicats en revistes (Medicina) reponame:Dipòsit Digital de la UB instname:Universidad de Barcelona |
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Universidad de Barcelona |
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Dipòsit Digital de la UB |
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Dipòsit Digital de la UB |
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