Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.

Maren H Harms; Rozanne C de Veer; Willem J Lammers; Christophe Corpechot; Douglas Thorburn; Harry L A Janssen; Keith D Lindor; Palak J Trivedi; Gideon M Hirschfield; Albert Pares; Annarosa Floreani; Marlyn J Mayo; Pietro Invernizzi; Pier Maria Battezzati; Frederik Nevens; Cyriel Y Ponsioen; Andrew L Mason; Kris V Kowdley; Bettina E Hansen; Henk R van Buuren; Adriaan J van der Meer

Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.

Maren H Harms
, Rozanne C de Veer
, Willem J Lammers
, Christophe Corpechot
, Douglas Thorburn
, Harry L A Janssen
, Keith D Lindor
, Palak J Trivedi
, Gideon M Hirschfield
, Albert Pares
, Annarosa Floreani
, Marlyn J Mayo
, Pietro Invernizzi
, Pier Maria Battezzati
, Frederik Nevens
, Cyriel Y Ponsioen
, Andrew L Mason
, Kris V Kowdley
, Bettina E Hansen
, Henk R van Buuren

Adriaan J van der Meer

Research output: Contribution to journal › Article

Abstract

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 (NNT

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.

Original language	American English
Journal	Articles, Abstracts, and Reports
State	Published - Aug 1 2020

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Harms, M. H., de Veer, R. C., Lammers, W. J., Corpechot, C., Thorburn, D., Janssen, H. L. A., Lindor, K. D., Trivedi, P. J., Hirschfield, G. M., Pares, A., Floreani, A., Mayo, M. J., Invernizzi, P., Battezzati, P. M., Nevens, F., Ponsioen, C. Y., Mason, A. L., Kowdley, K. V., Hansen, B. E., ... van der Meer, A. J. (2020). Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis. Articles, Abstracts, and Reports. http://digitalcommons.providence.org/publications/4690

@article{be51d57591e54529bb7dd30bf42e23dc,

title = "Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.",

abstract = "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 (NNT 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.",

author = "Harms, \{Maren H\} and \{de Veer\}, \{Rozanne C\} and Lammers, \{Willem J\} and Christophe Corpechot and Douglas Thorburn and Janssen, \{Harry L A\} and Lindor, \{Keith D\} and Trivedi, \{Palak J\} and Hirschfield, \{Gideon M\} and Albert Pares and Annarosa Floreani and Mayo, \{Marlyn J\} and Pietro Invernizzi and Battezzati, \{Pier Maria\} and Frederik Nevens and Ponsioen, \{Cyriel Y\} and Mason, \{Andrew L\} and Kowdley, \{Kris V\} and Hansen, \{Bettina E\} and \{van Buuren\}, \{Henk R\} and \{van der Meer\}, \{Adriaan J\}",

year = "2020",

month = aug,

day = "1",

language = "American English",

journal = "Articles, Abstracts, and Reports",

}

Harms, MH, de Veer, RC, Lammers, WJ, Corpechot, C, Thorburn, D, Janssen, HLA, Lindor, KD, Trivedi, PJ, Hirschfield, GM, Pares, A, Floreani, A, Mayo, MJ, Invernizzi, P, Battezzati, PM, Nevens, F, Ponsioen, CY, Mason, AL, Kowdley, KV, Hansen, BE, van Buuren, HR & van der Meer, AJ 2020, 'Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.', Articles, Abstracts, and Reports. <http://digitalcommons.providence.org/publications/4690>

TY - JOUR

T1 - Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.

AU - Harms, Maren H

AU - de Veer, Rozanne C

AU - Lammers, Willem J

AU - Corpechot, Christophe

AU - Thorburn, Douglas

AU - Janssen, Harry L A

AU - Lindor, Keith D

AU - Trivedi, Palak J

AU - Hirschfield, Gideon M

AU - Pares, Albert

AU - Floreani, Annarosa

AU - Mayo, Marlyn J

AU - Invernizzi, Pietro

AU - Battezzati, Pier Maria

AU - Nevens, Frederik

AU - Ponsioen, Cyriel Y

AU - Mason, Andrew L

AU - Kowdley, Kris V

AU - Hansen, Bettina E

AU - van Buuren, Henk R

AU - van der Meer, Adriaan J

PY - 2020/8/1

Y1 - 2020/8/1

N2 - 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 (NNT 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.

AB - 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 (NNT 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.

M3 - Article

JO - Articles, Abstracts, and Reports

JF - Articles, Abstracts, and Reports

ER -

Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis.

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