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

doi:10.1136/gutjnl-2019-319057

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 BuurenAdriaan J van der Meer

Immunology and Immunotherapy

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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 (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.

Original language	English
Journal	Gut
Early online date	16 Dec 2019
DOIs	https://doi.org/10.1136/gutjnl-2019-319057
Publication status	E-pub ahead of print - 16 Dec 2019

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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. (2019). Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis. Gut. Advance online publication. https://doi.org/10.1136/gutjnl-2019-319057

@article{25bdc44d00f646dd8fa728e19f77ca6c,

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 (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.",

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 Buuren, {Henk R van} and {van der Meer}, {Adriaan J}",

note = "{\textcopyright} Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY. Published by BMJ.",

year = "2019",

month = dec,

day = "16",

doi = "10.1136/gutjnl-2019-319057",

language = "English",

journal = "Gut",

issn = "0017-5749",

publisher = "BMJ Publishing Group",

}

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, Buuren, HRV & van der Meer, AJ 2019, 'Number needed to treat with ursodeoxycholic acid therapy to prevent liver transplantation or death in primary biliary cholangitis', Gut. https://doi.org/10.1136/gutjnl-2019-319057

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 - Buuren, Henk R van

AU - van der Meer, Adriaan J

PY - 2019/12/16

Y1 - 2019/12/16

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 (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.

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 (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.

U2 - 10.1136/gutjnl-2019-319057

DO - 10.1136/gutjnl-2019-319057

M3 - Article

C2 - 31843787

SN - 0017-5749

JO - Gut

JF - Gut

ER -

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

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