Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy

Willem J. Lammers; Gideon M. Hirschfield; Christophe Corpechot; Frederik Nevens; Keith D. Lindor; Harry L A Janssen; Annarosa Floreani; Cyriel Y. Ponsioen; Marlyn J. Mayo; Pietro Invernizzi; Pier M. Battezzati; Albert Parés; Andrew K. Burroughs; Andrew L. Mason; Kris V. Kowdley; Teru Kumagi; Maren H. Harms; Palak J. Trivedi; Raoul Poupon; Angela Cheung; Ana Lleo; Llorenç Caballeria; Bettina E. Hansen; Henk R. Van Buuren

doi:10.1053/j.gastro.2015.07.061

Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy

Willem J. Lammers, Gideon M. Hirschfield, Christophe Corpechot, Frederik Nevens, Keith D. Lindor, Harry L A Janssen, Annarosa Floreani, Cyriel Y. Ponsioen, Marlyn J. Mayo, Pietro Invernizzi, Pier M. Battezzati, Albert Parés, Andrew K. Burroughs, Andrew L. Mason, Kris V. Kowdley, Teru Kumagi, Maren H. Harms, Palak J. Trivedi, Raoul Poupon, Angela CheungAna Lleo, Llorenç Caballeria, Bettina E. Hansen, Henk R. Van Buuren

Research output: Contribution to journal › Article › peer-review

219 Scopus citations

Abstract

Background & Aims Approaches to risk stratification for patients with primary biliary cirrhosis (PBC) are limited, single-center based, and often dichotomous. We aimed to develop and validate a better model for determining prognoses of patients with PBC. Methods We performed an international, multicenter meta-analysis of 4119 patients with PBC treated with ursodeoxycholic acid at liver centers in 8 European and North American countries. Patients were randomly assigned to derivation (n = 2488 [60%]) and validation cohorts (n = 1631 [40%]). A risk score (GLOBE score) to predict transplantation-free survival was developed and validated with univariate and multivariable Cox regression analyses using clinical and biochemical variables obtained after 1 year of ursodeoxycholic acid therapy. Risk score outcomes were compared with the survival of age-, sex-, and calendar time-matched members of the general population. The prognostic ability of the GLOBE score was evaluated alongside those of the Barcelona, Paris-1, Rotterdam, Toronto, and Paris-2 criteria. Results Age (hazard ratio = 1.05; 95% confidence interval [CI]: 1.04-1.06; P 0.30 had significantly shorter times of transplant-free survival than matched healthy individuals (P

Original language	English (US)
Pages (from-to)	1804-1812e4
Journal	Gastroenterology
Volume	149
Issue number	7
DOIs	https://doi.org/10.1053/j.gastro.2015.07.061
State	Published - Dec 1 2015
Externally published	Yes

Keywords

Autoimmune Liver Disease
Cholestasis
Predictive Factor
Prognosis

ASJC Scopus subject areas

Gastroenterology

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10.1053/j.gastro.2015.07.061

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Lammers, W. J., Hirschfield, G. M., Corpechot, C., Nevens, F., Lindor, K. D., Janssen, H. L. A., Floreani, A., Ponsioen, C. Y., Mayo, M. J., Invernizzi, P., Battezzati, P. M., Parés, A., Burroughs, A. K., Mason, A. L., Kowdley, K. V., Kumagi, T., Harms, M. H., Trivedi, P. J., Poupon, R., ... Van Buuren, H. R. (2015). Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy. Gastroenterology, 149(7), 1804-1812e4. https://doi.org/10.1053/j.gastro.2015.07.061

Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy. / Lammers, Willem J.; Hirschfield, Gideon M.; Corpechot, Christophe; Nevens, Frederik; Lindor, Keith D.; Janssen, Harry L A; Floreani, Annarosa; Ponsioen, Cyriel Y.; Mayo, Marlyn J.; Invernizzi, Pietro; Battezzati, Pier M.; Parés, Albert; Burroughs, Andrew K.; Mason, Andrew L.; Kowdley, Kris V.; Kumagi, Teru; Harms, Maren H.; Trivedi, Palak J.; Poupon, Raoul; Cheung, Angela; Lleo, Ana; Caballeria, Llorenç; Hansen, Bettina E.; Van Buuren, Henk R.

In: Gastroenterology, Vol. 149, No. 7, 01.12.2015, p. 1804-1812e4.

Research output: Contribution to journal › Article › peer-review

Lammers, WJ, Hirschfield, GM, Corpechot, C, Nevens, F, Lindor, KD, Janssen, HLA, Floreani, A, Ponsioen, CY, Mayo, MJ, Invernizzi, P, Battezzati, PM, Parés, A, Burroughs, AK, Mason, AL, Kowdley, KV, Kumagi, T, Harms, MH, Trivedi, PJ, Poupon, R, Cheung, A, Lleo, A, Caballeria, L, Hansen, BE & Van Buuren, HR 2015, 'Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy', Gastroenterology, vol. 149, no. 7, pp. 1804-1812e4. https://doi.org/10.1053/j.gastro.2015.07.061

Lammers, Willem J. ; Hirschfield, Gideon M. ; Corpechot, Christophe ; Nevens, Frederik ; Lindor, Keith D. ; Janssen, Harry L A ; Floreani, Annarosa ; Ponsioen, Cyriel Y. ; Mayo, Marlyn J. ; Invernizzi, Pietro ; Battezzati, Pier M. ; Parés, Albert ; Burroughs, Andrew K. ; Mason, Andrew L. ; Kowdley, Kris V. ; Kumagi, Teru ; Harms, Maren H. ; Trivedi, Palak J. ; Poupon, Raoul ; Cheung, Angela ; Lleo, Ana ; Caballeria, Llorenç ; Hansen, Bettina E. ; Van Buuren, Henk R. / Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy. In: Gastroenterology. 2015 ; Vol. 149, No. 7. pp. 1804-1812e4.

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abstract = "Background & Aims Approaches to risk stratification for patients with primary biliary cirrhosis (PBC) are limited, single-center based, and often dichotomous. We aimed to develop and validate a better model for determining prognoses of patients with PBC. Methods We performed an international, multicenter meta-analysis of 4119 patients with PBC treated with ursodeoxycholic acid at liver centers in 8 European and North American countries. Patients were randomly assigned to derivation (n = 2488 [60%]) and validation cohorts (n = 1631 [40%]). A risk score (GLOBE score) to predict transplantation-free survival was developed and validated with univariate and multivariable Cox regression analyses using clinical and biochemical variables obtained after 1 year of ursodeoxycholic acid therapy. Risk score outcomes were compared with the survival of age-, sex-, and calendar time-matched members of the general population. The prognostic ability of the GLOBE score was evaluated alongside those of the Barcelona, Paris-1, Rotterdam, Toronto, and Paris-2 criteria. Results Age (hazard ratio = 1.05; 95% confidence interval [CI]: 1.04-1.06; P 0.30 had significantly shorter times of transplant-free survival than matched healthy individuals (P ",

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AU - Hirschfield, Gideon M.

AU - Corpechot, Christophe

AU - Nevens, Frederik

AU - Lindor, Keith D.

AU - Janssen, Harry L A

AU - Floreani, Annarosa

AU - Ponsioen, Cyriel Y.

AU - Mayo, Marlyn J.

AU - Invernizzi, Pietro

AU - Battezzati, Pier M.

AU - Parés, Albert

AU - Burroughs, Andrew K.

AU - Mason, Andrew L.

AU - Kowdley, Kris V.

AU - Kumagi, Teru

AU - Harms, Maren H.

AU - Trivedi, Palak J.

AU - Poupon, Raoul

AU - Cheung, Angela

AU - Lleo, Ana

AU - Caballeria, Llorenç

AU - Hansen, Bettina E.

AU - Van Buuren, Henk R.

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AB - Background & Aims Approaches to risk stratification for patients with primary biliary cirrhosis (PBC) are limited, single-center based, and often dichotomous. We aimed to develop and validate a better model for determining prognoses of patients with PBC. Methods We performed an international, multicenter meta-analysis of 4119 patients with PBC treated with ursodeoxycholic acid at liver centers in 8 European and North American countries. Patients were randomly assigned to derivation (n = 2488 [60%]) and validation cohorts (n = 1631 [40%]). A risk score (GLOBE score) to predict transplantation-free survival was developed and validated with univariate and multivariable Cox regression analyses using clinical and biochemical variables obtained after 1 year of ursodeoxycholic acid therapy. Risk score outcomes were compared with the survival of age-, sex-, and calendar time-matched members of the general population. The prognostic ability of the GLOBE score was evaluated alongside those of the Barcelona, Paris-1, Rotterdam, Toronto, and Paris-2 criteria. Results Age (hazard ratio = 1.05; 95% confidence interval [CI]: 1.04-1.06; P 0.30 had significantly shorter times of transplant-free survival than matched healthy individuals (P

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Development and Validation of a Scoring System to Predict Outcomes of Patients with Primary Biliary Cirrhosis Receiving Ursodeoxycholic Acid Therapy

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