A novel prognostic model for transplant-free survival in primary sclerosing cholangitis

<strong>Objective</strong> Most prognostic models for primary sclerosing cholangitis (PSC) are based on patients referred to tertiary care and may not be applicable for the majority of patients with PSC. The aim of this study was to construct and externally validate a novel, broadly appl...

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Main Authors: de Vries, E, Wang, J, Williamson, K, Leeflang, M, Boonstra, K, Weersma, R, Beuers, U, Chapman, R, Geskus, R, Ponsioen, C
Format: Journal article
Published: BMJ Publishing Group 2017
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author de Vries, E
Wang, J
Williamson, K
Leeflang, M
Boonstra, K
Weersma, R
Beuers, U
Chapman, R
Geskus, R
Ponsioen, C
author_facet de Vries, E
Wang, J
Williamson, K
Leeflang, M
Boonstra, K
Weersma, R
Beuers, U
Chapman, R
Geskus, R
Ponsioen, C
author_sort de Vries, E
collection OXFORD
description <strong>Objective</strong> Most prognostic models for primary sclerosing cholangitis (PSC) are based on patients referred to tertiary care and may not be applicable for the majority of patients with PSC. The aim of this study was to construct and externally validate a novel, broadly applicable prognostic model for transplant-free survival in PSC, based on a large, predominantly population-based cohort using readily available variables. <strong>Design</strong> The derivation cohort consisted of 692 patients with PSC from the Netherlands, the validation cohort of 264 patients with PSC from the UK. Retrospectively, clinical and biochemical variables were collected. We derived the prognostic index from a multivariable Cox regression model in which predictors were selected and parameters were estimated using the least absolute shrinkage and selection operator. The composite end point of PSC-related death and liver transplantation was used. To quantify the models’ predictive value, we calculated the C-statistic as discrimination index and established its calibration accuracy by comparing predicted curves with Kaplan-Meier estimates. <strong>Results</strong> The final model included the variables: PSC subtype, age at PSC diagnosis, albumin, platelets, aspartate aminotransferase, alkaline phosphatase and bilirubin. The C-statistic was 0.68 (95% CI 0.51 to 0.85). Calibration was satisfactory. The model was robust in the sense that the C-statistic did not change when prediction was based on biochemical variables collected at follow-up. <strong>Conclusion</strong> The Amsterdam-Oxford model for PSC showed adequate performance in estimating PSC-related death and/or liver transplant in a predominantly population-based setting. The transplant-free survival probability can be recalculated when updated biochemical values are available.
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spelling oxford-uuid:bdfaae4a-d268-44f1-af33-052b7fcb66e32022-03-27T05:35:54ZA novel prognostic model for transplant-free survival in primary sclerosing cholangitisJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:bdfaae4a-d268-44f1-af33-052b7fcb66e3Symplectic Elements at OxfordBMJ Publishing Group2017de Vries, EWang, JWilliamson, KLeeflang, MBoonstra, KWeersma, RBeuers, UChapman, RGeskus, RPonsioen, C<strong>Objective</strong> Most prognostic models for primary sclerosing cholangitis (PSC) are based on patients referred to tertiary care and may not be applicable for the majority of patients with PSC. The aim of this study was to construct and externally validate a novel, broadly applicable prognostic model for transplant-free survival in PSC, based on a large, predominantly population-based cohort using readily available variables. <strong>Design</strong> The derivation cohort consisted of 692 patients with PSC from the Netherlands, the validation cohort of 264 patients with PSC from the UK. Retrospectively, clinical and biochemical variables were collected. We derived the prognostic index from a multivariable Cox regression model in which predictors were selected and parameters were estimated using the least absolute shrinkage and selection operator. The composite end point of PSC-related death and liver transplantation was used. To quantify the models’ predictive value, we calculated the C-statistic as discrimination index and established its calibration accuracy by comparing predicted curves with Kaplan-Meier estimates. <strong>Results</strong> The final model included the variables: PSC subtype, age at PSC diagnosis, albumin, platelets, aspartate aminotransferase, alkaline phosphatase and bilirubin. The C-statistic was 0.68 (95% CI 0.51 to 0.85). Calibration was satisfactory. The model was robust in the sense that the C-statistic did not change when prediction was based on biochemical variables collected at follow-up. <strong>Conclusion</strong> The Amsterdam-Oxford model for PSC showed adequate performance in estimating PSC-related death and/or liver transplant in a predominantly population-based setting. The transplant-free survival probability can be recalculated when updated biochemical values are available.
spellingShingle de Vries, E
Wang, J
Williamson, K
Leeflang, M
Boonstra, K
Weersma, R
Beuers, U
Chapman, R
Geskus, R
Ponsioen, C
A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title_full A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title_fullStr A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title_full_unstemmed A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title_short A novel prognostic model for transplant-free survival in primary sclerosing cholangitis
title_sort novel prognostic model for transplant free survival in primary sclerosing cholangitis
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