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Inference about time-dependent prognostic accuracy measures in the presence of competing risks

BACKGROUND: Evaluating a candidate marker or developing a model for predicting risk of future conditions is one of the major goals in medicine. However, model development and assessment for a time-to-event outcome may be complicated in the presence of competing risks. In this manuscript, we propose...

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Autores principales: Dey, Rajib, Sebastiani, Giada, Saha-Chaudhuri, Paramita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7456384/
https://www.ncbi.nlm.nih.gov/pubmed/32859153
http://dx.doi.org/10.1186/s12874-020-01100-0
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author Dey, Rajib
Sebastiani, Giada
Saha-Chaudhuri, Paramita
author_facet Dey, Rajib
Sebastiani, Giada
Saha-Chaudhuri, Paramita
author_sort Dey, Rajib
collection PubMed
description BACKGROUND: Evaluating a candidate marker or developing a model for predicting risk of future conditions is one of the major goals in medicine. However, model development and assessment for a time-to-event outcome may be complicated in the presence of competing risks. In this manuscript, we propose a local and a global estimators of cause-specific AUC for right-censored survival times in the presence of competing risks. METHODS: The local estimator - cause-specific weighted mean rank (cWMR) - is a local average of time-specific observed cause-specific AUCs within a neighborhood of given time t. The global estimator - cause-specific fractional polynomials (cFPL) - is based on modelling the cause-specific AUC as a function of t through fractional polynomials. RESULTS: We investigated the performance of the proposed cWMR and cFPL estimators through simulation studies and real-life data analysis. The estimators perform well in small samples, have minimal bias and appropriate coverage. CONCLUSIONS: The local estimator cWMR and the global estimator cFPL will provide computationally efficient options for assessing the prognostic accuracy of markers for time-to-event outcome in the presence of competing risks in many practical settings.
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spelling pubmed-74563842020-08-31 Inference about time-dependent prognostic accuracy measures in the presence of competing risks Dey, Rajib Sebastiani, Giada Saha-Chaudhuri, Paramita BMC Med Res Methodol Technical Advance BACKGROUND: Evaluating a candidate marker or developing a model for predicting risk of future conditions is one of the major goals in medicine. However, model development and assessment for a time-to-event outcome may be complicated in the presence of competing risks. In this manuscript, we propose a local and a global estimators of cause-specific AUC for right-censored survival times in the presence of competing risks. METHODS: The local estimator - cause-specific weighted mean rank (cWMR) - is a local average of time-specific observed cause-specific AUCs within a neighborhood of given time t. The global estimator - cause-specific fractional polynomials (cFPL) - is based on modelling the cause-specific AUC as a function of t through fractional polynomials. RESULTS: We investigated the performance of the proposed cWMR and cFPL estimators through simulation studies and real-life data analysis. The estimators perform well in small samples, have minimal bias and appropriate coverage. CONCLUSIONS: The local estimator cWMR and the global estimator cFPL will provide computationally efficient options for assessing the prognostic accuracy of markers for time-to-event outcome in the presence of competing risks in many practical settings. BioMed Central 2020-08-28 /pmc/articles/PMC7456384/ /pubmed/32859153 http://dx.doi.org/10.1186/s12874-020-01100-0 Text en © The Author(s) 2020 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Technical Advance
Dey, Rajib
Sebastiani, Giada
Saha-Chaudhuri, Paramita
Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title_full Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title_fullStr Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title_full_unstemmed Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title_short Inference about time-dependent prognostic accuracy measures in the presence of competing risks
title_sort inference about time-dependent prognostic accuracy measures in the presence of competing risks
topic Technical Advance
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7456384/
https://www.ncbi.nlm.nih.gov/pubmed/32859153
http://dx.doi.org/10.1186/s12874-020-01100-0
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