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Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials

Competing risks data are commonly encountered in randomized clinical trials or observational studies. Ignoring competing risks in survival analysis leads to biased risk estimates and improper conclusions. Often, one of the competing events is of primary interest and the rest competing events are han...

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Autores principales: Wen, Jiyang, Wang, Mei-Cheng, Hu, Chen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10315219/
https://www.ncbi.nlm.nih.gov/pubmed/37035880
http://dx.doi.org/10.1002/sim.9728
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author Wen, Jiyang
Wang, Mei-Cheng
Hu, Chen
author_facet Wen, Jiyang
Wang, Mei-Cheng
Hu, Chen
author_sort Wen, Jiyang
collection PubMed
description Competing risks data are commonly encountered in randomized clinical trials or observational studies. Ignoring competing risks in survival analysis leads to biased risk estimates and improper conclusions. Often, one of the competing events is of primary interest and the rest competing events are handled as nuisances. These approaches can be inadequate when multiple competing events have important clinical interpretations and thus of equal interest. For example, in COVID-19 in-patient treatment trials, the outcomes of COVID-19 related hospitalization are either death or discharge from hospital, which have completely different clinical implications and are of equal interest, especially during the pandemic. In this paper we develop nonparametric estimation and simultaneous inferential methods for multiple cumulative incidence functions (CIFs) and corresponding restricted mean times. Based on Monte Carlo simulations and a data analysis of COVID-19 in-patient treatment clinical trial, we demonstrate that the proposed method provides global insights of the treatment effects across multiple endpoints.
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spelling pubmed-103152192023-07-02 Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials Wen, Jiyang Wang, Mei-Cheng Hu, Chen Stat Med Article Competing risks data are commonly encountered in randomized clinical trials or observational studies. Ignoring competing risks in survival analysis leads to biased risk estimates and improper conclusions. Often, one of the competing events is of primary interest and the rest competing events are handled as nuisances. These approaches can be inadequate when multiple competing events have important clinical interpretations and thus of equal interest. For example, in COVID-19 in-patient treatment trials, the outcomes of COVID-19 related hospitalization are either death or discharge from hospital, which have completely different clinical implications and are of equal interest, especially during the pandemic. In this paper we develop nonparametric estimation and simultaneous inferential methods for multiple cumulative incidence functions (CIFs) and corresponding restricted mean times. Based on Monte Carlo simulations and a data analysis of COVID-19 in-patient treatment clinical trial, we demonstrate that the proposed method provides global insights of the treatment effects across multiple endpoints. 2023-06-30 2023-04-10 /pmc/articles/PMC10315219/ /pubmed/37035880 http://dx.doi.org/10.1002/sim.9728 Text en https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.
spellingShingle Article
Wen, Jiyang
Wang, Mei-Cheng
Hu, Chen
Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title_full Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title_fullStr Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title_full_unstemmed Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title_short Simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
title_sort simultaneous hypothesis testing for multiple competing risks in comparative clinical trials
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10315219/
https://www.ncbi.nlm.nih.gov/pubmed/37035880
http://dx.doi.org/10.1002/sim.9728
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