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Statistical methods to compare functional outcomes in randomized controlled trials with high mortality

Mortality is a common primary endpoint in randomized controlled trials of patients with a high severity of illness, such as critically ill patients. However, researchers are increasingly evaluating functional outcomes, such as quality of life. Importantly, in such trials some patients may die before...

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Autores principales: Colantuoni, Elizabeth, Scharfstein, Daniel O, Wang, Chenguang, Hashem, Mohamed D, Leroux, Andrew, Needham, Dale M, Girard, Timothy D
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group Ltd. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5751848/
https://www.ncbi.nlm.nih.gov/pubmed/29298779
http://dx.doi.org/10.1136/bmj.j5748
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author Colantuoni, Elizabeth
Scharfstein, Daniel O
Wang, Chenguang
Hashem, Mohamed D
Leroux, Andrew
Needham, Dale M
Girard, Timothy D
author_facet Colantuoni, Elizabeth
Scharfstein, Daniel O
Wang, Chenguang
Hashem, Mohamed D
Leroux, Andrew
Needham, Dale M
Girard, Timothy D
author_sort Colantuoni, Elizabeth
collection PubMed
description Mortality is a common primary endpoint in randomized controlled trials of patients with a high severity of illness, such as critically ill patients. However, researchers are increasingly evaluating functional outcomes, such as quality of life. Importantly, in such trials some patients may die before the assessment of a functional outcome, resulting in the functional outcome being “truncated due to death.” As described in this paper, defining and testing treatment effects on functional outcomes in this setting requires careful consideration. Data from a completed trial of critically ill patients are used to highlight key differences among three statistical approaches used when analyzing such trials.
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spelling pubmed-57518482018-02-12 Statistical methods to compare functional outcomes in randomized controlled trials with high mortality Colantuoni, Elizabeth Scharfstein, Daniel O Wang, Chenguang Hashem, Mohamed D Leroux, Andrew Needham, Dale M Girard, Timothy D BMJ Research Methods & Reporting Mortality is a common primary endpoint in randomized controlled trials of patients with a high severity of illness, such as critically ill patients. However, researchers are increasingly evaluating functional outcomes, such as quality of life. Importantly, in such trials some patients may die before the assessment of a functional outcome, resulting in the functional outcome being “truncated due to death.” As described in this paper, defining and testing treatment effects on functional outcomes in this setting requires careful consideration. Data from a completed trial of critically ill patients are used to highlight key differences among three statistical approaches used when analyzing such trials. BMJ Publishing Group Ltd. 2018-01-03 /pmc/articles/PMC5751848/ /pubmed/29298779 http://dx.doi.org/10.1136/bmj.j5748 Text en Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.
spellingShingle Research Methods & Reporting
Colantuoni, Elizabeth
Scharfstein, Daniel O
Wang, Chenguang
Hashem, Mohamed D
Leroux, Andrew
Needham, Dale M
Girard, Timothy D
Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title_full Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title_fullStr Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title_full_unstemmed Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title_short Statistical methods to compare functional outcomes in randomized controlled trials with high mortality
title_sort statistical methods to compare functional outcomes in randomized controlled trials with high mortality
topic Research Methods & Reporting
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5751848/
https://www.ncbi.nlm.nih.gov/pubmed/29298779
http://dx.doi.org/10.1136/bmj.j5748
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