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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...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BMJ Publishing Group Ltd.
2018
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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. |
format | Online Article Text |
id | pubmed-5751848 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | BMJ Publishing Group Ltd. |
record_format | MEDLINE/PubMed |
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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