Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.

When the individual outcomes within a composite outcome appear to have different treatment effects, either in magnitude or direction, researchers may question the validity or appropriateness of using this composite outcome as a basis for measuring overall treatment effect in a randomized controlled...

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Autores principales:	Janice Pogue, P J Devereaux, Lehana Thabane, Salim Yusuf
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2012
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/6eba52c19fa64496ad554387d8c41211
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id	oai:doaj.org-article:6eba52c19fa64496ad554387d8c41211
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spelling	oai:doaj.org-article:6eba52c19fa64496ad554387d8c412112021-11-18T07:21:57ZDesigning and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.1932-620310.1371/journal.pone.0034785https://doaj.org/article/6eba52c19fa64496ad554387d8c412112012-01-01T00:00:00Zhttps://www.ncbi.nlm.nih.gov/pmc/articles/pmid/22529934/?tool=EBIhttps://doaj.org/toc/1932-6203When the individual outcomes within a composite outcome appear to have different treatment effects, either in magnitude or direction, researchers may question the validity or appropriateness of using this composite outcome as a basis for measuring overall treatment effect in a randomized controlled trial. The question remains as to how to distinguish random variation in estimated treatment effects from important heterogeneity within a composite outcome. This paper suggests there may be some utility in directly testing the assumption of homogeneity of treatment effect across the individual outcomes within a composite outcome. We describe a treatment heterogeneity test for composite outcomes based on a class of models used for the analysis of correlated data arising from the measurement of multiple outcomes for the same individuals. Such a test may be useful in planning a trial with a primary composite outcome and at trial end with final analysis and presentation. We demonstrate how to determine the statistical power to detect composite outcome treatment heterogeneity using the POISE Trial data. Then we describe how this test may be incorporated into a presentation of trial results with composite outcomes. We conclude that it may be informative for trialists to assess the consistency of treatment effects across the individual outcomes within a composite outcome using a formalized methodology and the suggested test represents one option.Janice PogueP J DevereauxLehana ThabaneSalim YusufPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 7, Iss 4, p e34785 (2012)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Janice Pogue P J Devereaux Lehana Thabane Salim Yusuf Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
description	When the individual outcomes within a composite outcome appear to have different treatment effects, either in magnitude or direction, researchers may question the validity or appropriateness of using this composite outcome as a basis for measuring overall treatment effect in a randomized controlled trial. The question remains as to how to distinguish random variation in estimated treatment effects from important heterogeneity within a composite outcome. This paper suggests there may be some utility in directly testing the assumption of homogeneity of treatment effect across the individual outcomes within a composite outcome. We describe a treatment heterogeneity test for composite outcomes based on a class of models used for the analysis of correlated data arising from the measurement of multiple outcomes for the same individuals. Such a test may be useful in planning a trial with a primary composite outcome and at trial end with final analysis and presentation. We demonstrate how to determine the statistical power to detect composite outcome treatment heterogeneity using the POISE Trial data. Then we describe how this test may be incorporated into a presentation of trial results with composite outcomes. We conclude that it may be informative for trialists to assess the consistency of treatment effects across the individual outcomes within a composite outcome using a formalized methodology and the suggested test represents one option.
format	article
author	Janice Pogue P J Devereaux Lehana Thabane Salim Yusuf
author_facet	Janice Pogue P J Devereaux Lehana Thabane Salim Yusuf
author_sort	Janice Pogue
title	Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
title_short	Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
title_full	Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
title_fullStr	Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
title_full_unstemmed	Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
title_sort	designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.
publisher	Public Library of Science (PLoS)
publishDate	2012
url	https://doaj.org/article/6eba52c19fa64496ad554387d8c41211
work_keys_str_mv	AT janicepogue designingandanalyzingclinicaltrialswithcompositeoutcomesconsiderationofpossibletreatmentdifferencesbetweentheindividualoutcomes AT pjdevereaux designingandanalyzingclinicaltrialswithcompositeoutcomesconsiderationofpossibletreatmentdifferencesbetweentheindividualoutcomes AT lehanathabane designingandanalyzingclinicaltrialswithcompositeoutcomesconsiderationofpossibletreatmentdifferencesbetweentheindividualoutcomes AT salimyusuf designingandanalyzingclinicaltrialswithcompositeoutcomesconsiderationofpossibletreatmentdifferencesbetweentheindividualoutcomes
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Designing and analyzing clinical trials with composite outcomes: consideration of possible treatment differences between the individual outcomes.

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