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Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data
BACKGROUND: The primary analysis in a longitudinal randomized controlled trial is sometimes a comparison of arms at a single time point. While a two-sample t-test is often used, missing data are common in longitudinal studies and decreases power by reducing sample size. Mixed models for repeated mea...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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BioMed Central
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4828848/ https://www.ncbi.nlm.nih.gov/pubmed/27068578 http://dx.doi.org/10.1186/s12874-016-0144-0 |
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author | Ashbeck, Erin L. Bell, Melanie L. |
author_facet | Ashbeck, Erin L. Bell, Melanie L. |
author_sort | Ashbeck, Erin L. |
collection | PubMed |
description | BACKGROUND: The primary analysis in a longitudinal randomized controlled trial is sometimes a comparison of arms at a single time point. While a two-sample t-test is often used, missing data are common in longitudinal studies and decreases power by reducing sample size. Mixed models for repeated measures (MMRM) can test treatment effects at specific time points, have been shown to give unbiased estimates in certain missing data contexts, and may be more powerful than a two sample t-test. METHODS: We conducted a simulation study to compare the performance of a complete-case t-test to a MMRM in terms of power and bias under different missing data mechanisms. Impact of within- and between-person variance, dropout mechanism, and variance-covariance structure were all considered. RESULTS: While both complete-case t-test and MMRM provided unbiased estimation of treatment differences when data were missing completely at random, MMRM yielded an absolute power gain of up to 12 %. The MMRM provided up to 25 % absolute increased power over the t-test when data were missing at random, as well as unbiased estimation. CONCLUSIONS: Investigators interested in single time point comparisons should use a MMRM with a contrast to gain power and unbiased estimation of treatment effects instead of a complete-case two sample t-test. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s12874-016-0144-0) contains supplementary material, which is available to authorized users. |
format | Online Article Text |
id | pubmed-4828848 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2016 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-48288482016-04-13 Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data Ashbeck, Erin L. Bell, Melanie L. BMC Med Res Methodol Research Article BACKGROUND: The primary analysis in a longitudinal randomized controlled trial is sometimes a comparison of arms at a single time point. While a two-sample t-test is often used, missing data are common in longitudinal studies and decreases power by reducing sample size. Mixed models for repeated measures (MMRM) can test treatment effects at specific time points, have been shown to give unbiased estimates in certain missing data contexts, and may be more powerful than a two sample t-test. METHODS: We conducted a simulation study to compare the performance of a complete-case t-test to a MMRM in terms of power and bias under different missing data mechanisms. Impact of within- and between-person variance, dropout mechanism, and variance-covariance structure were all considered. RESULTS: While both complete-case t-test and MMRM provided unbiased estimation of treatment differences when data were missing completely at random, MMRM yielded an absolute power gain of up to 12 %. The MMRM provided up to 25 % absolute increased power over the t-test when data were missing at random, as well as unbiased estimation. CONCLUSIONS: Investigators interested in single time point comparisons should use a MMRM with a contrast to gain power and unbiased estimation of treatment effects instead of a complete-case two sample t-test. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s12874-016-0144-0) contains supplementary material, which is available to authorized users. BioMed Central 2016-04-12 /pmc/articles/PMC4828848/ /pubmed/27068578 http://dx.doi.org/10.1186/s12874-016-0144-0 Text en © Ashbeck and Bell. 2016 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. 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. |
spellingShingle | Research Article Ashbeck, Erin L. Bell, Melanie L. Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title | Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title_full | Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title_fullStr | Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title_full_unstemmed | Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title_short | Single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
title_sort | single time point comparisons in longitudinal randomized controlled trials: power and bias in the presence of missing data |
topic | Research Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4828848/ https://www.ncbi.nlm.nih.gov/pubmed/27068578 http://dx.doi.org/10.1186/s12874-016-0144-0 |
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