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Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups

We have previously derived power calculation formulas for cohort studies and clinical trials using the longitudinal mixed effects model with random slopes and intercepts to compare rate of change across groups [Ard & Edland, Power calculations for clinical trials in Alzheimer’s disease. J Alzhei...

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Autores principales: Zhao, Yu, Edland, Steven D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: De Gruyter 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9156336/
https://www.ncbi.nlm.nih.gov/pubmed/33581000
http://dx.doi.org/10.1515/ijb-2020-0107
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author Zhao, Yu
Edland, Steven D.
author_facet Zhao, Yu
Edland, Steven D.
author_sort Zhao, Yu
collection PubMed
description We have previously derived power calculation formulas for cohort studies and clinical trials using the longitudinal mixed effects model with random slopes and intercepts to compare rate of change across groups [Ard & Edland, Power calculations for clinical trials in Alzheimer’s disease. J Alzheim Dis 2011;21:369–77]. We here generalize these power formulas to accommodate 1) missing data due to study subject attrition common to longitudinal studies, 2) unequal sample size across groups, and 3) unequal variance parameters across groups. We demonstrate how these formulas can be used to power a future study even when the design of available pilot study data (i.e., number and interval between longitudinal observations) does not match the design of the planned future study. We demonstrate how differences in variance parameters across groups, typically overlooked in power calculations, can have a dramatic effect on statistical power. This is especially relevant to clinical trials, where changes over time in the treatment arm reflect background variability in progression observed in the placebo control arm plus variability in response to treatment, meaning that power calculations based only on the placebo arm covariance structure may be anticonservative. These more general power formulas are a useful resource for understanding the relative influence of these multiple factors on the efficiency of cohort studies and clinical trials, and for designing future trials under the random slopes and intercepts model.
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spelling pubmed-91563362022-06-21 Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups Zhao, Yu Edland, Steven D. Int J Biostat Article We have previously derived power calculation formulas for cohort studies and clinical trials using the longitudinal mixed effects model with random slopes and intercepts to compare rate of change across groups [Ard & Edland, Power calculations for clinical trials in Alzheimer’s disease. J Alzheim Dis 2011;21:369–77]. We here generalize these power formulas to accommodate 1) missing data due to study subject attrition common to longitudinal studies, 2) unequal sample size across groups, and 3) unequal variance parameters across groups. We demonstrate how these formulas can be used to power a future study even when the design of available pilot study data (i.e., number and interval between longitudinal observations) does not match the design of the planned future study. We demonstrate how differences in variance parameters across groups, typically overlooked in power calculations, can have a dramatic effect on statistical power. This is especially relevant to clinical trials, where changes over time in the treatment arm reflect background variability in progression observed in the placebo control arm plus variability in response to treatment, meaning that power calculations based only on the placebo arm covariance structure may be anticonservative. These more general power formulas are a useful resource for understanding the relative influence of these multiple factors on the efficiency of cohort studies and clinical trials, and for designing future trials under the random slopes and intercepts model. De Gruyter 2021-01-18 /pmc/articles/PMC9156336/ /pubmed/33581000 http://dx.doi.org/10.1515/ijb-2020-0107 Text en © 2020 Yu Zhao and Steven D. Edland, published by De Gruyter, Berlin/Boston https://creativecommons.org/licenses/by/4.0/This work is licensed under the Creative Commons Attribution 4.0 International License.
spellingShingle Article
Zhao, Yu
Edland, Steven D.
Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title_full Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title_fullStr Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title_full_unstemmed Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title_short Power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
title_sort power formulas for mixed effects models with random slope and intercept comparing rate of change across groups
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9156336/
https://www.ncbi.nlm.nih.gov/pubmed/33581000
http://dx.doi.org/10.1515/ijb-2020-0107
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