Volume 33, Issue 22
Research Article

Sample size determination for longitudinal designs with binary response

Kush Kapur

Corresponding Author

Clinical Research Center and Department of Neurology, Boston Children's Hospital, Harvard Medical School, 21 Autumn St., Boston, MA 02215, U.S.A.

Correspondence to: Kush Kapur, Clinical Research Center and Department of Neurology, Boston Children's Hospital, Harvard Medical School, 21 Autumn St., Boston, MA 02215, U.S.A.

E‐mail: kush.kapur@childrens.harvard.edu

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Runa Bhaumik

Department of Psychiatry and Division of Epidemiology and Biostatistics, University of Illinois at Chicago, 1601 W. Taylor St., Chicago, IL 60612, U.S.A.

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X. Charlene Tang

Cooperative Studies Program Coordinating Center, Hines VA Hospital, 5000 South 5th Avenue, Building 1, Hines, IL 60141, U.S.A.

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Kwan Hur

Center for Medication Safety, Pharmacy Benefit Management Services, Hines, IL 60141, U.S.A.

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Domenic J. Reda

Cooperative Studies Program Coordinating Center, Hines VA Hospital, 5000 South 5th Avenue, Building 1, Hines, IL 60141, U.S.A.

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Dulal K. Bhaumik

Department of Psychiatry and Division of Epidemiology and Biostatistics, University of Illinois at Chicago, 1601 W. Taylor St., Chicago, IL 60612, U.S.A.

Cooperative Studies Program Coordinating Center, Hines VA Hospital, 5000 South 5th Avenue, Building 1, Hines, IL 60141, U.S.A.

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First published: 12 May 2014
Citations: 4

Abstract

In this article, we develop appropriate statistical methods for determining the required sample size while comparing the efficacy of an intervention to a control with repeated binary response outcomes. Our proposed methodology incorporates the complexity of the hierarchical nature of underlying designs and provides solutions when varying attrition rates are present over time. We explore how the between‐subject variability and attrition rates jointly influence the computation of sample size formula. Our procedure also shows how efficient estimation methods play a crucial role in power analysis. A practical guideline is provided when information regarding individual variance component is unavailable. The validity of our methods is established by extensive simulation studies. Results are illustrated with the help of two randomized clinical trials in the areas of contraception and insomnia. Copyright © 2014 John Wiley & Sons, Ltd.

Number of times cited according to CrossRef: 4

  • Sample size determination for multilevel hierarchical designs using generalized linear mixed models, Biometrics, 10.1111/biom.12764, 74, 2, (673-684), (2017).
  • Power and sample size for the S:T repeated measures design combined with a linear mixed-effects model allowing for missing data , Journal of Biopharmaceutical Statistics, 10.1080/10543406.2017.1293083, 27, 6, (963-974), (2017).
  • Sample size estimation for a two-group comparison of repeated count outcomes using GEE, Communications in Statistics - Theory and Methods, 10.1080/03610926.2015.1134572, 46, 14, (6743-6753), (2016).
  • On the repeated measures designs and sample sizes for randomized controlled trials, Biostatistics, 10.1093/biostatistics/kxv047, 17, 2, (334-349), (2015).

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