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Research Article

Using the potential outcome framework to estimate optimal sample size for cluster randomized trials: a simulation-based algorithm

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Pages 3744-3770 | Received 06 Jul 2020, Accepted 19 Jun 2021, Published online: 15 Jul 2021

References

  • Moberg J, Kramer M. A brief history of the cluster randomised trial design. J R Soc Med. 2015;108(5):192–198.
  • Donner A, Klar N. Design and analysis of cluster randomization trials in health research. London: Arnold; 2000.
  • Donner A, Klar N. Pitfalls of and controversies in cluster randomization trials. Am J Public Health. 2004;94(3):416–422.
  • Rotondi MA, Donner A. Sample size estimation in cluster randomized educational trials: an empirical Bayes approach. J Educ Behav Stat. 2009;34(2):229–237.
  • Hayes RJ, Bennett S. Simple sample size calculation for cluster-randomized trials. Int J Epidemiol. 1999;28(2):319–326.
  • Rotondi MA. CRTSize: sample size estimation functions for cluster randomized trials; 2015. R package version 1.0. Available from: https://CRAN.R-project.org/package=CRTSize.
  • Rutterford C, Copas A, Eldridge S. Methods for sample size determination in cluster randomized trials. Int J Epidemiol. 2015;44(3):1051–1067.
  • Browne WJ, Lahi MG, Parker RMA. A guide to sample size calculations for random effect models via simulation and the MLPowSim software package. Bristol (UK): University of Bristol; 2009.
  • Hooper R. Versatile sample-size calculation using simulation. Stata J. 2013;13(1):21–38.
  • Kleinman K, Huang SS. Calculating power by bootstrap, with an application to cluster-randomized trials. eGEMs. 2016;4(1):1202.
  • Lohr SL. Sampling: design and analysis: design and analysis. Boca Raton: Chapman and Hall/CRC; 2019.
  • Rosenberger WF, Lachin JM. Randomization in clinical trials: theory and practice. New York: John Wiley & Sons; 2015.
  • Cook AJ, Delong E, Murray DM, et al. Statistical lessons learned for designing cluster randomized pragmatic clinical trials from the NIH health care systems collaboratory biostatistics and design core. Clinical Trials. 2016;13(5):504–512.
  • Imbens GW, Rubin DB. Causal inference in statistics, social, and biomedical sciences. New York: Cambridge University Press; 2015.
  • Rubin DB. Comment. J Am Stat Assoc. 1980;75(371):591–593.
  • Rubin DB. 2 statistical inference for causal effects, with emphasis on applications in epidemiology and medical statistics. Handbook Stat. 2007;27:28–63.
  • Rubin DB. Bayesian inference for causal effects: the role of randomization. Ann Stat. 1978;6:34–58.
  • Donner A, Birkett N, Buck C. Randomization by cluster: sample size requirements and analysis. Am J Epidemiol. 1981;114(6):906–914.
  • van Breukelen GJP, Candel MJJM. How to design and analyse cluster randomized trials with a small number of clusters? Comment on Leyrat et al. Int J Epidemiol. 2018;47(3):998–1001.
  • Lauer SA, Kleinman KP, Reich NG. The effect of cluster size variability on statistical power in cluster-randomized trials. PLoS ONE. 2015;10(4):e0119074.
  • Manatunga AK, Hudgens MG, Chen S. Sample size estimation in cluster randomized studies with varying cluster size. Biom J. 2001;43(1):75–86.
  • van Breukelen GJP, Candel MJJM, Berger MPF. Relative efficiency of unequal versus equal cluster sizes in cluster randomized and multicentre trials. Stat Med. 2007;26(13):2589–2603.
  • Ridout MS, Demetrio CGB, Firth D. Estimating intraclass correlation for binary data. Biometrics. 1999;55(1):137–148.
  • Wu S, Crespi CM, Weng KW. Comparison of methods for estimating the intraclass correlation coefficient for binary responses in cancer prevention cluster randomized trials. Contemp Clin Trials. 2012;33(5):869–880.
  • StataCorp. Stata statistical software: release 16. College Station (TX): StataCorp LLC; 2019.
  • Gail MH, Mark SD, Carroll RJ, et al. On design considerations and randomization-based inference for community intervention trials. Stat Med. 1996;15(11):1069–1092.
  • Small DS, Ten Have TR, Rosenbaum PR. Randomization inference in a group–randomized trial of treatments for depression: covariate adjustment, noncompliance, and quantile effects. J Am Stat Assoc. 2008;103(481):271–279.
  • Wang R, De Gruttola V. The use of permutation tests for the analysis of parallel and stepped-wedge cluster-randomized trials. Stat Med. 2017;36(18):2831–2843.
  • Kempthorne O, Hinkelmann K. Design and analysis of experiments. Vol. 1: Introduction to Experimental Design. Hoboken, NJ: Wiley; 2007.
  • Rosenbaum PR. Observational studies. New York: Springer; 2010.
  • Ding P. et al. A paradox from randomization-based causal inference. Stat Sci. 2017;32(3):331–345.
  • Flores I, Madpis G. Average binary search length for dense ordered lists. Commun ACM. 1971;14(9):602–603.
  • Steiner C, Elixhauser A, Schnaier J. The healthcare cost and utilization project: an overview. Eff Clin Pract. 2002;5(3):143–151.
  • Cameron AC, Trivedi PK. Regression analysis of count data. Vol. 53. Cambridge, United Kingdom: Cambridge University Press; 2013.
  • Zuur AF, Ieno EN, Walker NJ, et al. Zero-truncated and zero-inflated models for count data. Mixed effects models and extensions in ecology with R. New York, NY: Springer; 2009. p. 261–291.
  • Gravenstein S, Davidson HE, Taljaard M, et al. Comparative effectiveness of high-dose versus standard-dose influenza vaccination on numbers of us nursing home residents admitted to hospital: a cluster-randomised trial. Lancet Respir Med. 2017;5(9):738–746.
  • Schulz AJ, Ed Mullings L. Gender, race, class, & health: intersectional approaches. San Francisco, CA: Jossey-Bass; 2006.
  • Kernan WN, Viscoli CM, Makuch RW, et al. Stratified randomization for clinical trials. J Clin Epidemiol. 1999;52(1):19–26.
  • Raudenbush SW. Statistical analysis and optimal design for cluster randomized trials. Psychol Meth. 1997;2(2):173–185.

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