Power and sample size calculations for cluster randomized trials with binary outcomes when intracluster correlation coefficients vary by treatment arm

1. Hayes, RJ, Moulton, LH. Cluster randomised trials. 2nd ed. Boca Raton, FL: CRC Press, 2017.
Google Scholar2. Campbell, MK, Fayers, PM, Grimshaw, JM. Determinants of the intracluster correlation coefficient in cluster randomized trials: the case of implementation research. Clin Trials 2005; 2(2): 99–107.
Google Scholar | SAGE Journals | ISI3. Choudhry, NK, Avorn, J, Glynn, RJ, et al. Full coverage for preventive medications after myocardial infarction. N Engl J Med 2011; 365(22): 2088–2097.
Google Scholar | Crossref | Medline | ISI4. Benger, JR, Kirby, K, Black, S, et al. Effect of a strategy of a supraglottic airway device vs tracheal intubation during out-of-hospital cardiac arrest on functional outcome: the AIRWAYS-2 randomized clinical trial. JAMA 2018; 320(8): 779–791.
Google Scholar | Crossref | Medline5. Shih, WJ . Sample size and power calculations for periodontal and other studies with clustered samples using the method of generalized estimating equations. Biom J 1997; 39: 899–908.
Google Scholar | Crossref | ISI6. Wang, R, Goyal, R, Lei, Q, et al. Sample size considerations in the design of cluster randomized trials of combination HIV prevention. Clin Trials 2014; 11(3): 309–318.
Google Scholar | SAGE Journals | ISI7. Theiss-Nyland, K, Qadri, F, Colin-Jones, R, et al. Assessing the impact of a Vi-polysaccharide conjugate vaccine in preventing typhoid infection among Bangladeshi children: a protocol for a phase IIIb trial. Clin Inf Dis 2019; 68(Supp. 2): S74–S82.
Google Scholar | Crossref | Medline8. Gupta, A, Swindells, S, Kim, S, et al. Feasibility of identifying household contacts of rifampin- and multidrug-resistant tuberculosis cases at high risk of progression to tuberculosis disease. Clin Inf Dis 2020; 70: 425–435.
Google Scholar | Crossref | Medline9. Mitjà, O, Corbacho-Monné, M, Ubals, M, et al. A cluster-randomized trial of hydroxychloroquine for prevention of COVID-19. N Engl J Med 2021; 384(5): 417–427.
Google Scholar | Crossref | Medline10. Donner, A, Klar, N. Design and analysis of cluster randomization trials in health research. London: John Wiley & Sons, 2000.
Google Scholar11. Liang, KY, Zeger, SL. Longitudinal data analysis using generalized linear models. Biometrika 1986; 73(1): 13–22.
Google Scholar | Crossref | ISI12. Fitzmaurice, GM . A caveat concerning independence estimating equations with multivariate binary data. Biometrics 1995; 51(1): 309–317.
Google Scholar | Crossref | Medline13. Spiess, M, Hamerle, A. On the properties of GEE estimators in the presence of invariant covariates. Biom J 1996; 38(8): 931–940.
Google Scholar | Crossref14. Ziegler, A, Vens, M. Generalized estimating equations: notes on the choice of the working correlation matrix. Methods Inf Med 2010; 49(5): 421–425.
Google Scholar | Crossref | Medline15. Pan, W . Sample size and power calculations with correlated binary data. Control Clin Trials 2001; 22(3): 211–227.
Google Scholar | Crossref | Medline16. Gulliford, MC, Adams, G, Ukoumunne, OC, et al. Intraclass correlation coefficient and outcome prevalence are associated in clustered binary data. J Clin Epidemiol 2005; 58(3): 246–251.
Google Scholar | Crossref | Medline | ISI17. Lin, A, Ercumen, A, Benjamin-Chung, J, et al. Effects of water, sanitation, handwashing, and nutritional interventions on child enteric protozoan infections in rural Bangladesh: a cluster-randomized controlled trial. Clin Inf Dis 2018; 67(10): 1515–1522.
Google Scholar | Medline18. Rutterford, C, Copas, A, Eldridge, S. Methods for sample size determination in cluster randomized trials. Int J Epidemiol 2015; 44(3): 1051–1067.
Google Scholar | Crossref | Medline19. Zeger, SL, Liang, KY. Longitudinal data analysis for discrete and continuous outcomes. Biometrics 1986; 42(1): 121–130.
Google Scholar | Crossref | Medline | ISI20. Kennedy-Shaffer, L, Hughes, MD. Sample size estimation for stratified individual and cluster randomized trials with binary outcomes. Stat Med 2020; 39(10): 1489–1513.
Google Scholar | Crossref | Medline21. Yelland, LN, Salter, AB, Ryan, P, et al. Adjusted intraclass correlation coefficients for binary data: methods and estimates from a cluster-randomized trial in primary care. Clin Trials 2011; 8(1): 48–58.
Google Scholar | SAGE Journals | ISI22. Crowder, M . On the use of a working correlation matrix in using generalised linear models for repeated measures. Biometrika 1995; 82(2): 407–410.
Google Scholar | Crossref23. Eldridge, SM, Ukoumunne, OC, Carlin, JB. The intra-cluster correlation coefficient in cluster randomized trials: a review of definitions. Int Stat Rev 2009; 77(3): 378–394.
Google Scholar | Crossref | ISI24. Demidenko, E . Sample size determination for logistic regression revisited. Stat Med 2007; 26: 3385–3397.
Google Scholar | Crossref | Medline | ISI25. Consul, PC, Jain, GC. A generalization of the Poisson distribution. Technometrics 1973; 15(4): 791–799.
Google Scholar | Crossref26. Scollnik, DPM . On the analysis of the truncated generalized Poisson distribution using a Bayesian method. ASTIN Bull 1998; 28(1): 135–152.
Google Scholar | Crossref27. Horton, NJ, Lipsitz, SR. Review of software to fit generalized estimating equation regression models. Am Stat 1999; 53(2): 160–169.
Google Scholar28. Halekoh, U, Højsgaard, S, Yan, J. The R package geepack for generalized estimating equations. J Stat Softw 2006; 15: 1.
Google Scholar | Crossref | ISI29. Campbell, MK, Grimshaw, JM, Elbourne, DR. Intracluster correlation coefficients in cluster randomized trials: empirical insights into how should they be reported. BMC Med Res Methodol 2004; 4: 9.
Google Scholar | Crossref | Medline30. Eldridge, SM, Costelloe, CE, Kahan, BC, et al. How big should the pilot study for my cluster randomised trial be?. Stat Methods Med Res 2016; 25(3): 1039–1056.
Google Scholar | SAGE Journals | ISI31. Mancl, LA, DeRouen, TA. A covariance estimator for GEE with improved small-sample properties. Biometrics 2001; 57(1): 126–134.
Google Scholar | Crossref | Medline | ISI32. Huang, S, Fiero, MH, Bell, ML. Generalized estimating equations in cluster randomized trials with a small number of clusters: review of practice and simulation study. Clin Trials 2016; 13(4): 445–449.
Google Scholar | SAGE Journals | ISI

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