Estimation and sample size considerations for clustered binary responses.

Although there is much literature on sample size determination for clinical trials or experiments with independent responses, there is a lack of methodology to obtain sample sizes for dependent outcomes. This paper presents a simple way to calculate sample size for estimating treatment effects and diagnostic accuracy in the case of correlated binary outcomes. The proposed weighted procedure also has use in estimation, whose advantages we demonstrate through simulation. Recommendations are made for practical application.

[1]  Robert Tibshirani,et al.  Bootstrap Methods for Standard Errors, Confidence Intervals, and Other Measures of Statistical Accuracy , 1986 .

[2]  A Donner,et al.  Adjustments to the Mantel-Haenszel chi-square statistic and odds ratio variance estimator when the data are clustered. , 1987, Statistics in medicine.

[3]  Jacob Cohen A Coefficient of Agreement for Nominal Scales , 1960 .

[4]  W G Henderson,et al.  The statistical analysis of graft patency data in a clinical trial of antiplatelet agents following coronary artery bypass grafting. , 1988, Controlled clinical trials.

[5]  Gauderman Wj,et al.  Sample size calculations for ophthalmologic studies. , 1992 .

[6]  D Zucker,et al.  Testing the effect of treatment in experiments with correlated binary outcomes. , 1992, Biometrics.

[7]  S. Zeger,et al.  Longitudinal data analysis using generalized linear models , 1986 .

[8]  J. Lachin Introduction to sample size determination and power analysis for clinical trials. , 1981, Controlled clinical trials.

[9]  A. Donner,et al.  Randomization by cluster. Sample size requirements and analysis. , 1981, American journal of epidemiology.

[10]  A. Scott,et al.  A simple method for the analysis of clustered binary data. , 1992, Biometrics.

[11]  A Donner,et al.  Approaches to sample size estimation in the design of clinical trials--a review. , 1984, Statistics in medicine.

[12]  M M Shoukri,et al.  Estimating the number of clusters for the analysis of correlated binary response variables from unbalanced data. , 1992, Statistics in medicine.

[13]  J. Kalbfleisch,et al.  A Comparison of Cluster-Specific and Population-Averaged Approaches for Analyzing Correlated Binary Data , 1991 .

[14]  William G. Cochran,et al.  Sampling Techniques, 3rd Edition , 1963 .

[15]  G. Bonney,et al.  Logistic regression for dependent binary observations. , 1987, Biometrics.

[16]  Beth C. Gladen,et al.  The Use of the Jackknife to Estimate Proportions from Toxicological Data in the Presence of Litter Effects , 1979 .

[17]  Z Feng,et al.  Correlated binomial variates: properties of estimator of intraclass correlation and its effect on sample size calculation. , 1992, Statistics in medicine.

[18]  F. Hsieh,et al.  Sample size formulae for intervention studies with the cluster as unit of randomization. , 1988, Statistics in medicine.