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. 2022 Jul 29;23(4):1056–1073. doi: 10.1093/biostatistics/kxac027

Table 4.

Average percent bias, empirical SE, and CP of the 95Inline graphic CI of Inline graphic, and Inline graphic across 1000 simulation replicates

 No. of testing sites Testing site size Method Inline graphic Inline graphic Inline graphic Inline graphic
Bias (SE) CP Bias (SE) CP Bias (SE) CP Bias (SE) CP
50 30 IndCov 0.2Inline graphic (0.134) 0.964 0.8Inline graphic (0.110) 0.992 3.9Inline graphic (0.124) 0.976 0.0Inline graphic (0.071) 0.952
ExchCov 0.6Inline graphic (0.125) 0.958 2.5Inline graphic (0.075) 0.971 1.1Inline graphic (0.085) 0.952 0.4Inline graphic (0.094) 0.958
ExchTwoS Inline graphic 0.1Inline graphic (0.066) 0.949 Inline graphic 0.1Inline graphic (0.067) 0.944 0.4Inline graphic (0.075) 0.938 0.1Inline graphic (0.053) 0.945
50 60 IndCov 0.1Inline graphic (0.088) 0.965 Inline graphic 0.7Inline graphic (0.077) 0.991 4.1Inline graphic (0.087) 0.980 0.0Inline graphic (0.051) 0.950
ExchCov 0.5Inline graphic (0.085) 0.958 1.5Inline graphic (0.055) 0.961 1.3Inline graphic (0.057) 0.966 0.3Inline graphic (0.067) 0.955
ExchTwoS 0.1Inline graphic (0.045) 0.955 0.1Inline graphic (0.045) 0.956 0.8Inline graphic (0.050) 0.939 0.0Inline graphic (0.039) 0.946
100 30 IndCov Inline graphic 0.5Inline graphic (0.089) 0.937 Inline graphic 5.0Inline graphic (0.079) 0.983 4.2Inline graphic (0.086) 0.967 0.1Inline graphic (0.048) 0.948
ExchCov Inline graphic 0.2Inline graphic (0.082) 0.949 Inline graphic 3.6Inline graphic (0.056) 0.958 2.2Inline graphic (0.059) 0.945 0.3Inline graphic (0.063) 0.957
ExchTwoS Inline graphic 0.1Inline graphic (0.046) 0.959 Inline graphic 1.2Inline graphic (0.051) 0.952 0.8Inline graphic (0.055) 0.940 0.2Inline graphic (0.037) 0.961
100 60 IndCov Inline graphic 0.4Inline graphic (0.059) 0.953 Inline graphic 5.1Inline graphic (0.052) 0.978 3.8Inline graphic (0.056) 0.969 0.1Inline graphic (0.035) 0.939
ExchCov Inline graphic 0.1Inline graphic (0.057) 0.954 Inline graphic 3.2Inline graphic (0.039) 0.941 1.8Inline graphic (0.040) 0.953 0.2Inline graphic (0.045) 0.956
ExchTwoS Inline graphic 0.2Inline graphic (0.033) 0.948 Inline graphic 0.9Inline graphic (0.034) 0.940 0.4Inline graphic (0.035) 0.955 0.0Inline graphic (0.027) 0.950

A mixed-effects model is used to estimate the treatment probability and the effects of cluster-level unmeasured confounder on the treatment are generated from a skewed normal distribution with location parameter to be Inline graphic1, scale parameter being 0.1 and shape parameter equals to 100. For the one-stage method, we consider the following situations: (1) using a working independent covariance matrix (IndCov); (2) using a working exchangeable covariance matrix for within subject correlation and assume independence between subjects (ExchCov). For the two-stage method, we consider using a working exchangeable covariance matrix for the first stage cluster-specific MSMs and combine the results using mixed-effects meta-analysis (ExchTwoS).