Ukoumunne Obioha C, Davison Anthony C, Gulliford Martin C, Chinn Susan
Department of Public Health Sciences, King's College London, 5th Floor, Capital House, 42 Weston Street, London SE1 3QD, UK.
Stat Med. 2003 Dec 30;22(24):3805-21. doi: 10.1002/sim.1643.
The intraclass correlation coefficient rho plays a key role in the design of cluster randomized trials. Estimates of rho obtained from previous cluster trials and used to inform sample size calculation in planned trials may be imprecise due to the typically small numbers of clusters in such studies. It may be useful to quantify this imprecision. This study used simulation to compare different methods for assigning bootstrap confidence intervals to rho for continuous outcomes from a balanced design. Data were simulated for combinations of numbers of clusters (10, 30, 50), intraclass correlation coefficients (0.001, 0.01, 0.05, 0.3) and outcome distributions (normal, non-normal continuous). The basic, bootstrap-t, percentile, bias corrected and bias corrected accelerated bootstrap intervals were compared with new methods using the basic and bootstrap-t intervals applied to a variance stabilizing transformation of rho. The standard bootstrap methods provided coverage levels for 95 per cent intervals that were markedly lower than the nominal level for data sets with only 10 clusters, and only provided close to 95 per cent coverage when there were 50 clusters. Application of the bootstrap-t method to the variance stabilizing transformation of rho improved upon the performance of the standard bootstrap methods, providing close to nominal coverage.
组内相关系数ρ在整群随机试验的设计中起着关键作用。由于此类研究中整群的数量通常较少,从先前的整群试验中获得并用于为计划试验中的样本量计算提供信息的ρ估计值可能不准确。量化这种不精确性可能会很有用。本研究使用模拟方法比较了在平衡设计中为连续结果的ρ分配自助置信区间的不同方法。针对整群数量(10、30、50)、组内相关系数(0.001、0.01、0.05、0.3)和结果分布(正态、非正态连续)的组合进行了数据模拟。将基本区间、自助t区间、百分位数区间、偏差校正区间和偏差校正加速自助区间与使用应用于ρ的方差稳定变换的基本区间和自助t区间的新方法进行了比较。标准自助方法为95%区间提供的覆盖水平明显低于仅包含10个整群的数据集的名义水平,并且只有当有50个整群时才提供接近95%的覆盖。将自助t方法应用于ρ的方差稳定变换改善了标准自助方法的性能,提供了接近名义覆盖。