Hemming Karla, Lilford Richard, Girling Alan J
Department of Public Health, Epidemiology and Biostatistics, University of Birmingham, U.K.
Stat Med. 2015 Jan 30;34(2):181-96. doi: 10.1002/sim.6325. Epub 2014 Oct 24.
Stepped-wedge cluster randomised trials (SW-CRTs) are being used with increasing frequency in health service evaluation. Conventionally, these studies are cross-sectional in design with equally spaced steps, with an equal number of clusters randomised at each step and data collected at each and every step. Here we introduce several variations on this design and consider implications for power. One modification we consider is the incomplete cross-sectional SW-CRT, where the number of clusters varies at each step or where at some steps, for example, implementation or transition periods, data are not collected. We show that the parallel CRT with staggered but balanced randomisation can be considered a special case of the incomplete SW-CRT. As too can the parallel CRT with baseline measures. And we extend these designs to allow for multiple layers of clustering, for example, wards within a hospital. Building on results for complete designs, power and detectable difference are derived using a Wald test and obtaining the variance-covariance matrix of the treatment effect assuming a generalised linear mixed model. These variations are illustrated by several real examples. We recommend that whilst the impact of transition periods on power is likely to be small, where they are a feature of the design they should be incorporated. We also show examples in which the power of a SW-CRT increases as the intra-cluster correlation (ICC) increases and demonstrate that the impact of the ICC is likely to be smaller in a SW-CRT compared with a parallel CRT, especially where there are multiple levels of clustering. Finally, through this unified framework, the efficiency of the SW-CRT and the parallel CRT can be compared.
阶梯楔形整群随机试验(SW-CRTs)在卫生服务评估中的使用频率越来越高。传统上,这些研究采用等间距步骤的横断面设计,每一步随机分配的整群数量相等,并在每一步收集数据。在此,我们介绍这种设计的几种变体,并考虑其对检验效能的影响。我们考虑的一种修改是不完全横断面SW-CRT,即每一步的整群数量不同,或者在某些步骤(例如实施期或过渡期)不收集数据。我们表明,具有交错但平衡随机化的平行整群随机试验(CRT)可被视为不完全SW-CRT的一种特殊情况。具有基线测量的平行CRT也是如此。并且我们将这些设计扩展到允许多层整群,例如医院内的病房。基于完全设计的结果,使用Wald检验并假设广义线性混合模型获得治疗效应的方差-协方差矩阵,推导出检验效能和可检测差异。通过几个实际例子对这些变体进行了说明。我们建议,虽然过渡期对检验效能的影响可能较小,但如果它们是设计的一个特点,则应将其纳入考虑。我们还展示了一些例子,其中SW-CRT的检验效能随着组内相关系数(ICC)的增加而增加,并证明与平行CRT相比,ICC在SW-CRT中的影响可能较小,特别是在存在多层整群的情况下。最后,通过这个统一的框架,可以比较SW-CRT和平行CRT的效率。