Grantham Kelsey L, Forbes Andrew B, Hooper Richard, Kasza Jessica
School of Public Health and Preventive Medicine, Monash University, Melbourne, VIC, Australia.
Wolfson Institute of Population Health, Queen Mary University of London, London, UK.
Stat Methods Med Res. 2025 Apr;34(4):701-716. doi: 10.1177/09622802251317613. Epub 2025 Feb 16.
The stepped wedge design is an appealing longitudinal cluster randomised trial design. However, it places a large burden on participating clusters by requiring all clusters to collect data in all periods of the trial. The staircase design may be a desirable alternative: treatment sequences consist of a limited number of measurement periods before and after the implementation of the intervention. In this article, we explore the relative efficiency of the stepped wedge design to several variants of the 'basic staircase' design, which has one control followed by one intervention period in each sequence. We model outcomes using linear mixed models and consider a sampling scheme where each participant is measured once. We first consider a basic staircase design embedded within the stepped wedge design, then basic staircase designs with either more clusters or larger cluster-period sizes, with the same total number of participants and with fewer total participants than the stepped wedge design. The relative efficiency of the designs depends on the intracluster correlation structure, correlation parameters and the trial configuration, including the number of sequences and cluster-period size. For a wide range of realistic trial settings, a basic staircase design will deliver greater statistical power than a stepped wedge design with the same number of participants, and in some cases, with even fewer total participants.
阶梯楔形设计是一种有吸引力的纵向整群随机试验设计。然而,它给参与的群组带来了巨大负担,因为要求所有群组在试验的所有阶段都收集数据。阶梯式设计可能是一种理想的替代方案:治疗序列由干预实施前后数量有限的测量阶段组成。在本文中,我们探讨了阶梯楔形设计相对于“基本阶梯式”设计的几种变体的相对效率,在每个序列中,基本阶梯式设计有一个对照阶段,随后是一个干预阶段。我们使用线性混合模型对结果进行建模,并考虑一种每个参与者只测量一次的抽样方案。我们首先考虑嵌入在阶梯楔形设计中的基本阶梯式设计,然后考虑具有更多群组或更大群组-阶段规模的基本阶梯式设计,参与者总数相同且比阶梯楔形设计的参与者总数更少。设计的相对效率取决于群组内相关结构、相关参数和试验配置,包括序列数量和群组-阶段规模。对于广泛的实际试验设置,与参与者数量相同甚至在某些情况下参与者总数更少的阶梯楔形设计相比,基本阶梯式设计将具有更大的统计效力。