选择最佳的纵向集群随机设计与连续的结果:平行臂,交叉,或阶梯形
Jingxia Liu1,2, Fan Li3,4, Siobhan Sutcliffe1
1Division of Public Health Sciences, Department of Surgery, Washington University School of Medicine, St Louis, MO, USA.
Statistical methods in medical research
|August 11, 2025
概括
本研究确定了纵向集群随机试验的最佳设计. 最佳的闭合队列集群随机交叉试验在各种设计中提供了低成本和高功率的最佳平衡.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 流行病学 流行病学
背景情况:
- 纵向集群随机试验 (CRT) 对于评估小组级研究中的干预措施至关重要.
- 对平行臂,CRXO和SW-CRT设计的最佳设计 (OD) 已经独立研究.
- 不同的纵向设计中缺乏对全球OD的统一理解.
研究的目的:
- 在完整的纵向集群随机试验设计中比较最佳设计特征.
- 确定一个全球最佳设计,尽量减少所需功率的成本或在固定预算内最大限度地提高功率.
- 为了比较六个特定的纵向集群随机试验设计.
主要方法:
- 利用基于概括估计方程 (GEE) 的成本效益框架.
- 开发了算法来确定每个设计的每个周期的最佳集群和每个集群的最佳参与者数量.
- 采用区块可交换的相关性结构进行公平的比较.
主要成果:
- 根据所需功率的最低成本或固定预算的最高功率来确定最佳设计.
- 建议使用L=T-1治疗序列来获得最佳的闭合队列和重复的横截面SW-CRT,以最大限度地降低成本.
- 最佳的闭合队列CRXO试验成为全球的OD,实现了最低成本和最大功率.
结论:
- 最佳的闭合队列集群随机交叉 (CRXO) 试验代表了纵向研究的全球最佳设计.
- 与其他评估的纵向CRT设计相比,这种设计提供了最具成本效益和强大的方法.
- 结果为选择集群随机试验中最有效的设计提供了指导.
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