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组响应 - 适应性随机化与延迟和缺失的响应
Guannan Zhai1, Yang Li2, Lixin Zhang3,4
1Department of Statistics, George Washington University, Washington, DC.
Statistics in medicine
|September 16, 2024
概括
这项研究为临床试验引入了一种新的群体响应适应性随机化 (RAR) 方法,通过更新基于群体响应而不是个人响应的分配来提高实用性. 新型设计保持了理论性质,并且在延迟或缺失数据的情况下表现良好.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 医学研究方法学 医学研究方法学
背景情况:
- 传统的响应适应性随机化 (RAR) 在每个患者响应后更新了分配,在许多临床环境中提出了实际挑战.
- 现有的RAR方法经常因患者反应数据延迟或缺失而困难,限制了它们在现实世界中的适用性.
研究的目的:
- 为临床试验提出一系列新的群体响应适应性随机化 (RAR) 程序.
- 通过引入基于组的更新来解决基于个人响应的RAR的不切实际性.
- 评估拟议的组RAR设计的性能和理论特性,特别是在数据延迟或丢失的场景中.
主要方法:
- 开发一种新的群体响应适应性随机化程序,根据患者总体响应更新分配.
- 对拟议设计的理论特性进行分析,并将其与现有的双适应性偏见硬币设计 (DBCD) 进行比较.
- 数字模拟用于在各种条件下评估性能,包括延迟和随机丢失的响应.
主要成果:
- 拟议的组双适应偏见硬币设计 (DBCD) 保持了与现有的DBCD相似的基本理论特性.
- 组RAR设计在涉及延迟和随机缺失患者反应的模拟中显示出强大的性能.
- 在真正的临床试验中成功地应用了新组RAR设计,展示了其实用性和优势.
结论:
- 拟议的群体响应适应性随机化为临床试验中基于个体响应的RAR提供了一个实用和有效的替代方案.
- 该组RAR设计适用于处理延迟和缺失响应数据,提高试验效率.
- 这些发现支持在临床研究中更广泛地应用群体响应适应性设计.
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