测试治疗效果异质性的样本大小要求在集群随机试验中的二元结果.
Lara Maleyeff1, Rui Wang1,2, Sebastien Haneuse1
1Department of Biostatistics, Harvard T. H. Chan School of Public Health, Boston, Massachusetts, USA.
Statistics in medicine
|November 17, 2023
概括
本研究引入了对集群随机试验 (CRT) 具有二进制结果的新样本大小方法. 这些方法有助于研究人员检测治疗效果异质性,这是强大的临床试验设计的关键步骤.
科学领域:
- 生物统计学 生物统计学
- 临床试验方法论 临床试验方法论
- 医疗保健服务研究 医疗服务研究
背景情况:
- 集群随机试验 (CRT) 被广泛使用,但检测治疗效果异质与二进制结果的样本大小确定仍然是一个挑战.
- 在CRT中对异质治疗效果的现有方法主要局限于连续结果,为二进制结果留下了一个空白.
研究的目的:
- 开发和验证样本大小程序,以测试二级集群随机试验中的治疗效果异质性.
- 为研究人员规划涉及二进制结果和效果修饰器的CRT提供实用工具.
主要方法:
- 在一个通用线性混合模型框架内开发二元效应修饰器的闭式样本大小表达式.
- 对连续效果修饰器实施计算效率高的蒙特卡洛方法.
- 模拟研究以评估拟议的样本大小方法的准确性,并与现有方法进行比较.
主要成果:
- 拟议的样本大小方法准确地估计了检测二进制结果的CRT中治疗效应异质性的所需样本大小.
- 公式和模拟结果证明了各种效果修饰器类型 (二进制和连续) 的方法的实用性.
- 举例说明,包括来自STOP CRC试验的数据,展示了开发的程序的实际应用.
结论:
- 开发的样本大小程序解决了二元结果CRT的关键方法差距.
- 这些方法将增强研究异质治疗效应的CRT的规划和统计能力.
- 该研究为研究人员提供了有价值的工具,旨在在复杂的试验设计中检测差异性治疗效应.
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