研究设计用于对复发事件和死亡的受限平均时间分析
1Department of Biostatistics and Medical Informatics, School of Medicine and Public Health, University of Wisconsin-Madison, Madison, Wisconsin, USA.
Biometrics
|August 23, 2023
概括
新的工具有助于研究人员计算临床试验的样本大小和功率,使用受限平均有利时间 (RMT-IF) 计算复发事件和死亡复合终点. 这种方法有助于更准确地设计未来的试验.
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
- 生存分析的分析.
背景情况:
- 在临床试验中,结合复发事件和死亡的复合终点至关重要.
- 受限制的平均时间有利 (RMT-IF) 是一种用于这些终点的新分析方法.
- 使用RMT-IF设计试验需要准确的样本大小和功率计算.
研究的目的:
- 为使用RMT-IF进行试验开发用于计算样本大小和功率的工具.
- 为从业者提供基于RMT-IF的设计未来临床试验的方法.
- 为了促进RMT-IF在分析复合终点中的应用.
主要方法:
- 制定结果作为一个多状态马尔科夫过程,对于反复发生的事件具有过渡状态,而对于死亡则具有吸收状态.
- 假设时间均的过渡强度,取决于过去事件的数量.
- 使用可西安分布属性和用于审查的减少估计器方差来推导RMT-IF效应大小.
主要成果:
- 在现实环境中开发精确的样本大小和功率近似公式.
- 通过分析心血管试验以确定未来试验设计参数来证明该方法的实用性.
- 在CRAN上可获得的"rmt" R包中包含的程序.
结论:
- 开发的工具为RMT-IF提供了准确的样本大小和功率计算.
- 这些方法支持设计和分析具有复合终点的临床试验.
- "rmt"套件为研究人员提供了实际实施.
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