在多个复合人群设计中,对正常数据和基线调整的盲目样本大小进行重新计算
Roland G Gera1, Tim Friede1,2
1Department of Medical Statistics, University Medical Centre Göttingen, Göttingen, Germany.
Biometrical journal. Biometrische Zeitschrift
|June 13, 2023
概括
这项研究引入了一种新的试验设计,用于分析个性化医学中的复合人群. 该方法有效测试治疗效果并管理样本大小,确保针对性治疗的可靠结果.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 个性化医疗是个性化的医疗.
背景情况:
- 对个性化医学和向治疗的亚种群分析越来越感兴趣.
- 需要强大的试验设计和分析方法来评估特定患者群体的治疗效果.
研究的目的:
- 提出一种新的试验设计,用于分析复合群体,定义为分离子集的集合.
- 开发测试治疗效果和计算这些复合群体样本大小的方法.
主要方法:
- 定义复合种群并将试验设计应用于具有随机基线共变量的正常分布的终点.
- 在复合人群测试中使用逆正常组合函数组合子集水平的p值.
- 使用封闭的测试程序和多变量正常分布进行假设测试和样本大小计算.
主要成果:
- 模拟显示I型错误率没有显著的膨胀.
- 建议的方法在重新计算样本大小后有效地实现或接近目标功率.
结论:
- 开发的试验设计和分析方法在个性化医学中对复合人群有效.
- 该方法为评估亚群中的向治疗提供了一个统计学上健全的框架.
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