临床试验中的随机化,采用小样本大小,使用组序列设计
Daniel Bodden1, Ralf-Dieter Hilgers1, Franz König2
1Institute of Medical Statistics, RWTH Aachen University, Aachen, Germany.
PloS one
|June 13, 2025
概括
随机化程序在组序列试验中显著影响I型错误和功率. 适当的选择和报告对于小样本临床试验的有效性至关重要.
科学领域:
- 临床试验 临床试验
- 生物统计学 生物统计学
- 统计学方法论 统计学方法论
背景情况:
- 组序列设计允许早期试验停止有效性或徒劳性.
- 在这些试验的中间和最终分析中,往往需要平衡的样本大小.
- 随机化程序的选择可能受到平衡样本大小的需要的限制.
研究的目的:
- 调查随机化程序对I型错误和功率在小样本临床试验中的影响,使用组序列设计.
- 评估已发表的群体顺序试验中的随机化方法报告情况.
- 为组序列设计提出选择适当随机化程序的框架.
主要方法:
- 关于在组顺序试验中随机化报告的文献综述.
- 模拟研究通过不同的随机化程序和组序列设计 (Pocock,O'Brien-Fleming,Lan-DeMets,反向正常组合测试) 评估I型错误和功率.
主要成果:
- 许多已发表的组序列试验缺乏足够的随机化细节.
- 糟糕的随机化实现可以膨胀I型错误率.
- 某些组合 (例如,反向正常测试与 permuted block随机化) 可以降低统计能力;Lan-DeMets设计对分配比率偏差更有稳定性.
结论:
- 建议为选择小试验最佳组序列设计和随机化程序组合的框架.
- 需要在团队顺序临床试验中改进随机化方法的报告.
- 组序列设计的有效性可能会受到不适当或报告不充分的随机化程序的损害.
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