两个阶段的响应适应性随机化设计,用于二元结果的多臂试验
1Department of Biostatistics, University of Florida, Gainesville, Florida, USA.
Journal of biopharmaceutical statistics
|July 15, 2023
概括
响应适应性随机化通过使用中间结果来调整治疗分配来改善多臂临床试验. 玩赢家规则在对二进制结果的模拟中表现良好.
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:
- 适应性随机化方法提高了临床试验的效率和灵活性.
- 多臂双阶段设计传统上使用平等的随机化,可能低利用中间数据.
研究的目的:
- 开发响应适应性随机化为多臂临床试验的两阶段设计,具有二进制结果.
- 通过利用中间结果来改进治疗选择和分配.
主要方法:
- 拟议的应答自适应随机化为多臂试验的两阶段设计.
- 基于顺序设计和玩胜利者的规则,被认为是最佳分配.
- 基于最小化预期故障,平均样本大小和零假设下的样本大小的优化设计.
主要成果:
- 模拟研究表明,采用玩胜者规则的拟议适应性设计表现良好.
- 与传统的平等随机化相比,自适应设计可以更好地利用中间结果.
结论:
- 响应适应性随机化两阶段设计对于具有二元结果的多臂临床试验是有效的.
- 玩胜利者的规则是这些自适应设计的有前途的分配方法.
- 在胰腺腺癌的II期试验中证明了应用.
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