步骤设计的样本大小计算,治疗效果可能随着干预时间的持续时间而变化
James P Hughes1, Wen-Yu Lee2, Andrea B Troxel2
1Department of Biostatistics, University of Washington, Seattle, WA, 98195, USA. jphughes@uw.edu.
概括
如果干预效应随着时间的推移而发生变化,阶段形试验可能会产生误导性的结果. 本研究介绍了使用暴露时间指标模型计算时间变化的治疗效应的功率试验方法.
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
- 公共卫生干预措施 公共卫生干预措施
背景情况:
- 阶段形设计是评估集群随机干预的常见方法.
- 标准分析通常假设立即和持续的干预效应 (即时治疗效应假设).
- 违反这一假设可能会导致显著偏差的结果.
研究的目的:
- 为了解决阶段形试验中即时治疗效果假设的局限性.
- 提供用于驱动阶段试验的方法,当治疗效果随着暴露时间的变化而变化时.
- 在这个设计中引入一个分析时间变化的治疗效果的框架.
主要方法:
- 讨论在当立即治疗效果假设被违反时,需要适当的效果措施.
- 描述了如何使用曝光时间指标 (ETI) 模型为步骤试验提供动力.
- 专注于时间变化的治疗效应的加权平均值作为估计值.
主要成果:
- 展示了如何在治疗效果不立即或不稳定时为阶段形试验提供动力.
- 将拟议的方法应用于ADDRESS-BP试验.
- 强调考虑干预效应的时间性质的重要性.
结论:
- 在阶段形试验中,立即治疗效果的假设可能是有问题的.
- 暴露时间指标模型提供了一种可靠的方法来分析时间变化的治疗效应.
- 准确的试验动力和分析需要仔细考虑随时间推移的干预效应动态.
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