对比分析策略,以平衡站点级特征在阶段集群随机试验:一个模拟研究研究
Clement Ma1,2,3, Alina Lee1,2, Darren Courtney4,5
1Biostatistics Core, Centre for Addiction and Mental Health, Toronto, ON, Canada.
BMC medical research methodology
|September 12, 2023
概括
在阶梯集群随机试验 (SWCRT) 中平衡集群特征可以提高效率,特别是当治疗效果随着时间的推移而增加时. 在这些复杂的研究设计中,预平衡对于减轻效率损失至关重要.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 步集群随机试验 (SWCRT) 顺序地将集群随机化为干预措施.
- 集群特征的顺序失衡可能会导致SWCRT估计偏差.
- 本研究研究了SWCRT中平衡集群级特征的影响.
研究的目的:
- 检查SWCRT中平衡集群级特征的影响.
- 用一个新的指数量化集群级失衡.
- 在不同情景下评估不平衡对试验效率的影响.
主要方法:
- 开发了一种基于斯皮尔曼相关性和等级回归的新型失衡指数.
- 进行了模拟研究,对不同地点数量,样本大小,交叉时间点,ICC和效果大小进行了变化.
- 评估了持续和渐进的"学习"治疗效应,通过RRMSE测量效率和相对平均偏差.
主要成果:
- 完全平衡的设计始终显示出最高的效率 (最低的RRMSE),特别是学习效应.
- 不平衡的增加导致了效率的下降趋势.
- 在12个站点的示例中,效率损失范围从52.5%到191.9%,在更大的样本大小,更多站点,更小的ICC和更大的效果大小中观察到的改善.
结论:
- 集群特征的预平衡显著提高了SWCRT的效率,特别是当治疗效应表现出学习曲线时.
- 预平衡的好处在考虑动态治疗效应时最为明显.
- 这凸显了前平衡对于具有时间变化的效应的SWCRT中可靠估计的重要性.
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