两项心力衰竭试验的可转移到疾病登记处,使用个人患者数据
Lili Wei1, David M Phillippo2, Anoop Shah3
1School of Health and Wellbeing, University of Glasgow, Glasgow, UK.
Journal of clinical epidemiology
|September 2, 2023
概括
统计运输方法可以将随机对照试验 (RCT) 结果与现实世界注册表数据相适应. 这些方法,就像采样重量的逆概率一样,在心力衰竭患者中保持治疗效果的有效性.
科学领域:
- 临床流行病学 临床流行病学
- 生物统计学 生物统计学
- 医疗保健服务研究 医疗服务研究
背景情况:
- 随机对照试验 (RCT) 对治疗疗效至关重要,但往往缺乏现实世界的适用性.
- 统计传输方法提供了一种方法来弥合试验概括性和现实世界的设置之间的差距.
- 心力衰竭 (HF) 注册表代表了不同患者群体,通常被排除在RCT之外.
研究的目的:
- 评估统计传输方法在调整高射频RCT治疗效果到现实世界高射频注册表中的有用性.
- 为了比较基于回归和反向概率的采样重量 (IOSW) 方法治疗效应运输.
主要方法:
- 从COMET和DIG试验中获得的个人患者数据,以及来自苏格兰HF注册表的数据.
- 治疗对死亡率和复合结果的影响使用基于回归和IOSW方法进行了转运.
- 登记患者的特征 (年龄,功能,尿剂使用) 与试验参与者进行了比较.
主要成果:
- 注册患者通常年龄较大,功能较差,接受的循环利尿剂比试验参与者更多.
- 治疗效果的点估计在原始试验数据和使用IOSW运输的估计之间是相似的.
- 基于回归的运输也产生了类似的治疗效应估计,即使在注册表中分析不同的风险子组.
结论:
- 基于回归和IOSW的运输方法可用于调整试验效果估计的行政或注册数据.
- 这些方法可以提高RCT发现对更广泛的患者群体的适用性,只有中度的精度损失.
- 运输增强了临床试验证据对现实世界医疗保健决策的外部有效性.
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