在RCT中对异质治疗效果的预测建模:一个范围审查
Joe V Selby1, Carolien C H M Maas2,3, Bruce H Fireman1
1Division of Research, Kaiser Permanente Northern California, Pleasanton.
JAMA network open
|July 22, 2025
概括
随机临床试验 (RCT) 的预测建模在37%的研究中发现了临床上重要的治疗效应 (HTE) 异质性. 风险建模对于HTE来说比效果建模更具可信性,对效果模型来说验证至关重要.
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:
- 2020年预测方法治疗效果异质性 (PATH) 声明引入了预测建模,用于在随机临床试验 (RCT) 中识别治疗效果 (HTE) 的异质性.
- 提出了两种主要方法:风险建模和效果建模.
- 风险建模预测个人基线风险,检查跨风险层的治疗效果,而效果建模直接预测个人治疗效果.
研究的目的:
- 识别,描述和评估引用PATH声明的研究结果.
- 评估HTE在RCT中的预测建模的应用和可信性.
- 确定已识别的HTE的临床重要性.
主要方法:
- 在PubMed,谷歌学者,科学网和SCOPUS.US进行了全面的文献搜索.
- 报告被系统地审查,使用双重审查程序与裁决.
- 使用已确定的标准评估了HTE发现的可信性和临床重要性.
主要成果:
- 确定了分析162个RCT的65份报告,其中31份使用风险模型,41份使用效果模型.
- 可信的,临床上重要的HTE在37%的审查报告中被发现.
- 与效果建模 (32%) 相比,风险建模显示出更高的可信度 (87%),外部验证提高了效果模型的可信度.
结论:
- 多变量预测建模在37%的RCT报告中确定了可信和临床重要的HTE.
- 风险建模在确定HTE方面比效果建模更具可信性.
- 外部验证对于确定探索性效应模型发现的可信性至关重要.
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