与随机试验对比观察健康记录数据的校准
David Merola1, Ulka Campbell1, David Lenis1
1Aetion, New York, New York.
JAMA network open
|September 30, 2024
概括
与电子健康记录 (EHR) 模拟随机临床试验 (RCT) 显示了不同的生存结果. 这凸显了在电子健康记录中需要精选的瘤学数据,以准确评估治疗有效性.
科学领域:
- 在瘤学瘤学.
- 医疗信息学 医疗信息学
- 临床试验方法论 临床试验方法论
背景情况:
- 使用健康记录数据评估治疗有效性需要明确的准确性条件.
- 使用电子健康记录 (EHR) 数据模拟随机临床试验 (RCT) 可以帮助澄清这些情况.
- 这项研究试验了KEYNOTE-189 RCT的模拟,使用美国的EHR数据源.
研究的目的:
- 使用商用EHR数据源对KEYNOTE-189 RCT进行模拟试验.
- 为了比较基于EHR的队列和原始RCT之间的结果.
- 识别潜在的差异,并为未来使用EHR数据进行临床试验模拟提供信息.
主要方法:
- 一项回顾性队列研究利用了从2007年4月到2023年2月的EHR数据库,涵盖了52个美国卫生系统.
- 资格标准与KEYNOTE-189 RCT非常相似,包括转移性非小细胞肺癌患者接受一线治疗.
- 排除标准包括状组织学,其他原发性恶性瘤和EGFR/ALK变异. 结果是12个月生存概率和死亡风险比率.
主要成果:
- 这项研究包括1854名患者,589人接受了潘布罗利祖马布加化疗,1265人仅接受化疗.
- 在EHR pembrolizumab组中,12个月的生存概率为0.60,而在RCT中为0.69,在EHR化疗组中为0.58,而在RCT中为0.49.
- 在EHR队列中,死亡风险比为0.95,而在KEYNOTE-189RCT中为0.49.
结论:
- 基于EHR的RCT仿真结果与基准KEYNOTE-189试验不一致.
- 差异可能源于患者群体的差异,治疗管理,数据采集,根据指示进行混和治疗交叉.
- 未来的可行性评估需要EHR数据源与精心策划的,瘤学特异性的措施,以准确的临床试验模拟.
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