使用真实世界的数据进行随机对照试验的模拟的考虑:从模拟MONALEESA-2的学习
Amanda M Kong1, Dionna Andrean1, Saarah Khan1
1Aetion, New York, NY 10001, USA.
现实世界数据 (RWD) 无法复制MONALEESA-2试验的研究结果,用于晚期乳腺癌治疗. 临床试验中的患者群体与常规护理中的患者群体有显著差异,这影响了治疗结果的概括性.
科学领域:
- 在瘤学瘤学.
- 药物监督 药物监督 药物监督
- 现实世界的证据.
背景情况:
- 随机对照试验 (RCT) 可能会排除可能从批准后治疗中受益的患者群体.
- 试验群体可能不准确地代表接受常规护理的患者,限制了概括性.
- 在MONALEESA-2试验中显示,在晚期乳腺癌中, ribociclib 加上 letrozole 对生存有好处.
研究的目的:
- 使用真实世界数据 (RWD) 模拟MONALEESA-2试验.
- 评估修改资格标准对试验模拟的影响.
- 为了在现实世界中比较 ribociclib + letrozole 和单独的 letrozole 之间的结果.
主要方法:
- 从链接的电子健康记录和索赔数据中识别出患有复发或转移性乳腺癌的绝经后妇女.
- 在 ribociclib 加上 letrozole 和 letrozole 组之间比较整体生存期.
- 利用Cox比例危险模型,对治疗权重和倾向得分匹配的反向概率.
主要成果:
- 实验队列 (n=132,406) 在应用试验入学标准后缩小到3912名患者.
- 与现实患者相比,试验参与者更年轻,患有更多的肝/肺转移.
- 实验中接受里博西基利布加莱特醇的患者具有较高的并发症评分和转移性负担;试验结果的模拟没有成功 (所有HR>1).
结论:
- 现实世界患者和临床试验参与者之间存在显著差异.
- 在RWD中的局限性,包括缺少的临床信息和不完整的死亡数据,阻碍了试验模拟.
- RWD对于了解患者护理和在常规临床实践中评估新疗法至关重要.
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