PCSK9抑制剂的有效性:基于现实世界电子健康记录的目标试验仿真框架
Giulia Barbati1, Caterina Gregorio1,2,3, Arjuna Scagnetto4
1Biostatistics Unit, Department of Medical Sciences, University of Trieste, Trieste, Italy.
PloS one
|August 22, 2024
概括
蛋白转化酶抑制剂 (PCSK9-i) 显著降低了符合条件的患者的死亡风险,尽管处方率低. 这项观察性研究证实了PCSK9-i在预防心血管事件,特别是所有原因死亡率方面的有效性.
科学领域:
- 心血管医学 心血管医学
- 药理学 药理学是指药理学的学科.
- 医疗保健服务研究 医疗服务研究
背景情况:
- 低密度脂蛋白 (LDL) 胆固醇是预防和治疗心血管疾病 (CVD) 的关键目标.
- 蛋白转化酶抑制剂 (PCSK9-i) 是一种用于降低LDL和预防心血管事件的新疗法.
- 意大利药品管理局 (AIFA) 最近在2022年6月对资格标准的变化影响了PCSK9-i的可访问性.
研究的目的:
- 估计符合PCSK9-i治疗条件的受试者的患病率.
- 使用目标试验仿真 (TTE) 方法评估PCSK9-i在减少心血管事件方面的有效性.
- 评估PCSK9-i在所有原因的死亡和住院治疗中的实际有效性.
主要方法:
- 2017年7月至2020年12月使用电子健康记录 (EHR) 的观察性研究.
- 根据AIFA标准确定了符合条件的受试者,并分析了那些接受PCSK9-i治疗的人.
- 使用治疗权重的逆概率 (IPTW) 和竞争风险模型来估计平均治疗效应 (ATE) 和条件平均治疗效应 (CATE).
主要成果:
- 在1976名符合条件的受试者中,只有161人 (8%) 接受PCSK9-i治疗.
- 接受治疗的患者患有更严重的心血管疾病,年轻,主要是男性,更经常服用他类药物.
- 治疗PCSK9-i与所有原因死亡的显著减少以及首次住院的相对风险较低有关.
结论:
- PCSK9-i被处方给少数符合条件的患者,突出了潜在的接入障碍.
- 经过TTE分析证实PCSK9-i在降低心血管风险方面的有效性,与随机临床试验 (RCT) 结果一致.
- PCSK9-i显示出对所有死因的显著保护,扩大了RCT复合CV结果之外的证据.
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