用临床试验和现实世界的数据来源在异形性肺纤维化中使用外部控制武器
Aparna C Swaminathan1,2, Laurie D Snyder1,2, Hwanhee Hong2,3
1Department of Medicine.
概括
来自历史临床试验的外部控制 (EC) 提高了异常性肺纤维化 (IPF) 试验的效率. 然而,像患者登记册或电子健康记录 (EHR) 这样的现实数据源对IPF研究没有可比的结果.
科学领域:
- 肺病学和临床试验方法学
背景情况:
- 异形性肺纤维化 (IPF) 是一种渐进的肺病,需要新的治疗策略.
- 外部控制 (EC) 具有提高IPF临床试验效率的潜力.
- 来自各种数据源的EC与并发控制的直接可比性仍然未确定.
研究的目的:
- 通过使用历史随机临床试验 (RCT),肺纤维化基金会患者登记册和电子健康记录 (EHR) 数据,为IPF开发适合目的的外部控制 (EC).
- 在BMS-986020的II阶段RCT中,评估这些EC与并发控制臂的终点可比性.
主要方法:
- 从历史的RCT,患者登记册和EHR数据的数据策划,创建IPF EC.
- 从基线到26周的强迫生命能力 (FVC) 变化率的比较,在BMS-986020治疗臂,BMS-安慰剂臂和发达EC之间.
- 应用混合效应模型与反向概率权重进行比较分析.
主要成果:
- 在BMS-安慰剂组中,FVC下降率在26周时为-130.09毫升,与最初的RCT一致.
- 来自历史RCT安慰剂手臂的外部控制表明,治疗效果点估计在原始RCT的95%置信区间内.
- 来自肺纤维化基金会患者登记册和EHR数据的外部对照显示,FVC下降速度较慢,产生了原始RCT95%置信区间之外的治疗效果估计.
结论:
- 从历史的RCT安慰剂手臂中生成的外部对照提供了与IPF原始临床试验中观察到的初级治疗效果相似的初级治疗效果.
- 从真实世界数据源 (注册表或EHR) 获得的外部控制不能产生可比的初级处理效果.
- 来自历史RCT的EC可以作为未来IPFRCT的宝贵补充,而现实数据EC需要进一步的方法精细化,以便直接比较.
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