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在老年AML患者的前性随机试验HOVON-103中,外部和内部对照患者的可比性

Sjoerd J F Hermans¹, Jurjen Versluis¹, Erik D van Werkhoven^1,2

¹Erasmus University Medical Center Cancer Institute, Rotterdam, The Netherlands.

British journal of haematology

|May 26, 2025

概括

作为外部控制所使用的真实世界数据 (RWD) 需要验证. 虽然HARMONY联盟队列的生存率与试验对照表现相似,但荷兰癌症注册表队列的生存率较低,这突出了数据的局限性.

科学领域:

血液学血液学血液学
临床试验临床试验
现实世界的数据分析.

背景情况:

现实世界数据 (RWD) 在单臂临床试验中越来越多地被用作外部对照.
在使用RWD时存在方法上的挑战,需要验证RWD控制队列.
急性髓性白血病 (AML) 是一种严重的血液性恶性瘤.

研究的目的:

为了验证来自RWD的两个外部对照队伍与随机对照试验 (RCT) 进行对比.
评估RWD对照队伍的可比性,以便将来在比较分析中使用.
评估数据完整性和患者特征对RWD队列有效性的影响.

主要方法:

两个RWD队列 (HARMONY联盟和荷兰癌症注册) 与HOVON-103试验的对照组进行比较.
包括新诊断的65岁以上的AML患者接受标准诱导化疗.
应用1:1倾向得分校准匹配 (PSM) 来提高队列可比性.
评估整体存活率 (OS) 和无复发存活率 (RFS).

主要成果:

哈蒙尼联盟队列在PSM后2年,与HOVON-103对照组相比,显示了非显著不同的OS和RFS.
与HOVON-103对照组相比,荷兰癌症注册表队列的生存率降低了12%.
与试验对照组相比,外部队列的数据元素较少,缺失的数据更多,特别是关于世卫组织绩效评分.

结论:

在将RWD纳入比较分析之前,对外部控制队伍的验证至关重要.
缺少的数据,特别是并发症和残留混,可能会限制RWD对照队伍的可比性.
需要仔细的方法评估,以确保在临床试验中作为外部对照使用的RWD的可靠性.

关键词:

急性髓性白血病急性骨髓性白血病临床试验是指临床试验中的临床试验.外部控制臂的外部控制臂外部控制器的外部控制非随机化研究是非随机化研究.现实世界的数据数据.

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