对自适应平台试验的实践审查
Natalia Alejandra Angeloni1, Neill Kj Adhikari2, François Lamontagne3
1Ross Tilley Burn Centre, Sunnybrook Health Sciences Centre - 2075 Bayview Ave, Toronto, Ontario, Canada; Institute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto - 155 College Street, Toronto, Ontario, Canada.
Transfusion medicine reviews
|November 29, 2025
概括
适应性平台试验 (APT) 提供高效的多疗法测试,但可能依赖于不那么严格的终点或复杂的方法,引入新的风险. 仔细评估APTs至关重要,因为它们扩展到新的医学领域.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 基于证据的医学基于证据的医学.
背景情况:
- 传统的随机对照试验 (RCT) 是严格的,但缓慢和资源密集的.
- 适应性平台试验 (APT) 允许在一个协议下测试多种干预措施,随着数据的出现增加或放弃武器.
- 应用技术具有潜在的优势,但也存在需要批判性评估的权衡.
研究的目的:
- 批判性地评估三个具有里程碑意义的适应性平台试验.
- 确定APT设计的好处和潜在的陷.
- 为临床医生和研究人员提供有关APT有效使用的信息.
主要方法:
- 对三个特定的适应性平台试验进行了审查和批判性评估:I-SPY2,RECOVERY和REMAP-CAP.
- 对试验设计,自适应随机化方法,终点选择和报告质量的分析.
- 综合了最近关于APT异质性和报告的系统审查中的发现.
主要成果:
- I-SPY2使用贝叶斯适应性随机化和代用终点加速了瘤学研究,但效率依赖于中间结果.
- RECOVERY试验的成功受到了卫生系统基础设施和流行病环境的影响,而不仅仅是其设计.
- REMAP-CAP的响应适应性随机化放大了噪音,使患者暴露在无效的COVID-19疗法中.
- 一项系统性审查发现APT之间存在显著的异质性和次优报告.
结论:
- APT并非本质上优于传统的RCT;速度增长可能涉及不那么严格的终点或复杂的方法,增加错误风险.
- 像RECOVERY这样的APT的成功取决于环境,受外部因素的影响.
- 临床医生和研究人员必须了解APT的潜在好处和固有的风险,因为它们被采用在输血医学等新领域.
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