随后的抗癌疗法在随机分配给单独安慰剂或最佳支持性护理的患者中
Christopher Rios1, Alyson Haslam2, Timothée Olivier3
1School of Medicine, University of California, San Francisco, CA, USA.
概括
在与非活性对照进行的瘤学试验中,近一半的患者接受了后续治疗,质疑这些设计的有效性. 这凸显了在癌症临床试验中需要合理的比较者选择的需要.
科学领域:
- 在瘤学瘤学.
- 临床试验设计 临床试验设计
- 监管科学 监管科学
背景情况:
- 瘤随机对照试验 (RCT) 有时使用安慰剂或最佳支持性护理 (BSC) 作为对照组.
- 当对照患者后来接受活性抗癌疗法时,这些惰性对照的适当性是可疑的.
研究的目的:
- 评估最近FDA对抗癌药物注册试验中使用惰性控制臂的合理性.
- 评估随后的抗癌治疗对这些试验中的对照患者的影响.
主要方法:
- 对2009年1月至2024年11月期间批准的抗癌药物FDA注册试验的横截面分析.
- 包括在晚期/转移性疾病中仅使用安慰剂或BSC对照臂的RCT,报告了对照者的随后系统治疗.
- 抽象的试验特征,交叉设计,后续治疗率和结果;使用描述性统计和回归分析.
主要成果:
- 在18项符合条件的试验中,49%的对照组患者接受了随后的全身治疗.
- 与非交叉试验 (40.7%) 相比,交叉设计试验的后续治疗率更高 (66.0%).
- 发现了一个显著的反向关联:每增加10%的对照患者接受后续治疗时,危险比率下降0.07.
结论:
- 在对照组中后续治疗的高率挑战了瘤学RCT中惰性比较剂的适当性.
- 不适当地使用非活性对照剂可能会人工膨胀观察到的治疗效应.
- 强调需要监管机构和伦理委员会确保合理的比较者选择,特别是在弱势患者群体中.
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