使用真实数据模拟现有的前列腺癌治疗试验:挑战和经验教训
Caroline Chesang1, Linda D Sharples1, Christen M Gray2
1Department of Medical Statistics, London School of Hygiene and Tropical Medicine, Keppel Street, WC1E 7HT, London, UK.
Journal of clinical epidemiology
|March 27, 2025
概括
现实世界数据 (RWD) 成功模拟了前列腺癌试验,显示放射治疗加激素治疗 (RT + HT) 与单独激素治疗 (HT) 相比,改善了生存率. 这证实了RWD用于估计高风险前列腺癌患者的治疗效果.
科学领域:
- 在瘤学瘤学.
- 流行病学 流行病学
- 医疗信息学 医疗信息学
背景情况:
- 随机对照试验 (RCT) 是黄金标准,但成本高且耗时.
- 现实世界数据 (RWD) 为估计治疗效果提供了一个潜在的替代方案.
- 模拟RWD的RCT可以增加对其对试验未解决的研究问题有效性的信心.
研究的目的:
- 模拟PR07试验使用来自英格兰的国家链接数据集.
- 为了比较放射治疗加激素治疗 (RT+HT) 与激素治疗 (HT) 的有效性,仅用于高风险的前列腺癌.
- 评估使用RWD模仿RCT方法的可行性和挑战.
主要方法:
- 利用来自英格兰 (2014-2020) 的国家链接的行政和临床数据集.
- 模拟了PR07试验设计,因RWD特征而调整更长的宽限期 (4-6个月).
- 采用克隆-审查-加权 (CCW) 和基准-CCW方法来控制混并考虑治疗开始时间.
主要成果:
- 模拟试验包括2690名高风险前列腺癌患者.
- CCW分析 (6个月宽限期) 显示RT+HT与0.48 (95%CI:0.34-0.60) 的危险比率 (HR) 和15.1%更高的7年生存率相关.
- 里程碑式的CCW方法产生了0.58的HR (95%CI:0.51-0.65) 和10.9%更高的RT+HT的7年生存率.
结论:
- 来自RWD模拟的结果与PR07试验基本一致,表明RT+HT比单独的HT提高了生存率.
- 精确的复制没有实现,差异归因于定义零时间和宽限期的挑战.
- 该研究证实了RWD对估计治疗效果的有用性,并提供了解决模拟挑战的方法.
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