超越危险比率:适当的统计方法来量化免疫瘤治疗的临床有效性 - 荷兰的例子
Isaac Corro Ramos1, Venetia Qendri2, Maiwenn Al2
1Institute for Medical Technology Assessment (iMTA), Erasmus University Rotterdam, Burgemeester Oudlaan 50, Rotterdam, 3062 PA, The Netherlands. corroramos@imta.eur.nl.
BMC medical research methodology
|October 31, 2024
概括
评估瘤治疗的PASKWIL-2023标准不适用于免疫瘤 (IO) 疗法,因为违反了比例危险假设. 建议使用其他方法,如限制平均存活时间,以准确评估治疗效果.
科学领域:
- 在瘤学瘤学.
- 生物统计学 生物统计学
- 临床试验分析
背景情况:
- 荷兰癌症药物评估委员会使用PASKWIL-2023标准来评估息治疗.
- 这些标准依赖于总生存 (OS) 和无进展生存 (PFS) 终点,并为中位生存和危险比率 (HR) 设定了特定的值.
- 对于HR有效性至关重要的比例危险 (PH) 假设在免疫瘤学 (IO) 疗法中经常被违反,使得标准不合适.
研究的目的:
- 确定和评估用于表示IO疗法的临床疗效的统计方法.
- 评估目前PASKWIL-2023标准对IO治疗的适用性.
- 在IO治疗评估中提出量化治疗有效性的替代方法.
主要方法:
- 对MEDLINE和EMBASE数据库进行了系统的文献审查.
- 包括的研究重点是IO治疗有效性的统计方法,生存数据分析,并解决了PH假设问题.
- 搜索涵盖了从创建到2022年8月31日的数据,包括方法研究,随机对照试验和讨论论文.
主要成果:
- 在选了1,035个独特的参考文献后,包括了17个出版物,另外43个通过雪球识别.
- 当PH假设不满足时,PASKWIL-2023标准在方法上有缺陷.
- 在非PH的情况下,单个总结统计数据是不够的;解释需要卡普兰-梅尔曲线和"无参数"措施,如受限制的平均存活时间 (RMST) 是首选的.
结论:
- 危险比率 (HR) 经常在没有验证比例危险 (PH) 假设的情况下使用,从而损害了PASKWIL-2023标准对息性瘤治疗的有效性.
- 在非PH条件下,除了HR之外的替代措施是必要的,以准确量化治疗效果.
- 建议采取逐步的方法来选择合适的方法,可能导致修订PASKWIL-2023标准或其他临床领域的类似框架.
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