监测间隔对无进展生存的异质性影响
Zihang Zhong1, Min Yang1, Senmiao Ni1
1Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing, People's Republic of China.
Journal of applied statistics
|February 28, 2024
概括
癌症试验中的异质监测间隔可能会扭曲无进展生存 (PFS) 结果. 不一致的监测可能会增加假阳性率并高估治疗疗效,影响试验有效性.
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
- 瘤学研究研究
背景情况:
- 无进展生存率 (PFS) 是癌症临床试验中的关键代用终点.
- 准确的PFS评估受到计划监测间隔的挑战,导致未知的真实进展时间.
- 将治疗臂与不同的监测计划进行比较是一种常见的,但可能存在问题的做法.
研究的目的:
- 调查异质监测间隔对基于PFS的疗效结论有效性的影响.
- 量化监测间隔的变化对试验结果产生偏差的程度.
主要方法:
- 为一项双臂随机对照试验进行了全面的模拟研究.
- 用预定义的监测间隔模拟生存数据,考虑到各种审查率.
- 危险比率,假阳性率,功率和偏差在不同的环境下被分析.
主要成果:
- 监测间隔长度的异质性增加与更高的假阳性率有关.
- 在异质的监测间隔下观察到统计功率的高估.
- 监测间隔异质性的影响取决于瘤预后和数据审查比例.
结论:
- 建议在设计比较性癌症试验时保持一致的监测间隔,以确保有效性.
- 当不一致的间隔不可避免时,在分析过程中必须仔细考虑它们对PFS数据的影响.
- 这些发现使用第三期转移性结直肠癌试验数据集来说明.
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