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一个警告故事:在现实世界的案例研究中评估治疗切换调整方法的性能
Nicholas R Latimer1,2, Alice Dewdney3, Marco Campioni4
1Sheffield Centre for Health and Related Research (SCHARR), University of Sheffield, Regent Court, 30 Regent Street, Sheffield, South Yorkshire, S1 4DA, UK. n.latimer@sheffield.ac.uk.
在随机对照试验 (RCT) 中治疗转换的统计调整方法可以产生不可靠的结果. 对这些方法的应用方式进行仔细审查对于准确的医疗技术评估至关重要.
科学领域:
- 卫生技术评估 卫生技术评估
- 临床试验方法论 临床试验方法论
- 在瘤学瘤学.
背景情况:
- 随机对照试验 (RCT) 中的治疗转换使健康技术评估复杂化.
- 统计方法用于调整治疗转换,但其实际性能没有得到充分记录.
- 一个结直肠癌RCT比较panitumumab与最佳支持性护理 (BSC),其中76%的患者从BSC切换到panitumumab,用于本案例研究.
研究的目的:
- 在现实世界RCT环境中评估治疗转换的统计调整方法的性能.
- 为了比较NICE决策支持单元技术支持文件16中描述的调整方法的有效性.
- 评估不同统计方法在临床试验中处理治疗转换时的可靠性.
主要方法:
- 在RCT数据集中应用了审查权重 (IPCW) 的反向概率,排名保存结构失效时间模型 (RPSFTM) 和两阶段估计 (TSE).
- 通过随后的RCT和不允许转换治疗的研究,确定了panitumumab的真正有效性.
- 估计的治疗效果就像BSC患者没有转换为panitumumab一样.
主要成果:
- 一些反向概率审查权重 (IPCW) 和两阶段估计 (TSE) 应用程序产生了接近真实值的治疗效应估计.
- 其他IPCW和TSE的应用产生了在预期的真实置信区间的边界或外的估计.
- 保持等级的结构性故障时间模型 (RPSFTM) 的表现不佳,产生接近1的估计,置信区间宽.
结论:
- 在RCT中改变治疗的统计调整方法可能会产生不可靠的结果.
- 每种调整方法的具体应用和实施必须严格评估可靠性.
- 需要仔细审查,以确保医疗技术评估中调整的分析结果的有效性.
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