调整开关到多种处理:开关应该单独处理还是组合处理?

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES Statistical Methods in Medical Research Pub Date : 2025-01-17 DOI:10.1177/09622802241300049
Helen Bell Gorrod, Shahrul Mt-Isa, Jingyi Xuan, Kristel Vandormael, William Malbecq, Victoria Yorke-Edwards, Ian R White, Nicholas Latimer
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引用次数: 0

摘要

治疗转换在随机对照试验(RCTs)中很常见。参与者可能会切换到各种不同的治疗方法,所有这些治疗方法都可能有不同的治疗效果。针对假设估计的调整分析——估计在没有转换治疗的情况下会观察到的结果——主要集中在单一类型的转换上。在本研究中,我们评估了加权逆概率(IPCW)和两阶段估计(TSE)的应用性能,它们通过(i)分别调整每种类型的开关(“单独处理”)或(ii)调整组合开关而不区分切换到处理(“组合处理”)来调整多个开关。我们模拟了48种随机对照试验参与者可能切换到多种治疗方法的情景。切换比例、处理效果、切换处理次数和审查比例各不相同。方法性能指标包括限制平均生存时间的平均百分比偏差和模型收敛频率。在TSE和IPCW应用中,联合处理和单独处理均产生类似程度的偏倚。在研究的场景中,单独调整每种类型的开关与一起调整所有开关相比,没有明显的优势。
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Adjusting for switches to multiple treatments: Should switches be handled separately or combined?

Treatment switching is common in randomised controlled trials (RCTs). Participants may switch onto a variety of different treatments, all of which may have different treatment effects. Adjustment analyses that target hypothetical estimands - estimating outcomes that would have been observed in the absence of treatment switching - have focused primarily on a single type of switch. In this study, we assess the performance of applications of inverse probability of censoring weights (IPCW) and two-stage estimation (TSE) which adjust for multiple switches by either (i) adjusting for each type of switching separately ('treatments separate') or (ii) adjusting for switches combined without differentiating between switched-to treatments ('treatments combined'). We simulate 48 scenarios in which RCT participants may switch to multiple treatments. Switch proportions, treatment effects, number of switched-to treatments and censoring proportions were varied. Method performance measures included mean percentage bias in restricted mean survival time and the frequency of model convergence. Similar levels of bias were produced by treatments combined and treatments separate in both TSE and IPCW applications. In the scenarios examined, there was no demonstrable advantage associated with adjusting for each type of switch separately, compared with adjusting for all switches together.

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来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
期刊介绍: Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)
期刊最新文献
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