Simple methods for the estimation and sensitivity analysis of principal strata effects using marginal structural models: Application to a bone fracture prevention trial
Simple methods for the estimation and sensitivity analysis of principal strata effects using marginal structural models: Application to a bone fracture prevention trial
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使用边缘结构模型估计和敏感性分析主层效应的简单方法：在骨折预防试验中的应用
DOI：
10.1002/bimj.201800038
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发表时间：
2019
影响因子：
1.7
通讯作者：
Chiba Yasutaka
中科院分区：
文献类型：
--
作者：
Uemura Yukari;Taguri Masataka;Kawahara Takuya;Chiba Yasutaka
在随机临床试验中，除了对事件本身的影响外，通常还需要估计治疗对生活质量（QOL）的影响。当某些患者在QOL评分评估前发生事件时，研究者可以在随机化后比较事件定义的患者亚组之间的QOL评分。然而，由于随机化后的选择偏倚，这种分析可能会误导研究者的治疗效果，并导致矛盾的结果。最近的日本骨质疏松干预试验（JOINT-02）比较了联合治疗与单药治疗预防骨折的获益，证实了这一点;联合治疗组中未骨折亚组的平均QOL评分较高，但骨折亚组较低。为了解决这一问题，文献中讨论了主要分层效应（PSE），即在按潜在中间变量分层的个体亚组内估计的治疗效应。在本文中，我们描述了一个简单的程序，估计的PSE使用边际结构模型。该程序使用SAS代码进行估计。此外，我们提出了一个简单的敏感性分析方法来检查由此产生的估计。使用这些方法对JOINT-02数据进行的分析显示，对于两个亚组，联合治疗组的QOL评分均高于单药治疗组。
In randomized clinical trials, it is often of interest to estimate the effect of treatment on quality of life (QOL), in addition to those on the event itself. When an event occurs in some patients prior to QOL score assessment, investigators may compare QOL scores between patient subgroups defined by the event after randomization. However, owing to postrandomization selection bias, this analysis can mislead investigators about treatment efficacy and result in paradoxical findings. The recent Japanese Osteoporosis Intervention Trial (JOINT‐02), which compared the benefits of a combination therapy for fracture prevention with those of a monotherapy, exemplifies the case in point; the average QOL score was higher in the combination therapy arm for the unfractured subgroup but was lower for the fractured subgroup. To address this issue, principal strata effects (PSEs), which are treatment effects estimated within subgroups of individuals stratified by potential intermediate variable, have been discussed in the literature. In this paper, we describe a simple procedure for estimating the PSEs using marginal structural models. This procedure utilizes SAS code for the estimation. In addition, we present a simple sensitivity analysis method for examining the resulting estimates. The analyses of JOINT‐02 data using these methods revealed that QOL scores were higher in the combination therapy arm than in the monotherapy arm for both subgroups.
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