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Quint: An R package for the identification of subgroups of clients who differ in which treatment alternative is best for them

机译:五胞胎:一个R程序包用于识别在哪种治疗方案最适合他们时有所不同的客户亚组

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摘要

In the analysis of randomized controlled trials (RCTs), treatment effect heterogeneity often occurs, implying differences across (subgroups of) clients in treatment efficacy. This phenomenon is typically referred to as treatment-subgroup interactions. The identification of subgroups of clients, defined in terms of pretreatment characteristics that are involved in a treatment-subgroup interaction, is a methodologically challenging task, especially when many characteristics are available that may interact with treatment and when no comprehensive a priori hypotheses on relevant subgroups are available. A special type of treatment-subgroup interaction occurs if the ranking of treatment alternatives in terms of efficacy differs across subgroups of clients (e.g., for one subgroup treatment A is better than B and for another subgroup treatment B is better than A). These are called qualitative treatment-subgroup interactions and are most important for optimal treatment assignment. The method QUINT (Qualitative INteraction Trees) was recently proposed to induce subgroups involved in such interactions from RCT data. The result of an analysis with QUINT is a binary tree from which treatment assignment criteria can be derived. The implementation of this method, the R package quint, is the topic of this paper. The analysis process is described step-by-step using data from the Breast Cancer Recovery Project, showing the reader all functions included in the package. The output is explained and given a substantive interpretation. Furthermore, an overview is given of the tuning parameters involved in the analysis, along with possible motivational concerns associated with choice alternatives that are available to the user.
机译:在对随机对照试验(RCT)进行分析时,常常会出现治疗效果异质性,这意味着不同治疗对象(子组)的治疗效果存在差异。这种现象通常称为治疗-亚组相互作用。根据治疗-亚组相互作用中涉及的预处理特征来定义客户亚组是一项方法学上的挑战性任务,尤其是当有许多可与治疗相互作用的特征可用时,以及在相关亚组上没有全面的先验假设时可用。如果治疗方案在功效方面的排名在客户的亚组中不同(例如,对于一个亚组,治疗A优于B,而对于另一个亚组治疗B优于A),则会发生特殊类型的治疗-亚组相互作用。这些被称为定性治疗-亚组相互作用,对于优化治疗分配最重要。最近提出了QUINT(定性交互树)方法,以从RCT数据中诱发参与此类交互作用的子组。 QUINT的分析结果是一个二叉树,可以从中导出处理分配标准。此方法的实现(R包五重奏)是本文的主题。使用来自乳腺癌恢复项目的数据逐步描述了分析过程,向读者显示了包装中包含的所有功能。将对输出进行解释并进行实质性解释。此外,概述了分析中涉及的调整参数,以及与用户可用的选择替代方案相关的可能的动机问题。

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