Estimation and visualization of heterogeneous treatment effects for multiple outcomes

Estimation and visualization of heterogeneous treatment effects for multiple outcomes
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多种结果的异质治疗效果的估计和可视化

DOI:
10.1002/sim.9638
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发表时间:
2022
影响因子:
2
通讯作者:
Yadohisa Hiroshi
Yadohisa Hiroshi
中科院分区:
医学3区
文献类型:
--
作者:
Yuki Shintaro;Tanioka Kensuke;Yadohisa Hiroshi

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我们在临床试验中考虑两组比较。目的是确定具有使治疗有效的特征的人群。这样的种群称为子群。这种识别可以通过估计治疗效果和识别治疗和协变量之间的相互作用来进行。对于单一结局,有几种方法可用于识别亚组。也有多个结果,但它们很难解释,不能应用于连续值以外的结果。因此,在本文中,我们提出了一种新的方法,允许一个简单的解释子群和处理连续和二进制的结果。该方法引入了潜在变量,并增加了Lasso稀疏约束的估计负荷,以方便解释的结果和协变量之间的关系。亚组的解释是通过可视化的治疗效果和潜在变量。由于我们正在进行稀疏估计,因此我们可以解释与治疗效应和亚组相关的协变量。仿真和真实的数据实例验证了该方法的有效性。
We consider two‐arm comparison in clinical trials. The objective is to identify a population with characteristics that make the treatment effective. Such a population is called a subgroup. This identification can be made by estimating the treatment effect and identifying the interactions between treatments and covariates. For a single outcome, there are several ways available to identify the subgroups. There are also multiple outcomes, but they are difficult to interpret and cannot be applied to outcomes other than continuous values. In this paper, we thus propose a new method that allows for a straightforward interpretation of subgroups and deals with both continuous and binary outcomes. The proposed method introduces latent variables and adds Lasso sparsity constraints to the estimated loadings to facilitate the interpretation of the relationship between outcomes and covariates. The interpretation of the subgroups is made by visualizing treatment effects and latent variables. Since we are performing sparse estimation, we can interpret the covariates related to the treatment effects and subgroups. Finally, simulation and real data examples demonstrate the effectiveness of the proposed method.
临床试验中部分患者的治疗效果模式。
DOI: 10.1093/biostatistics/5.3.465
发表时间: 2004
期刊: Biostatistics (Oxford, England)
影响因子: --
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