Yizhen Xu, Ji Soo Kim, Laura K Hummers, Ami A Shah, Scott L Zeger
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引用次数: 0
Abstract
Dynamic prediction of causal effects under different treatment regimens is an essential problem in precision medicine. It is challenging because the actual mechanisms of treatment assignment and effects are unknown in observational studies. We propose a multivariate generalized linear mixed-effects model and a Bayesian g-computation algorithm to calculate the posterior distribution of subgroup-specific intervention benefits of dynamic treatment regimes. Unmeasured time-invariant factors are included as subject-specific random effects in the assumed joint distribution of outcomes, time-varying confounders, and treatment assignments. We identify a sequential ignorability assumption conditional on treatment assignment heterogeneity, that is, analogous to balancing the latent treatment preference due to unmeasured time-invariant factors. We present a simulation study to assess the proposed method's performance. The method is applied to observational clinical data to investigate the efficacy of continuously using mycophenolate in different subgroups of scleroderma patients.
对不同治疗方案的因果效应进行动态预测是精准医学的一个基本问题。由于在观察性研究中,治疗分配和效果的实际机制尚不清楚,因此这项工作极具挑战性。我们提出了一种多变量广义线性混合效应模型和贝叶斯 g 计算算法,用于计算动态治疗方案的亚组特异性干预效益的后验分布。在假定的结果、时变混杂因素和治疗分配的联合分布中,未测量的时变因素作为特定受试者的随机效应被包含在内。我们确定了一个以治疗分配异质性为条件的连续无知假设,即类似于平衡未测量时变因素导致的潜在治疗偏好。我们通过模拟研究来评估所提出方法的性能。我们将该方法应用于观察性临床数据,以研究在硬皮病患者的不同亚组中持续使用霉酚酸酯的疗效。
期刊介绍:
The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.