Jo Booil, Wang Chen-Pin, Ialongo Nicholas S
Department of Psychiatry & Behavioral Sciences, Stanford University, Stanford, CA 94305-5795.
Stat Interface. 2009 Jan 1;2(4):403-412. doi: 10.4310/sii.2009.v2.n4.a2.
In longitudinal studies, outcome trajectories can provide important information about substantively and clinically meaningful underlying subpopulations who may also respond differently to treatments or interventions. Growth mixture analysis is an efficient way of identifying heterogeneous trajectory classes. However, given its exploratory nature, it is unclear how involvement of latent classes should be handled in the analysis when estimating causal treatment effects. In this paper, we propose a 2-step approach, where formulation of trajectory strata and identification of causal effects are separated. In Step 1, we stratify individuals in one of the assignment conditions (reference condition) into trajectory strata on the basis of growth mixture analysis. In Step 2, we estimate treatment effects for different trajectory strata, treating the stratum membership as partly known (known for individuals assigned to the reference condition and missing for the rest). The results can be interpreted as how subpopulations that differ in terms of outcome prognosis under one treatment condition would change their prognosis differently when exposed to another treatment condition. Causal effect estimation in Step 2 is consistent with that in the principal stratification approach (Frangakis and Rubin, 2002) in the sense that clarified identifying assumptions can be employed and therefore systematic sensitivity analyses are possible. Longitudinal development of attention deficit among children from the Johns Hopkins School Intervention Trial (Ialongo et al., 1999) will be presented as an example.
在纵向研究中,结果轨迹可以提供有关具有实质意义和临床意义的潜在亚群的重要信息,这些亚群对治疗或干预的反应可能也有所不同。生长混合分析是识别异质轨迹类别的有效方法。然而,鉴于其探索性本质,在估计因果治疗效果时,尚不清楚在分析中应如何处理潜在类别的参与情况。在本文中,我们提出了一种两步法,即将轨迹分层的制定和因果效应的识别分开。在第一步中,我们根据生长混合分析将处于一种分配条件(参考条件)下的个体分层为轨迹层。在第二步中,我们估计不同轨迹层的治疗效果,将层成员身份视为部分已知(对于分配到参考条件的个体已知,对于其余个体未知)。结果可以解释为,在一种治疗条件下,在结果预后方面存在差异的亚群在接触另一种治疗条件时,其预后将如何以不同方式变化。第二步中的因果效应估计与主要分层方法(Frangakis和Rubin,2002)中的估计一致,因为可以采用明确的识别假设,因此可以进行系统的敏感性分析。将以约翰霍普金斯学校干预试验(Ialongo等人,1999)中儿童注意力缺陷的纵向发展为例进行说明。