Methodology for evaluating a partially controlled longitudinal treatment using principal stratification, with application to a needle exchange program

• 成果类型: Article
• 署名作者: Frangakis, CE; Brookmeyer, RS; Varadhan, R; Safaeian, M; Vlahov, D; Strathdee, SA
• 署名单位: Johns Hopkins University; Johns Hopkins Bloomberg School of Public Health; Johns Hopkins University; Johns Hopkins Bloomberg School of Public Health; New York Academy of Medicine
• 刊物名称: JOURNAL OF THE AMERICAN STATISTICAL ASSOCIATION
• ISSN/ISSBN: 0162-1459
• DOI: 10.1198/016214504000000232
• 发表日期: 2004
• 页码: 239-249
• 关键词: time-varying exposure; intention-to-treat; bayesian-inference; randomized experiments; causal; mortality; models; noncompliance; infection; survival

摘要

We consider studies for evaluating the short-term effect of a treatment of interest on a time-to-event outcome. The studies we consider are partially controlled in the following sense: (I) Subjects' exposure to the treatment of interest can vary over time, but this exposure is not directly controlled by the study; (2) subjects' follow-up time is not directly controlled by the study; and (3) the study directly controls another factor that can affect subjects' exposure to the treatment of interest as well as subjects' follow-up time. When factors (1) and (2) are both present in the study, evaluating the treatment of interest using standard methods, including instrumental variables, does not generally estimate treatment effects. We develop the methodology for estimating the effect of treatment in this setting of partially controlled studies under explicit assumptions using the framework for principal stratification for causal inference. We illustrate our methods by a study to evaluate the efficacy of the Baltimore Needle Exchange Program to reduce the risk of human immunodeficiency virus (HIV) transmission, using data on distance of the program's sites from the subjects.