Scharfstein Daniel, McDermott Aidan, Díaz Iván, Carone Marco, Lunardon Nicola, Turkoz Ibrahim
Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, U.S.A.
Department of Healthcare Policy and Research, Weill Cornell Medicine, New York, New York, U.S.A.
Biometrics. 2018 Mar;74(1):207-219. doi: 10.1111/biom.12729. Epub 2017 May 23.
In practice, both testable and untestable assumptions are generally required to draw inference about the mean outcome measured at the final scheduled visit in a repeated measures study with drop-out. Scharfstein et al. (2014) proposed a sensitivity analysis methodology to determine the robustness of conclusions within a class of untestable assumptions. In their approach, the untestable and testable assumptions were guaranteed to be compatible; their testable assumptions were based on a fully parametric model for the distribution of the observable data. While convenient, these parametric assumptions have proven especially restrictive in empirical research. Here, we relax their distributional assumptions and provide a more flexible, semi-parametric approach. We illustrate our proposal in the context of a randomized trial for evaluating a treatment of schizoaffective disorder.
在实际操作中,对于一项存在失访的重复测量研究,通常需要可检验和不可检验的假设来推断在最终预定访视时测量的平均结果。Scharfstein等人(2014年)提出了一种敏感性分析方法,以确定一类不可检验假设下结论的稳健性。在他们的方法中,不可检验和可检验的假设被保证是兼容的;他们的可检验假设基于观测数据分布的完全参数模型。虽然方便,但这些参数假设在实证研究中已被证明具有特别的局限性。在此,我们放宽了分布假设,并提供了一种更灵活的半参数方法。我们在一项评估分裂情感性障碍治疗方法的随机试验背景下阐述我们的提议。