Department of Statistics, University of Florida, Gainesville, Florida, USA.
Edwards Lifesciences, Irvine, California, USA.
Biometrics. 2023 Dec;79(4):3907-3915. doi: 10.1111/biom.13894. Epub 2023 Jun 22.
In longitudinal studies, it is not uncommon to make multiple attempts to collect a measurement after baseline. Recording whether these attempts are successful provides useful information for the purposes of assessing missing data assumptions. This is because measurements from subjects who provide the data after numerous failed attempts may differ from those who provide the measurement after fewer attempts. Previous models for these designs were parametric and/or did not allow sensitivity analysis. For the former, there are always concerns about model misspecification and for the latter, sensitivity analysis is essential when conducting inference in the presence of missing data. Here, we propose a new approach which minimizes issues with model misspecification by using Bayesian nonparametrics for the observed data distribution. We also introduce a novel approach for identification and sensitivity analysis. We re-analyze the repeated attempts data from a clinical trial involving patients with severe mental illness and conduct simulations to better understand the properties of our approach.
在纵向研究中,在基线后多次尝试收集测量值并不罕见。记录这些尝试是否成功,对于评估缺失数据假设提供了有用的信息。这是因为在多次尝试失败后提供数据的受试者的测量值可能与在较少尝试后提供测量值的受试者的测量值不同。以前针对这些设计的模型是参数的,或者不允许进行敏感性分析。对于前者,总是存在对模型误设的担忧,对于后者,在存在缺失数据时进行推断时,敏感性分析是必不可少的。在这里,我们提出了一种新方法,通过对观测数据分布使用贝叶斯非参数化来最小化模型误设的问题。我们还引入了一种新的识别和敏感性分析方法。我们重新分析了一项涉及严重精神疾病患者的临床试验的重复尝试数据,并进行了模拟以更好地了解我们方法的特性。