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单调缺失纵向数据分析中的模式混合模型与选择模型

研究问题
纵向研究末期结局在部分受试者中缺失时,如何识别其总体分布参数,并考察结论对不可验证缺失假设的敏感性?
方法与思路
作者比较三种模式混合模型及其选择模型解释,再加入灵活的半参数限制,以构造适用于中等样本的估计量。
材料陈述的贡献
摘要称三种模型均不限制观测数据分布,只有一种参数化便于考察偏离序贯可忽略性的情形,并刻画相应估计量类别。
阅读关注点
应核对三种缺失假设的差异、识别与可行推断所需限制,以及敏感性参数的含义和临床试验应用中的设定。
阅读边界
仅依据所给 Crossref 摘要;缺失假设不可验证,摘要未列出具体条件,也不能据此判断中等样本表现或应用结论的稳健性。

原文摘要

Summary We examine three pattern–mixture models for making inference about parameters of the distribution of an outcome of interest Y that is to be measured at the end of a longitudinal study when this outcome is missing in some subjects. We show that these pattern–mixture models also have an interpretation as selection models. Because these models make unverifiable assumptions, we recommend that inference about the distribution of Y be repeated under a range of plausible assumptions. We argue that, of the three models considered, only one admits a parameterization that facilitates the examination of departures from the assumption of sequential ignorability. The three models are nonparametric in the sense that they do not impose restrictions on the class of observed data distributions. Owing to the curse of dimensionality, the assumptions that are encoded in these models are sufficient for identification but not for inference. We describe additional flexible and easily interpretable assumptions under which it is possible to construct estimators that are well behaved with moderate sample sizes. These assumptions define semiparametric models for the distribution of the observed data. We describe a class of estimators which, up to asymptotic equivalence, comprise all the consistent and asymptotically normal estimators of the parameters of interest under the postulated semiparametric models. We illustrate our methods with the analysis of data from a randomized clinical trial of contracepting women.

笔记与分组
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