Vexler Albert, Yu Jihnhee, Tian Lili, Liu Shuling
Department of Biostatistics, University at Buffalo, State University of New York, USA.
Biom J. 2010 Jun;52(3):348-61. doi: 10.1002/bimj.200900131.
The clinical pulmonary infection score (CPIS) and bronchoalveolar lavage (BAL) are important diagnostic variables of pneumonia for forcefully ventilated patients who are susceptible to nosocomial infection. Because of its invasive nature, BAL is performed for patients only if the CPIS is greater than a certain threshold value. Thus, CPIS and BAL are closely related, yet BAL values are substantially missing. In a randomized clinical trial, the control and oral treatment groups were compared based on the outcomes from these procedures. Because of the relevance of both outcomes with respect to evaluating the efficacy of treatments, we propose and examine a nonparametric test based on these outcomes, which employs the empirical likelihood methodology. While efficient parametric methods are available when data are observed incompletely, performing appropriate goodness-of-fit tests to justify the parametric assumptions is difficult. Our motivation is to provide an approach based on no particular distributional assumption, which enables us to use all observed bivariate data, whether completed or not in an approximate likelihood manner. A broad Monte Carlo study evaluates the asymptotic properties and efficiency of the proposed method based on various sample sizes and underlying distributions. The proposed technique is applied to a data set from a pneumonia study demonstrating its practical worth.
临床肺部感染评分(CPIS)和支气管肺泡灌洗(BAL)是易发生医院感染的机械通气患者肺炎的重要诊断变量。由于BAL具有侵入性,只有当CPIS大于某个阈值时才对患者进行BAL检查。因此,CPIS和BAL密切相关,但BAL值大量缺失。在一项随机临床试验中,根据这些检查的结果对对照组和口服治疗组进行了比较。由于这两个结果对于评估治疗效果都具有相关性,我们提出并检验了一种基于这些结果的非参数检验方法,该方法采用经验似然方法。虽然在数据不完全观测时可以使用有效的参数方法,但进行适当的拟合优度检验以证明参数假设是困难的。我们的动机是提供一种基于无特定分布假设的方法,该方法使我们能够以近似似然的方式使用所有观测到的双变量数据,无论其是否完整。一项广泛的蒙特卡罗研究基于各种样本量和基础分布评估了所提出方法的渐近性质和效率。所提出的技术应用于一项肺炎研究的数据集中,证明了其实际价值。