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不完全纵向数据无分布检验的经验功效及其在艾滋病临床试验中的应用

Empirical power for distribution-free tests of incomplete longitudinal data with applications to AIDS clinical trials.

作者信息

Kuhn A M, DeMasi R A

机构信息

Becton Dickinson Microbiology Systems, Sparks, Maryland 21152-0999, USA.

出版信息

J Biopharm Stat. 1999 Aug;9(3):401-16. doi: 10.1081/BIP-100101184.

Abstract

The design of AIDS clinical trials is of growing importance. These studies tend to be longitudinal and typically involve missing data. HIV-1 RNA is a common endpoint for these studies and is inherently non-normal, although viral load can be measured only within certain bounds, resulting in censored data. We compared several analysis methods, both univariate and multivariate, on the basis of empirical power and provide an illustrative example of data from a controlled clinical trial. Simulated viral load data demonstrate that methods adjusting for baseline data have power increasing with increasing positive intrasubject correlation expected with this type of data. Several summary measures considered have power compatible with multivariate tests.

摘要

艾滋病临床试验的设计愈发重要。这些研究往往具有纵向性,且通常涉及缺失数据。HIV-1 RNA是这些研究中常见的终点指标,其本质上不呈正态分布,尽管病毒载量只能在特定范围内测量,从而产生删失数据。我们基于经验检验效能比较了几种单变量和多变量分析方法,并给出了一项对照临床试验数据的示例。模拟的病毒载量数据表明,针对基线数据进行调整的方法,其检验效能会随着此类数据预期的受试者内正相关性增加而提高。所考虑的几种汇总测量指标的检验效能与多变量检验相当。

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