Lachin John M
The Biostatistics Center, The George Washington University, Rockville, Maryland, United States of America.
PLoS One. 2014 Oct 17;9(10):e108784. doi: 10.1371/journal.pone.0108784. eCollection 2014.
Many studies aim to assess whether a therapy has a beneficial effect on multiple outcomes simultaneously relative to a control. Often the joint null hypothesis of no difference for the set of outcomes is tested using separate tests with a correction for multiple tests, or using a multivariate T2-like MANOVA or global test. However, a more powerful test in this case is a multivariate one-sided or one-directional test directed at detecting a simultaneous beneficial treatment effect on each outcome, though not necessarily of the same magnitude. The Wei-Lachin test is a simple 1 df test obtained from a simple sum of the component statistics that was originally described in the context of a multivariate rank analysis. Under mild conditions this test provides a maximin efficient test of the null hypothesis of no difference between treatment groups for all outcomes versus the alternative hypothesis that the experimental treatment is better than control for some or all of the component outcomes, and not worse for any. Herein applications are described to a simultaneous test for multiple differences in means, proportions or life-times, and combinations thereof, all on potentially different scales. The evaluation of sample size and power for such analyses is also described. For a test of means of two outcomes with a common unit variance and correlation 0.5, the sample size needed to provide 90% power for two separate one-sided tests at the 0.025 level is 64% greater than that needed for the single Wei-Lachin multivariate one-directional test at the 0.05 level. Thus, a Wei-Lachin test with these operating characteristics is 39% more efficient than two separate tests. Likewise, compared to a T2-like omnibus test on 2 df, the Wei-Lachin test is 32% more efficient. An example is provided in which the Wei-Lachin test of multiple components has superior power to a test of a composite outcome.
许多研究旨在评估一种疗法相对于对照是否能同时对多个结局产生有益效果。通常,针对这组结局无差异的联合零假设,会使用经过多重检验校正的单独检验,或者使用类似多元T2的多变量方差分析(MANOVA)或全局检验来进行检验。然而,在这种情况下,一个更强大的检验是多变量单侧或单向检验,旨在检测对每个结局同时产生有益的治疗效果,尽管效果大小不一定相同。魏-拉钦检验是一种自由度为1的简单检验,它由各分量统计量的简单求和得到,最初是在多元秩分析的背景下描述的。在温和条件下,该检验为零假设提供了一种极大极小有效检验,即治疗组在所有结局上无差异,对立假设是实验性治疗在某些或所有分量结局上优于对照,且在任何结局上都不差。本文描述了该检验在均值、比例或寿命的多个差异同时检验中的应用,以及它们的组合应用,所有这些都可能在不同的尺度上。还描述了此类分析的样本量和检验效能评估。对于两个结局均值的检验,其共同单位方差为1且相关性为0.5,在0.025水平下为两个单独的单侧检验提供90%检验效能所需的样本量,比在0.05水平下进行单个魏-拉钦多变量单向检验所需的样本量大64%。因此,具有这些操作特性的魏-拉钦检验比两个单独检验的效率高39%。同样,与自由度为2的类似T2的综合检验相比,魏-拉钦检验的效率高32%。文中给出了一个例子,其中多个分量的魏-拉钦检验比复合结局检验具有更高的检验效能。