{"title":"A General Framework for the Multiple Nonparametric Behrens-Fisher Problem With Dependent Replicates.","authors":"Erin Sprünken, Robert Mertens, Frank Konietschke","doi":"10.1002/sim.10262","DOIUrl":null,"url":null,"abstract":"<p><p>In many trials and experiments, subjects are not only observed once but multiple times, resulting in a cluster of possibly correlated observations (e.g., brain regions per patient). Observations often do not fulfill model assumptions of mixed models and require the use of nonparametric methods. In this article, we develop and present a purely nonparametric rank-based procedure that flexibly allows the unbiased and consistent estimation of the Wilcoxon-Mann-Whitney effect <math> <semantics><mrow><mi>P</mi> <mo>(</mo> <mi>X</mi> <mo><</mo> <mi>Y</mi> <mo>)</mo> <mo>+</mo> <mfrac><mrow><mn>1</mn></mrow> <mrow><mn>2</mn></mrow> </mfrac> <mi>P</mi> <mo>(</mo> <mi>X</mi> <mo>=</mo> <mi>Y</mi> <mo>)</mo></mrow> <annotation>$$ P\\left(X<Y\\right)+\\frac{1}{2}P\\left(X=Y\\right) $$</annotation></semantics> </math> in clustered data designs. Compared with existing methods, we allow flexible weights to be used in effect estimation. Additionally, we develop global and multiple contrast test procedures to test null hypotheses formulated regarding the generalized Mann-Whitney effects and for the computation of range-preserving simultaneous confidence intervals in a unified way. Extensive simulation studies show that these methods control the type-I error rate well and have reasonable power to detect alternatives in various situations.</p>","PeriodicalId":21879,"journal":{"name":"Statistics in Medicine","volume":" ","pages":"5650-5666"},"PeriodicalIF":1.8000,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11639657/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistics in Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/sim.10262","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/11/11 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"MATHEMATICAL & COMPUTATIONAL BIOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
In many trials and experiments, subjects are not only observed once but multiple times, resulting in a cluster of possibly correlated observations (e.g., brain regions per patient). Observations often do not fulfill model assumptions of mixed models and require the use of nonparametric methods. In this article, we develop and present a purely nonparametric rank-based procedure that flexibly allows the unbiased and consistent estimation of the Wilcoxon-Mann-Whitney effect in clustered data designs. Compared with existing methods, we allow flexible weights to be used in effect estimation. Additionally, we develop global and multiple contrast test procedures to test null hypotheses formulated regarding the generalized Mann-Whitney effects and for the computation of range-preserving simultaneous confidence intervals in a unified way. Extensive simulation studies show that these methods control the type-I error rate well and have reasonable power to detect alternatives in various situations.
依赖复制的多重非参数 Behrens-Fisher 问题的一般框架》(A General Framework for the Multiple Nonparametric Behrens-Fisher Problem With Dependent Replicates.
在许多试验和实验中,受试者不仅会被观察一次,还会被观察多次,从而产生一组可能相关的观察结果(例如,每个患者的脑区)。观察结果往往不符合混合模型的模型假设,因此需要使用非参数方法。在本文中,我们开发并提出了一种纯粹的基于秩的非参数程序,可以在聚类数据设计中灵活地对 Wilcoxon-Mann-Whitney 效应 P ( X Y ) + 1 2 P ( X = Y ) $$ P\left(X 进行无偏且一致的估计。与现有方法相比,我们允许在效应估计中使用灵活的权重。此外,我们还开发了全局和多重对比检验程序,用于检验广义曼-惠特尼效应的零假设,并以统一的方式计算保留范围的同步置信区间。大量的模拟研究表明,这些方法能很好地控制 I 类错误率,并在各种情况下具有合理的检测能力。
期刊介绍:
The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.