使用多重边际模型进行同步推理。

IF 1.3 4区 医学 Q4 PHARMACOLOGY & PHARMACY Pharmaceutical Statistics Pub Date : 2024-08-21 DOI:10.1002/pst.2428
Ludwig A Hothorn, Christian Ritz, Frank Schaarschmidt, Signe M Jensen, Robin Ristl
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引用次数: 0

摘要

本教程介绍了单步低维同步推理,重点是调整后 p 值和兼容置信区间的可用性,而不仅仅是通常的均值比较。其基本思想是:首先,利用相关性对多元 t 分布的量值的影响:越高越不保守。此外,第二,使用多重边际模型方法(mmm),使用线性到广义线性混合模型类中的多重模型来估算相关矩阵的可估算性。使用选定的 R 软件包,通过几个真实数据场景讨论了使用 mmm 的基本 maxT 检验。令人惊讶的是,其中突出了不同的特点:(i) 分析不同尺度、相关的多个终点,(ii) 分析多个相关的二进制终点,(iii) 将剂量作为定性因子和/或定量协变量建模,(iv) 在 poly-k 趋势检验中联合考虑多个调整参数,(v) 联合检验剂量和时间、(viii) 多重线性混合效应模型;(ix) 广义估计方程;以及 (x) 非线性回归模型。
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Simultaneous Inference Using Multiple Marginal Models.

This tutorial describes single-step low-dimensional simultaneous inference with a focus on the availability of adjusted p values and compatible confidence intervals for more than just the usual mean value comparisons. The basic idea is, first, to use the influence of correlation on the quantile of the multivariate t-distribution: the higher the less conservative. In addition, second, the estimability of the correlation matrix using the multiple marginal models approach (mmm) using multiple models in the class of linear up to generalized linear mixed models. The underlying maxT-test using mmm is discussed by means of several real data scenarios using selected R packages. Surprisingly, different features are highlighted, among them: (i) analyzing different-scaled, correlated, multiple endpoints, (ii) analyzing multiple correlated binary endpoints, (iii) modeling dose as qualitative factor and/or quantitative covariate, (iv) joint consideration of several tuning parameters within the poly-k trend test, (v) joint testing of dose and time, (vi) considering several effect sizes, (vii) joint testing of subgroups and overall population in multiarm randomized clinical trials with correlated primary endpoints, (viii) multiple linear mixed effect models, (ix) generalized estimating equations, and (x) nonlinear regression models.

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来源期刊
Pharmaceutical Statistics
Pharmaceutical Statistics 医学-统计学与概率论
CiteScore
2.70
自引率
6.70%
发文量
90
审稿时长
6-12 weeks
期刊介绍: Pharmaceutical Statistics is an industry-led initiative, tackling real problems in statistical applications. The Journal publishes papers that share experiences in the practical application of statistics within the pharmaceutical industry. It covers all aspects of pharmaceutical statistical applications from discovery, through pre-clinical development, clinical development, post-marketing surveillance, consumer health, production, epidemiology, and health economics. The Journal is both international and multidisciplinary. It includes high quality practical papers, case studies and review papers.
期刊最新文献
Bayesian Solutions for Assessing Differential Effects in Biomarker Positive and Negative Subgroups. Pre-Posterior Distributions in Drug Development and Their Properties. Beyond the Fragility Index. A Model-Based Trial Design With a Randomization Scheme Considering Pharmacokinetics Exposure for Dose Optimization in Oncology. Potential Bias Models With Bayesian Shrinkage Priors for Dynamic Borrowing of Multiple Historical Control Data.
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