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The proposed method converts the challenging task of hypothesis testing for longitudinal trajectories into a more manageable equivalent form based on hypothesis testing for spline coefficients. More importantly, by leveraging posterior inference with natural uncertainty quantification, our Bayesian method offers a more robust and straightforward hypothesis testing procedure than frequentist methods. Extensive simulations demonstrate BayTetra's superior performance over alternatives. Applications to the Biomarkers of Cognitive Decline Among Normal Individuals (BIOCARD) study yield interpretable and valuable clinical insights. 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引用次数: 0
摘要
在许多生物医学应用中,测试不同群体之间的纵向轨迹差异是一项重要任务。受阿尔茨海默病应用的启发,我们开发了一种创新的贝叶斯半参数方法--BayTetra,用于估计和测试多变量纵向轨迹的群体差异。BayTetra 通过直接考虑不同反应之间的相关性,对多变量纵向数据进行联合建模,并使用基于 B 样条的半参数框架,以极大的灵活性捕捉非线性轨迹。为了避免过度拟合,BayTetra 通过对样条函数的平滑度施加惩罚,鼓励进行简洁的轨迹估计。所提出的方法将具有挑战性的纵向轨迹假设检验任务转换成了基于样条系数假设检验的更易于管理的等效形式。更重要的是,通过利用具有自然不确定性量化的后验推断,我们的贝叶斯方法提供了比频数法更稳健、更直接的假设检验程序。大量的模拟证明了 BayTetra 优于其他方法的性能。在 "正常人认知能力下降的生物标志物"(BIOCARD)研究中的应用产生了可解释的、有价值的临床见解。本文的一大贡献是我们开发了一个 R 软件包 BayTetra,它实现了所提出的贝叶斯半参数方法,是第一个基于灵活建模框架的轨迹差异假设检验公开软件。
BayTetra: A Bayesian Semiparametric Approach for Testing Trajectory Differences.
Testing differences in longitudinal trajectories among distinct groups of population is an important task in many biomedical applications. Motivated by an application in Alzheimer's disease, we develop BayTetra, an innovative Bayesian semiparametric approach for estimating and testing group differences in multivariate longitudinal trajectories. BayTetra jointly models multivariate longitudinal data by directly accounting for correlations among different responses, and uses a semiparametric framework based on B-splines to capture the non-linear trajectories with great flexibility. To avoid overfitting, BayTetra encourages parsimonious trajectory estimation by imposing penalties on the smoothness of the spline functions. The proposed method converts the challenging task of hypothesis testing for longitudinal trajectories into a more manageable equivalent form based on hypothesis testing for spline coefficients. More importantly, by leveraging posterior inference with natural uncertainty quantification, our Bayesian method offers a more robust and straightforward hypothesis testing procedure than frequentist methods. Extensive simulations demonstrate BayTetra's superior performance over alternatives. Applications to the Biomarkers of Cognitive Decline Among Normal Individuals (BIOCARD) study yield interpretable and valuable clinical insights. A major contribution of this paper is that we have developed an R package BayTetra, which implements the proposed Bayesian semiparametric approach and is the first publicly available software for hypothesis testing on trajectory differences based on a flexible modeling framework.
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.