A New Algorithm for Sampling Parameters in a Structured Correlation Matrix With Application to Estimating Optimal Combinations of Muscles to Quantify Progression in Duchenne Muscular Dystrophy

The goal of this paper is to estimate an optimal combination of biomarkers for individuals with Duchenne muscular dystrophy (DMD), which provides the most sensitive combinations of biomarkers to assess disease progression (in this case, optimal with respect to standardized response mean (SRM) for 4 muscle biomarkers). The biomarker data is incomplete (missing and irregular) multivariate longitudinal data. We propose a normal model with structured covariance designed for our setting. To sample from the posterior distribution of parameters, we develop a Markov Chain Monte Carlo (MCMC) algorithm to address the positive definiteness constraint on the structured correlation matrix. In particular, we propose a novel approach to compute the support of the parameters in the structured correlation matrix; we modify the approach from [1] on the set of the largest possible submatrices of the correlation matrix, where the correlation parameter is a unique element. For each posterior sample, we compute the optimal weights of our construct. We conduct data analysis and simulation studies to evaluate the algorithm and the frequentist properties of the posteriors of correlations and weights. We found that the lower extremities are the most responsive muscles at the early and late ambulatory disease stages, and the biceps brachii is the most responsive at the nonambulatory disease stage.

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Publication Details

Journal
Statistics in Medicine
Published
2025-09-01
DOI
https://doi.org/10.1002/sim.70252
Primary Topic
Muscle Physiology and Disorders
Type
article
Field-Weighted Citation Impact
0.00

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article

A New Algorithm for Sampling Parameters in a Structured Correlation Matrix With Application to Estimating Optimal Combinations of Muscles to Quantify Progression in Duchenne Muscular Dystrophy

William D. Rooney, Michael K. Kim, Krista Vandenborne, Michael J. Daniels et al.
Statistics in Medicine
Muscle Physiology and Disorders
article

A New Algorithm for Sampling Parameters in a Structured Correlation Matrix With Application to Estimating Optimal Combinations of Muscles to Quantify Progression in Duchenne Muscular Dystrophy

William D. Rooney, Michael K. Kim, Krista Vandenborne, Michael J. Daniels, Glenn A. Walter, Rebecca J. Willcocks
article en

Abstract

The goal of this paper is to estimate an optimal combination of biomarkers for individuals with Duchenne muscular dystrophy (DMD), which provides the most sensitive combinations of biomarkers to assess disease progression (in this case, optimal with respect to standardized response mean (SRM) for 4 muscle biomarkers). The biomarker data is incomplete (missing and irregular) multivariate longitudinal data. We propose a normal model with structured covariance designed for our setting. To sample from the posterior distribution of parameters, we develop a Markov Chain Monte Carlo (MCMC) algorithm to address the positive definiteness constraint on the structured correlation matrix. In particular, we propose a novel approach to compute the support of the parameters in the structured correlation matrix; we modify the approach from [1] on the set of the largest possible submatrices of the correlation matrix, where the correlation parameter is a unique element. For each posterior sample, we compute the optimal weights of our construct. We conduct data analysis and simulation studies to evaluate the algorithm and the frequentist properties of the posteriors of correlations and weights. We found that the lower extremities are the most responsive muscles at the early and late ambulatory disease stages, and the biceps brachii is the most responsive at the nonambulatory disease stage.

Statistics in MedicineVol. 44(20-22)
Oregon Health & Science University (US), University of Florida (US), Advanced Imaging Research (United States) (US)
National Science Foundation, McKnight Foundation, Oregon Health and Science University, National High Magnetic Field Laboratory, National Institutes of Health, Division of Materials Research, High Magnetic Field Laboratory, Chinese Academy of Sciences
Openalex Percentile: Top 79%
Muscle Physiology and Disorders
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