Power and Sample Size Calculation for Multivariate Longitudinal Trials Using the Longitudinal Rank Sum Test

Neurodegenerative diseases such as Alzheimer's and Parkinson's often exhibit complex, multivariate longitudinal outcomes that require advanced statistical methods to comprehensively evaluate treatment efficacy. The Longitudinal Rank Sum Test (LRST) offers a nonparametric framework to assess global treatment effects across multiple longitudinal endpoints without requiring multiplicity corrections. This study develops a robust methodology for power and sample size estimation specific to the LRST, integrating theoretical derivations, asymptotic properties, and practical estimation techniques under large sample conditions. Validation through numerical simulations demonstrates the accuracy of the proposed methods, while real-world applications to clinical trials in Alzheimer's disease (AD) and Parkinson's disease (PD) highlight their practical significance. This framework facilitates the design of efficient, well-powered trials, advancing the evaluation of treatments for complex diseases with multivariate longitudinal outcomes.

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Publication Details

Journal
Statistics in Medicine
Published
2025-09-01
DOI
https://doi.org/10.1002/sim.70261
Citations
1
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
2.63
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article

Power and Sample Size Calculation for Multivariate Longitudinal Trials Using the Longitudinal Rank Sum Test

Sheng Luo, Xiaoming Xu, Dhrubajyoti Ghosh
1 citations
Statistics in Medicine
Statistical Methods in Clinical Trials
2.63
article

Power and Sample Size Calculation for Multivariate Longitudinal Trials Using the Longitudinal Rank Sum Test

Sheng Luo, Xiaoming Xu, Dhrubajyoti Ghosh
article en
1 citations

Abstract

Neurodegenerative diseases such as Alzheimer's and Parkinson's often exhibit complex, multivariate longitudinal outcomes that require advanced statistical methods to comprehensively evaluate treatment efficacy. The Longitudinal Rank Sum Test (LRST) offers a nonparametric framework to assess global treatment effects across multiple longitudinal endpoints without requiring multiplicity corrections. This study develops a robust methodology for power and sample size estimation specific to the LRST, integrating theoretical derivations, asymptotic properties, and practical estimation techniques under large sample conditions. Validation through numerical simulations demonstrates the accuracy of the proposed methods, while real-world applications to clinical trials in Alzheimer's disease (AD) and Parkinson's disease (PD) highlight their practical significance. This framework facilitates the design of efficient, well-powered trials, advancing the evaluation of treatments for complex diseases with multivariate longitudinal outcomes.

Statistics in MedicineVol. 44(20-22)
Duke University (US)
Openalex Percentile: Top 9%
Statistical Methods in Clinical Trials
2.63
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