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Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models

BACKGROUND: Although nontremor and tremor Part 3 Movement Disorder Society–Unified Parkinson's Disease Rating Scale items measure different impairment domains, their distinct progression and drug responsivity remain unstudied longitudinally. The total score may obscure important time‐based and...

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Autores principales: Luo, Sheng, Zou, Haotian, Goetz, Christopher G., Choi, Dongrak, Oakes, David, Simuni, Tanya, Stebbins, Glenn T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8485609/
https://www.ncbi.nlm.nih.gov/pubmed/34631944
http://dx.doi.org/10.1002/mdc3.13311
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author Luo, Sheng
Zou, Haotian
Goetz, Christopher G.
Choi, Dongrak
Oakes, David
Simuni, Tanya
Stebbins, Glenn T.
author_facet Luo, Sheng
Zou, Haotian
Goetz, Christopher G.
Choi, Dongrak
Oakes, David
Simuni, Tanya
Stebbins, Glenn T.
author_sort Luo, Sheng
collection PubMed
description BACKGROUND: Although nontremor and tremor Part 3 Movement Disorder Society–Unified Parkinson's Disease Rating Scale items measure different impairment domains, their distinct progression and drug responsivity remain unstudied longitudinally. The total score may obscure important time‐based and treatment‐based changes occurring in the individual domains. OBJECTIVE: Using the unique advantages of item response theory (IRT), we developed novel longitudinal unidimensional and multidimensional models to investigate nontremor and tremor changes occurring in an interventional Parkinson's disease (PD) study. METHOD: With unidimensional longitudinal IRT, we assessed the 33 Part 3 item data (22 nontremor and 10 tremor items) of 336 patients with early PD from the STEADY‐PD III (Safety, Tolerability, and Efficacy Assessment of Isradipine for PD, placebo vs. isradipine) study. With multidimensional longitudinal IRT, we assessed the progression rates over time and treatment (in overall motor severity, nontremor, and tremor domains) using Markov Chain Monte Carlo implemented in Stan. RESULTS: Regardless of treatment, patients showed significant but different time‐based deterioration rates for total motor, nontremor, and tremor scores. Isradipine was associated with additional significant deterioration over placebo in total score and nontremor scores, but not in tremor score. Further highlighting the 2 separate latent domains, nontremor and tremor severity changes were positively but weakly correlated (correlation coefficient, 0.108). CONCLUSIONS: Longitudinal IRT analysis is a novel statistical method highly applicable to PD clinical trials. It addresses limitations of traditional linear regression approaches and previous IRT investigations that either applied cross‐sectional IRT models to longitudinal data or failed to estimate all parameters simultaneously. It is particularly useful because it can separate nontremor and tremor changes both over time and in response to treatment interventions.
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spelling pubmed-84856092021-10-07 Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models Luo, Sheng Zou, Haotian Goetz, Christopher G. Choi, Dongrak Oakes, David Simuni, Tanya Stebbins, Glenn T. Mov Disord Clin Pract Research Articles BACKGROUND: Although nontremor and tremor Part 3 Movement Disorder Society–Unified Parkinson's Disease Rating Scale items measure different impairment domains, their distinct progression and drug responsivity remain unstudied longitudinally. The total score may obscure important time‐based and treatment‐based changes occurring in the individual domains. OBJECTIVE: Using the unique advantages of item response theory (IRT), we developed novel longitudinal unidimensional and multidimensional models to investigate nontremor and tremor changes occurring in an interventional Parkinson's disease (PD) study. METHOD: With unidimensional longitudinal IRT, we assessed the 33 Part 3 item data (22 nontremor and 10 tremor items) of 336 patients with early PD from the STEADY‐PD III (Safety, Tolerability, and Efficacy Assessment of Isradipine for PD, placebo vs. isradipine) study. With multidimensional longitudinal IRT, we assessed the progression rates over time and treatment (in overall motor severity, nontremor, and tremor domains) using Markov Chain Monte Carlo implemented in Stan. RESULTS: Regardless of treatment, patients showed significant but different time‐based deterioration rates for total motor, nontremor, and tremor scores. Isradipine was associated with additional significant deterioration over placebo in total score and nontremor scores, but not in tremor score. Further highlighting the 2 separate latent domains, nontremor and tremor severity changes were positively but weakly correlated (correlation coefficient, 0.108). CONCLUSIONS: Longitudinal IRT analysis is a novel statistical method highly applicable to PD clinical trials. It addresses limitations of traditional linear regression approaches and previous IRT investigations that either applied cross‐sectional IRT models to longitudinal data or failed to estimate all parameters simultaneously. It is particularly useful because it can separate nontremor and tremor changes both over time and in response to treatment interventions. John Wiley & Sons, Inc. 2021-08-03 /pmc/articles/PMC8485609/ /pubmed/34631944 http://dx.doi.org/10.1002/mdc3.13311 Text en © 2021 The Authors. Movement Disorders Clinical Practice published by Wiley Periodicals LLC. on behalf of International Parkinson and Movement Disorder Society. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Research Articles
Luo, Sheng
Zou, Haotian
Goetz, Christopher G.
Choi, Dongrak
Oakes, David
Simuni, Tanya
Stebbins, Glenn T.
Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title_full Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title_fullStr Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title_full_unstemmed Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title_short Novel Approach to Movement Disorder Society–Unified Parkinson's Disease Rating Scale Monitoring in Clinical Trials: Longitudinal Item Response Theory Models
title_sort novel approach to movement disorder society–unified parkinson's disease rating scale monitoring in clinical trials: longitudinal item response theory models
topic Research Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8485609/
https://www.ncbi.nlm.nih.gov/pubmed/34631944
http://dx.doi.org/10.1002/mdc3.13311
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