MedElevator
Войти в исследования Movement disorders : official journal of the Movement Disorder Society · 2026
Automated Magnetic Resonance Volumetry to Measure Multiple System Atrophy Disease Progression in Clinical Trials.
СтатьяНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Quattrone Andrea, Wilkens Ida, Zhu Zeyu, Qureshi Irfan A, Franzmeier Nicolai, Höglinger Günter U, M‐STAR Study Group
Аннотация
BACKGROUND: Recent studies have reported longitudinal brain atrophy in multiple system atrophy (MSA) in relatively small patient cohorts.
OBJECTIVE: To compare longitudinal regional brain volume and clinical changes as endpoint measures for MSA clinical trials.
METHODS: MSA patients with 1-year longitudinal brain magnetic resonance imaging (MRI) were selected from the placebo arm of the M-STAR Study (NCT03952806) and stratified into MSA-cerebellar (MSA-C) and MSA-parkinsonism (MSA-P) subtypes. A multisite healthy control (HC) cohort from international datasets was included for comparison. Baseline and longitudinal atrophy were assessed through automated MRI volumetry. Standardized longitudinal effect size values with 95% confidence intervals (CIs) and sample sizes required to detect treatment effect were estimated for 42 individual regional brain volumes, four combined volumetric indexes, nine clinical scales, and serum neurofilament light chain.
RESULTS: The final study cohort included 129 MSA patients (76 MSA-C, 53 MSA-P) and 184 HC. The most atrophic structures at baseline were infratentorial regions in MSA-C and putamen in MSA-P. In both subtypes, infratentorial structures showed the largest longitudinal volume loss compared with HC. The annualized change of a cerebellum-pons-putamen volumetric index was the most powerful progression biomarker in MSA patients (effect size: -1.81 [95% CI: -2.14, -1.55]) and in both subtypes (MSA-C: -2.04 [95% CI: -2.58, -1.68], MSA-P: -1.55 [95% CI: -2.08, -1.22]), requiring the lowest sample size per arm (MSA, N = 55 to detect 30% treatment effect), and outperforming clinical measures (-59.6% patients required per arm).
CONCLUSION: These findings support longitudinal MRI-derived regional atrophy rates as sensitive imaging endpoints to assess treatment efficacy in MSA clinical trials. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
OBJECTIVE: To compare longitudinal regional brain volume and clinical changes as endpoint measures for MSA clinical trials.
METHODS: MSA patients with 1-year longitudinal brain magnetic resonance imaging (MRI) were selected from the placebo arm of the M-STAR Study (NCT03952806) and stratified into MSA-cerebellar (MSA-C) and MSA-parkinsonism (MSA-P) subtypes. A multisite healthy control (HC) cohort from international datasets was included for comparison. Baseline and longitudinal atrophy were assessed through automated MRI volumetry. Standardized longitudinal effect size values with 95% confidence intervals (CIs) and sample sizes required to detect treatment effect were estimated for 42 individual regional brain volumes, four combined volumetric indexes, nine clinical scales, and serum neurofilament light chain.
RESULTS: The final study cohort included 129 MSA patients (76 MSA-C, 53 MSA-P) and 184 HC. The most atrophic structures at baseline were infratentorial regions in MSA-C and putamen in MSA-P. In both subtypes, infratentorial structures showed the largest longitudinal volume loss compared with HC. The annualized change of a cerebellum-pons-putamen volumetric index was the most powerful progression biomarker in MSA patients (effect size: -1.81 [95% CI: -2.14, -1.55]) and in both subtypes (MSA-C: -2.04 [95% CI: -2.58, -1.68], MSA-P: -1.55 [95% CI: -2.08, -1.22]), requiring the lowest sample size per arm (MSA, N = 55 to detect 30% treatment effect), and outperforming clinical measures (-59.6% patients required per arm).
CONCLUSION: These findings support longitudinal MRI-derived regional atrophy rates as sensitive imaging endpoints to assess treatment efficacy in MSA clinical trials. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.