Longitudinal multi-modal muscle-based biomarker assessment in motor neuron disease.

ABSTRACT: BACKGROUND:Clinical phenotypic heterogeneity represents a major barrier to trials in motor neuron disease (MND) and objective surrogate outcome measures are required, especially for slowly progressive patients. We assessed responsiveness of clinical, electrophysiological and radiological muscle-based assessments to detect MND-related progression. MATERIALS AND METHODS:A prospective, longitudinal cohort study of 29 MND patients and 22 healthy controls was performed. Clinical measures, electrophysiological motor unit number index/size (MUNIX/MUSIX) and relative T2- and diffusion-weighted whole-body muscle magnetic resonance (MR) were assessed three times over 12 months. Multi-variable regression models assessed between-group differences, clinico-electrophysiological associations, and longitudinal changes. Standardized response means (SRMs) assessed sensitivity to change over 12 months. RESULTS:MND patients exhibited 18% higher whole-body mean muscle relative T2-signal than controls (95% CI 7-29%, p?

SUBMITTER: Jenkins TM 

PROVIDER: S-EPMC6954906 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature