Longitudinal diffusion MRI as surrogate outcome measure for myelopathy in adrenoleukodystrophy

Objective: To prospectively determine the potential of diffusion MRI (dMRI) of the cervical spinal cord and the corticospinal tracts in brain as surrogate outcome measure for progression of myelopathy in men with adrenoleukodystrophy, as better outcome measures to quantify progression of myelopathy would enable clinical trials with less patients and shorter follow-up.
Methods: Clinical assessment of myelopathy included Expanded Disability Status Scale (EDSS), Severity Scoring system for Progressive Myelopathy (SSPROM), timed up-and-go and 6-minute walk test. Applied dMRI metrics included fractional anisotropy, mean diffusivity, axial diffusivity and radial diffusivity.
Results: Data was available for 33 controls and 52 patients. First, cross-sectionally, differences between groups (controls vs. patients; controls vs. asymptomatic patients vs. symptomatic patients) were statistically significant for fractional anisotropy, mean diffusivity and radial diffusivity in spinal cord and brain...