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Updated: Jan 4, 2026
Diffusion Tensor Magnetic Resonance Imaging in the Analysis of Neurodegenerative Diseases
Published on: July 28, 2013
Longitudinal diffusion MRI as surrogate outcome measure for myelopathy in adrenoleukodystrophy
Irene C Huffnagel1, Wouter J C van Ballegoij1, Johanna M B W Vos1
1From the Department of Paediatric Neurology, Emma Children's Hospital (I.C.H., W.J.C.v.B., J.M.B.W.V., M.E.), Laboratory Genetic Metabolic Diseases (S.K.), and Department of Biomedical Engineering & Physics (M.W.A.C.), Amsterdam UMC, University of Amsterdam, the Netherlands.
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 fewer 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 were 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 corticospinal tracts (effect size 0.31-0.68). Correlations between dMRI metrics and clinical measures were moderate to strong (correlation coefficient 0.35-0.60). Second, longitudinally (n = 36), change on clinical measures was significant after 2-year follow-up for EDSS, SSPROM, and Timed Up-and-Go (p ≤ 0.021, effect size ≤0.14). Change on brain fractional anisotropy and radial diffusivity was slightly larger (p ≤ 0.002, effect sizes 0.16-0.28). In addition, a statistically significant change was detectable in asymptomatic patients using brain dMRI and not using the clinical measures. Change on clinical measures did not correlate to change on dMRI metrics.
Conclusion:
Although effect sizes were small, our prospective data illustrate the potential of dMRI as surrogate outcome measure for progression of myelopathy in men with adrenoleukodystrophy.
