Keywords: Rare Disease, Rare disease
Motivation: The emergence of demyelination, prediction of progression, appropriate disease-staging, and neurological prognosis post-treatment for cerebral adrenoleukodystrophy are significant challenges.
Goal(s): Quantify the spatial change in white matter microstructure across a lesion into distal normal appearing white matter to discriminate between disease progression vs arrest.
Approach: A region of interest analysis was performed along the corpus callosum from proximal to the lesion to the distal end in 36 DTI datasets. The slope of change in FA was calculated and compared between progressors vs non-progressors.
Results: There is a significant difference between the FA slope of the progressor group vs non-progressors.
Impact: This analytic approach demonstrated differences in change in microstructure between patients with ongoing disease vs those with disease arrest, indicating utility as a clinical imaging tool to guide imaging surveillance, prognosis, and appropriate patient selection for treatment in cerebral adrenoleukodystrophy.
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