Hematopoietic Stem Cell Transplantation in Late-Onset Krabbe Disease: No Evidence of Worsening Demyelination and Axonal Loss 4 Years Post-allograft

Journal of Neuroimaging : Official Journal of the American Society of Neuroimaging
Cornelia LauleSandra M Sirrs

Abstract

Late-onset adult Krabbe disease is a very rare demyelinating leukodystrophy, affecting less than 1 in a million people. Hematopoietic stem cell transplantation (HSCT) strategies can stop the accumulation of toxic metabolites that damage myelin-producing cells. We used quantitative advanced imaging metrics to longitudinally assess the impact of HSCT on brain abnormalities in adult-onset Krabbe disease. A 42-year-old female with late-onset Krabbe disease and an age/sex-matched healthy control underwent annual 3T MRI (baseline was immediately prior to HSCT for the Krabbe subject). Imaging included conventional scans, myelin water imaging, diffusion tensor imaging, and magnetic resonance spectroscopy. Brain abnormalities far beyond those visible on conventional imaging were detected, suggesting a global pathological process occurs in Krabbe disease with adult-onset etiology, with myelin being more affected than axons, and evidence of wide-spread gliosis. After HSCT, our patient showed clinical stability in all measures, as well as improvement in gait, dysarthria, and pseudobulbar affect at 7.5 years post-transplant. No MRI evidence of worsening demyelination and axonal loss was observed up to 4 years post-allograft. Clinical eviden...Continue Reading

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Citations

Feb 18, 2020·Journal of Neuroimaging : Official Journal of the American Society of Neuroimaging·Sarah R MorrisCornelia Laule
Jul 28, 2019·Journal of Neuroimaging : Official Journal of the American Society of Neuroimaging·Hanwen LiuCornelia Laule
Mar 18, 2020·Frontiers in Neuroscience·Frédéric DariosGiovanni Stevanin
Feb 18, 2020·The International Journal of Neuroscience·Xianghe MengCui Wang
Feb 27, 2021·Frontiers in Cellular Neuroscience·Stefanie PerrierGeneviève Bernard

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