Abstract #1191

Hematopoietic stem cell transplantation in late-onset Krabbe disease halts demyelination and axonal loss: A 4 year longitudinal case study

Cornelia Laule^1,2, Elham Shahinfard¹, Burkhard Maedler¹, Jing Zhang¹, Irene Vavasour¹, Ritu Aul³, David K.B. Li^1,4, Alex L. MacKay^1,5, and Sandra Sirrs⁶

¹Radiology, University of British Columbia, Vancouver, BC, Canada, ²Pathology & Laboratory Medicine, University of British Columbia, Vancouver, BC, Canada, ³Medical Genetics, North York General Hospital, Toronto, ON, Canada, ⁴Medicine (Neurology), University of British Columbia, Vancouver, BC, Canada, ⁵Physics & Astronomy, University of British Columbia, Vancouver, BC, Canada, ⁶Medicine (Endocrinology), University of British Columbia, Vancouver, BC, Canada

Late-onset Krabbe disease is a very rare demyelinating leukodystrophy. We found hematopoietic stem cell transplantation in Krabbe disease halts demyelination and axonal loss up to 4 years post-allograft. 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. However, the degree of Krabbe abnormality did not increase over time for any advanced MR metrics, which supports hematopoietic stem cell transplantation as an effective treatment strategy for stopping progression associated with late-onset Krabbe disease.

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