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Abstract #1191

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

Cornelia Laule1,2, Elham Shahinfard1, Burkhard Maedler1, Jing Zhang1, Irene Vavasour1, Ritu Aul3, David K.B. Li1,4, Alex L. MacKay1,5, 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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