Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy
Authors:
- Martje E. van EgmondMEMartje E. van Egmond
- Petra J. W. PouwelsPPPetra J. W. Pouwels
- Jaap-Jan BoelensJBJaap-Jan Boelens
- Caroline A. LindemansCLCaroline A. Lindemans
- Frederik BarkhofFBFrederik Barkhof
- Martijn D. SteenwijkMSMartijn D. Steenwijk
- Peter M. van HasseltPHPeter M. van Hasselt
- Marjo S. van der KnaapMKMarjo S. van der Knaap
- Nicole I. WolfNWNicole I. Wolf
Open Access
- 1 June 2013
- journal article
- case report
- Published by American Medical Association (AMA) in JAMA Neurology
- Vol. 70 (6) , 779-782
Abstract
ImportanceWe sought to illustrate improvement of cerebral white matter changes in metachromatic leukodystrophy after treatment with hematopoietic stem cell transplant (HSCT).ObservationsWe conducted serial magnetic resonance imaging (MRI) and proton magnetic resonance spectroscopy (1H-MRS) as standard follow-up after HSCT with cord blood in 1 patient with juvenile metachromatic leukodystrophy diagnosed before frank degenerative symptoms developed. We measured MRI and 1H-MRS changes. The white matter changes first increased after HSCT, then decreased in relation to the pre-HSCT MRI and 1H-MRS.Conclusions and RelevanceHematopoietic stem cell transplant, if performed early in metachromatic leukodystrophy, can not only stabilize but even improve cerebral white matter abnormalities. Our findings suggest a biological effect of HSCT.Keywords
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