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Case Reports
. 2013 Jun;70(6):779-82.
doi: 10.1001/jamaneurol.2013.629.

Improvement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy

Martje E van Egmond  1 , Petra J W Pouwels, Jaap-Jan Boelens, Caroline A Lindemans, Frederik Barkhof, Martijn D Steenwijk, Peter M van Hasselt, Marjo S van der Knaap, Nicole I Wolf
Affiliations
Case Reports

Improvement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy

Martje E van Egmond et al. JAMA Neurol. 2013 Jun.

Abstract

Importance: We sought to illustrate improvement of cerebral white matter changes in metachromatic leukodystrophy after treatment with hematopoietic stem cell transplant (HSCT).

Observations: We 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 relevance: Hematopoietic 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.

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