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JAMA Neurol. 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.

Author information

1
Department of Child Neurology, VU University Medical Center, Amsterdam, the Netherlands.

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.

PMID:
23608771
DOI:
10.1001/jamaneurol.2013.629
[Indexed for MEDLINE]

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