title: Improvement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy.
abstract: OBJECTIVE
  We sought to illustrate improvement of cerebral white matter changes in metachromatic leukodystrophy after treatment with hematopoietic stem cell transplant (HSCT).
  
  
  METHODS
  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
  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.
authors.affiliation: Department of Child Neurology, VU University Medical Center, Amsterdam, the Netherlands.
authors.forename: Martje E
authors.initials: ME
authors.lastname: van Egmond
authors.forename: Petra J W
authors.initials: PJ
authors.lastname: Pouwels
authors.forename: Jaap-Jan
authors.initials: JJ
authors.lastname: Boelens
authors.forename: Caroline A
authors.initials: CA
authors.lastname: Lindemans
authors.forename: Frederik
authors.initials: F
authors.lastname: Barkhof
authors.forename: Martijn D
authors.initials: MD
authors.lastname: Steenwijk
authors.forename: Peter M
authors.initials: PM
authors.lastname: van Hasselt
authors.forename: Marjo S
authors.initials: MS
authors.lastname: van der Knaap
authors.forename: Nicole I
authors.initials: NI
authors.lastname: Wolf
mesh_terms: D000293:Adolescent; D005260:Female; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000175:diagnosis* / Q000601:surgery*; D009413:Nerve Fibers, Myelinated / Q000473:pathology*; D059906:Neuroimaging / Q000639:trends; D033581:Stem Cell Transplantation / Q000639:trends