Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

M.E. van Egmond; P.J.W. Pouwels; J.J. Boelens; C.A. Lindemans; F. Barkhof; M.D. Steenwijk; P.M. van Hasselt; M.S. van der Knaap; N.I. Wolf

doi:https://doi.org/10.1001/jamaneurol.2013.629

Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

M.E. van Egmond, P.J.W. Pouwels, J.J. Boelens, C.A. Lindemans, F. Barkhof, M.D. Steenwijk, P.M. van Hasselt, M.S. van der Knaap, N.I. Wolf

Research output: Contribution to journal › Article › Academic › peer-review

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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.Wemeasured 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. ©2013 American Medical Association. All rights reserved.

Original language	English
Pages (from-to)	779-782
Journal	JAMA Neurology
Volume	70
Issue number	6
DOIs	https://doi.org/10.1001/jamaneurol.2013.629
Publication status	Published - 2013

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https://doi.org/10.1001/jamaneurol.2013.629

Cite this

@article{475893778a24448198703929cf76b607,

title = "Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy",

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.Wemeasured 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. {\textcopyright}2013 American Medical Association. All rights reserved.",

author = "{van Egmond}, M.E. and P.J.W. Pouwels and J.J. Boelens and C.A. Lindemans and F. Barkhof and M.D. Steenwijk and {van Hasselt}, P.M. and {van der Knaap}, M.S. and N.I. Wolf",

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doi = "https://doi.org/10.1001/jamaneurol.2013.629",

language = "English",

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T1 - Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

AU - van Egmond, M.E.

AU - Pouwels, P.J.W.

AU - Boelens, J.J.

AU - Lindemans, C.A.

AU - Barkhof, F.

AU - Steenwijk, M.D.

AU - van Hasselt, P.M.

AU - van der Knaap, M.S.

AU - Wolf, N.I.

PY - 2013

Y1 - 2013

N2 - 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.Wemeasured 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. ©2013 American Medical Association. All rights reserved.

AB - 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.Wemeasured 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. ©2013 American Medical Association. All rights reserved.

U2 - https://doi.org/10.1001/jamaneurol.2013.629

DO - https://doi.org/10.1001/jamaneurol.2013.629

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