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

Martje E. Van Egmond; 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

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, 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

Onderzoeksoutput: Bijdrage aan tijdschrift › Artikel › peer review

37 Citaten (Scopus)

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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.

Originele taal-2	Engels
Pagina's (van-tot)	779-782
Aantal pagina's	4
Tijdschrift	JAMA Neurology
Volume	70
Nummer van het tijdschrift	6
DOI's	https://doi.org/10.1001/jamaneurol.2013.629
Status	Gepubliceerd - jun. 2013
Extern gepubliceerd	Ja

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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.",

author = "{Van Egmond}, {Martje E.} and Pouwels, {Petra J.W.} and Boelens, {Jaap Jan} and Lindemans, {Caroline A.} and Frederik Barkhof and Steenwijk, {Martijn D.} and {Van Hasselt}, {Peter M.} and {Van Der Knaap}, {Marjo S.} and Wolf, {Nicole I.}",

year = "2013",

month = jun,

doi = "10.1001/jamaneurol.2013.629",

language = "English",

volume = "70",

pages = "779--782",

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T1 - Improvement of white matter changes on neuroimaging modalities after stem cell transplant in metachromatic leukodystrophy

AU - Van Egmond, Martje E.

AU - Pouwels, Petra J.W.

AU - Boelens, Jaap Jan

AU - Lindemans, Caroline A.

AU - Barkhof, Frederik

AU - Steenwijk, Martijn D.

AU - Van Hasselt, Peter M.

AU - Van Der Knaap, Marjo S.

AU - Wolf, Nicole I.

PY - 2013/6

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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.

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.

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

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