Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

P. D. Maaswinkel-Mooij; B. J.H.M. Poorthuis; P. M. Hoogerbrugge; O. F. Brouwer; J. M.J.J. Vossen

Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

P. D. Maaswinkel-Mooij, B. J.H.M. Poorthuis, P. M. Hoogerbrugge, O. F. Brouwer, J. M.J.J. Vossen

Onderzoeksoutput: Bijdrage aan tijdschrift › Artikel › peer review

4 Citaten (Scopus)

Samenvatting

The first report of a positive effect of allogeneic bone marrow transplantation (BMT) on the clinical course in a patient with a lysosomal storage disease was described in 1981. Since then, over 200 patients have been treated in this way but data are scarce and fragmentary. Allogeneic BMT involves replacement of the patient's haemopoietic system by that of a donor. The new cells that repopulate the body can correct the metabolic disturbance. Most experience with allogeneic BMT was gained in patients with mucopolysaccharidosis type I, metachromatic leukodystrophy and adrenoleukodystrophy. Allogeneic BMT reduces the amount of storage material in internal organs; skeletal abnormalities and neurological symptoms are at best stabilized. Transplantation-related mortality and morbidity are high. The applicability of allogeneic BMT is limited.

Vertaalde titel van de bijdrage	Allogeneic bone marrow transplantation in the treatment of (lysosomal) storage diseases
Originele taal-2	Nederlands
Pagina's (van-tot)	169-174
Aantal pagina's	6
Tijdschrift	Nederlands Tijdschrift voor Geneeskunde
Volume	142
Nummer van het tijdschrift	4
Status	Gepubliceerd - 24 jan. 1998
Extern gepubliceerd	Ja

Citeer dit

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title = "Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten",

abstract = "The first report of a positive effect of allogeneic bone marrow transplantation (BMT) on the clinical course in a patient with a lysosomal storage disease was described in 1981. Since then, over 200 patients have been treated in this way but data are scarce and fragmentary. Allogeneic BMT involves replacement of the patient's haemopoietic system by that of a donor. The new cells that repopulate the body can correct the metabolic disturbance. Most experience with allogeneic BMT was gained in patients with mucopolysaccharidosis type I, metachromatic leukodystrophy and adrenoleukodystrophy. Allogeneic BMT reduces the amount of storage material in internal organs; skeletal abnormalities and neurological symptoms are at best stabilized. Transplantation-related mortality and morbidity are high. The applicability of allogeneic BMT is limited.",

author = "Maaswinkel-Mooij, {P. D.} and Poorthuis, {B. J.H.M.} and Hoogerbrugge, {P. M.} and Brouwer, {O. F.} and Vossen, {J. M.J.J.}",

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AU - Maaswinkel-Mooij, P. D.

AU - Poorthuis, B. J.H.M.

AU - Hoogerbrugge, P. M.

AU - Brouwer, O. F.

AU - Vossen, J. M.J.J.

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AB - The first report of a positive effect of allogeneic bone marrow transplantation (BMT) on the clinical course in a patient with a lysosomal storage disease was described in 1981. Since then, over 200 patients have been treated in this way but data are scarce and fragmentary. Allogeneic BMT involves replacement of the patient's haemopoietic system by that of a donor. The new cells that repopulate the body can correct the metabolic disturbance. Most experience with allogeneic BMT was gained in patients with mucopolysaccharidosis type I, metachromatic leukodystrophy and adrenoleukodystrophy. Allogeneic BMT reduces the amount of storage material in internal organs; skeletal abnormalities and neurological symptoms are at best stabilized. Transplantation-related mortality and morbidity are high. The applicability of allogeneic BMT is limited.

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Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

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