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

Translated title of the contribution: Allogeneic bone marrow transplantation in the treatment of (lysosomal) storage diseases

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

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

4 Citations (Scopus)

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.

Translated title of the contribution	Allogeneic bone marrow transplantation in the treatment of (lysosomal) storage diseases
Original language	Dutch
Pages (from-to)	169-174
Number of pages	6
Journal	Nederlands Tijdschrift voor Geneeskunde
Volume	142
Issue number	4
Publication status	Published - 24 Jan 1998
Externally published	Yes

Cite this

@article{f51d40d6b7244a24a6c5a7745ef204be,

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.\}",

year = "1998",

month = jan,

day = "24",

language = "Nederlands",

volume = "142",

pages = "169--174",

journal = "Nederlands Tijdschrift voor Geneeskunde",

issn = "0028-2162",

publisher = "Vereniging Nederlands Tijdschrift voor Geneeskunde",

number = "4",

}

TY - JOUR

T1 - Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

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.

PY - 1998/1/24

Y1 - 1998/1/24

N2 - 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.

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.

UR - https://www.scopus.com/pages/publications/0032561969

M3 - Artikel

C2 - 9557021

AN - SCOPUS:0032561969

SN - 0028-2162

VL - 142

SP - 169

EP - 174

JO - Nederlands Tijdschrift voor Geneeskunde

JF - Nederlands Tijdschrift voor Geneeskunde

IS - 4

ER -

Allogene beenmergtransplantatie bij de behandeling van (lysosomale) stapelingsziekten

Abstract

Fingerprint

Cite this