[Allogeneic bone marrow transplantation in the treatment of (lysosomal) storage diseases].
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.
Maaswinkel-Mooij
P D
PD
Afd. Kindergeneeskunde, Academisch Ziekenhuis, Leiden.
Poorthuis
B J
BJ
Hoogerbrugge
P M
PM
Brouwer
O F
OF
Vossen
J M
JM
D000326:Adrenoleukodystrophy / Q000628:therapy; D000328:Adult; D016026:Bone Marrow Transplantation / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D007966:Leukodystrophy, Metachromatic / Q000628:therapy; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D008297:Male; D008059:Mucopolysaccharidosis I / Q000628:therapy; D016896:Treatment Outcome