Bone marrow transplantation in Gaucher's disease: effect of mixed chimeric state.
The effective dose and schedule of enzyme replacement therapy for Gaucher's disease have not been definitely established. We report a case of mixed chimeric state in an allogeneic BMT patient and followed her clinical and laboratory progress. The result shows that a low but sustained glucocerebrosidase level may provide symptomatic relief for this lysosomal disorder.
Chan
K W
KW
Department of Paediatrics, B.C.'s Children's Hospital, Vancouver, Canada.
Wong
L T
LT
Applegarth
D
D
Davidson
A G
AG
D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D002678:Chimera; D005260:Female; D005776:Gaucher Disease / Q000201:enzymology / Q000628:therapy*; D005962:Glucosylceramidase / Q000378:metabolism*; D006801:Humans; D014184:Transplantation, Homologous; D001617:beta-Glucosidase / Q000097:blood
D001617:beta-Glucosidase; D005962:Glucosylceramidase