Successful treatment of bone marrow failure in Gaucher's disease with low-dose modified glucocerebrosidase.
We report the beneficial effects of enzyme replacement therapy with mannose-terminated human glucocerebrosidase ('Ceredase') in a patient suffering from transfusion-dependent bone marrow failure due to Gaucher's disease. Treatment with low-dose enzyme infusions, given twice weekly, rapidly reversed the haematopoietic failure and incapacitating skeletal disease. It appears likely that prior splenectomy favourably influenced the response to this therapy.
Mistry
P K
PK
Department of Medicine, University of Cambridge School of Clinical Medicine, Addenbrooke's Hospital.
Davies
S
S
Corfield
A
A
Dixon
A K
AK
Cox
T M
TM
D001855:Bone Marrow Diseases / Q000188:drug therapy* / Q000209:etiology; D004334:Drug Administration Schedule; D005776:Gaucher Disease / Q000150:complications / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged
D005962:Glucosylceramidase