Successful treatment of bone marrow failure in Gaucher's disease with low-dose modified glucocerebrosidase
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P K Mistry1, S Davies, A Corfield
1Department of Medicine, University of Cambridge School of Clinical Medicine, Addenbrooke's Hospital.
The Quarterly Journal of Medicine
|July 1, 1992
Abstract:
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.
