I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with
Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based
on clinical severity of disease and response to therapy.
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed
by enzymatic or molecular assay At least 3 organ systems affected, based on the following
criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary
compromise Symptoms compromise daily activities or risk longevity No neurologic disease