Cerezyme (imiglucerase) is an enzyme replacement therapy indicated for managing hematologic, visceral, and skeletal manifestations of Type 1 Gaucher disease in patients ≥2 years, demonstrating efficacy in improving anemia, thrombocytopenia, hepatosplenomegaly, and bone pathology.

Therapeutic Effects of Cerezyme in Type 1 Gaucher Disease

Cerezyme, a recombinant β-glucocerebrosidase, hydrolyzes accumulated glucocerebroside in lysosomes, addressing the enzymatic deficiency in Type 1 Gaucher disease. Clinical trials (e.g., RC 91-0110) demonstrated significant improvements: hemoglobin increased by 1.9 g/dL, platelets by 22.7 ×10³/mL³, liver volume reduced by 11%, and spleen volume by 35% after 6 months. Long-term data (24 months) showed sustained efficacy, with hemoglobin rising 2.4 g/dL and spleen volume decreasing 57%. Bone imaging revealed cortical thickening and reduced lucencies in 64% of patients. Therapeutic effects are dose-dependent (2.5–60 units/kg) and tailored to disease severity. No efficacy data exist for neurological symptoms (not specified in Type 1) or pediatric patients <2 years (safety/effectiveness unestablished). Adverse reactions include hypersensitivity and infusion-related events but do not negate clinical benefits when managed appropriately.