ENZYME THERAPY IN TYPE-1 GAUCHER DISEASE - COMPARATIVE EFFICACY OF MANNOSE-TERMINATED GLUCOCEREBROSIDASE FROM NATURAL AND RECOMBINANT SOURCES

Objective: To compare the efficacy of mannoseterminated glucocerebrosi dase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Camb ridge, Massachusetts) and recombinant (imiglucerase; Cerezyme, Genzyme Corp.) sources in treating type 1 Gaucher disease. Design: Double-bli nd, randomized, parallel trial. Setting: University medical center and clinical research hospital. Patients: 15 patients (4 children and 11 adults) randomly assigned to receive Ceredase and 15 patients (3 child ren and 12 adults) assigned to receive Cerezyme. Intervention: Ceredas e and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight. Outcome Measures: Hemoglobin levels, platelet count s, and serum acid phosphatase and angiotensin-converting enzyme activi ties were monitored every 2 weeks during the trial. Hepatic and spleni c volumes were assessed at the time of randomization and after 6 and 9 months of enzyme infusion. Formation of IgG antibodies to Ceredase or Cerezyme was monitored every 3 months by radioimmunoprecipitation ass ay. Results: No significant differences were found in the rate or exte nt of improvement in hemoglobin levels, platelet counts, serum acid ph osphatase or angiotensin-converting enzyme activities, or hepatic or s plenic volumes between either treatment group. The incidence of IgG an tibody formation was greater in the Ceredase group (40%) than in the C erezyme group (20%). No major immunologic adverse events occurred in e ither group. Conclusions: Our study shows the therapeutic similarity o f Ceredase and Cerezyme. Cerezyme has the advantage of being theoretic ally unlimited in supply and free of potential pathogenic contaminants .