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- Title
Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources.
- Authors
Grabowski, G A; Barton, N W; Pastores, G; Dambrosia, J M; Banerjee, T K; McKee, M A; Parker, C; Schiffmann, R; Hill, S C; Brady, R O
- Abstract
<bold>Objective: </bold>To compare the efficacy of mannose-terminated glucocerbrosidase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Cambridge, Massachusetts) and recombinant (imiglucerase; Cerezyme, Genzyme Corp.) sources in treating type 1 Gaucher disease.<bold>Design: </bold>Double-blind, randomized, parallel trial.<bold>Setting: </bold>University medical center and clinical research hospital.<bold>Patients: </bold>15 patients (4 children and 11 adults) randomly assigned to receive Ceredase and 15 patients (3 children and 12 adults) assigned to receive Cerezyme.<bold>Intervention: </bold>Ceredase and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight.<bold>Outcome Measures: </bold>Hemoglobin levels, platelet counts, and serum acid phosphatase and angiotensin-converting enzyme activities were monitored every 2 weeks during the trial. Hepatic and splenic 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 assay.<bold>Results: </bold>No significant differences were found in the rate or extent of improvement in hemoglobin levels, platelet counts, serum acid phosphatase or angiotensin-converting enzyme activities, or hepatic or splenic volumes between either treatment group. The incidence of IgG antibody formation was greater in the Ceredase group (40%) than in the Cerezyme group (20%). No major immunologic adverse events occurred in either group.<bold>Conclusions: </bold>Our study shows the therapeutic similarity of Ceredase and Cerezyme. Cerezyme has the advantage of being theoretically unlimited in supply and free of potential pathogenic contaminants.
- Publication
Annals of Internal Medicine, 1995, Vol 122, Issue 1, p33
- ISSN
0003-4819
- Publication type
journal article
- DOI
10.7326/0003-4819-122-1-199501010-00005