OBJECTIVE: 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. DESIGN: Double-blind, 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 children and 12 adults) assigned toreceive Cerezyme. INTERVENTION: Ceredase and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight. OUTCOME MEASURES: 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. RESULTS: 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. CONCLUSIONS: 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.
RCT Entities:
OBJECTIVE: 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. DESIGN: Double-blind, 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 children and 12 adults) assigned to receive Cerezyme. INTERVENTION: Ceredase and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight. OUTCOME MEASURES: 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. RESULTS: 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. CONCLUSIONS: 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.
Authors: Martin D Witte; Wouter W Kallemeijn; Jan Aten; Kah-Yee Li; Anneke Strijland; Wilma E Donker-Koopman; Adrianus M C H van den Nieuwendijk; Boris Bleijlevens; Gertjan Kramer; Bogdan I Florea; Berend Hooibrink; Carla E M Hollak; Roelof Ottenhoff; Rolf G Boot; Gijsbert A van der Marel; Herman S Overkleeft; Johannes M F G Aerts Journal: Nat Chem Biol Date: 2010-10-31 Impact factor: 15.040
Authors: Paige Kaplan; Hagit Baris; Linda De Meirleir; Maja Di Rocco; Amal El-Beshlawy; Martina Huemer; Ana Maria Martins; Ioana Nascu; Marianne Rohrbach; Lynne Steinbach; Ian J Cohen Journal: Eur J Pediatr Date: 2012-07-08 Impact factor: 3.183
Authors: Xu He; Jason D Galpin; Michael B Tropak; Don Mahuran; Thomas Haselhorst; Mark von Itzstein; Daniel Kolarich; Nicolle H Packer; Yansong Miao; Liwen Jiang; Gregory A Grabowski; Lorne A Clarke; Allison R Kermode Journal: Glycobiology Date: 2011-11-07 Impact factor: 4.313
Authors: John Marshall; Kerry Anne McEachern; Wei-Lien Chuang; Elizabeth Hutto; Craig S Siegel; James A Shayman; Greg A Grabowski; Ronald K Scheule; Diane P Copeland; Seng H Cheng Journal: J Inherit Metab Dis Date: 2010-03-25 Impact factor: 4.982