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Enzyme Therapy in Type 1 Gaucher Disease: Comparative Efficacy of Mannose-Terminated Glucocerebrosidase from Natural and Recombinant Sources

Gregory A. Grabowski, MD; Norman W. Barton, MD, PhD; Gregory Pastores, MD; James M. Dambrosia, PhD; Tapas K. Banerjee, MD; Mary Ann McKee, MD; Colette Parker, MD; Raphael Schiffmann, MD; Suvimol C. Hill, MD; and Roscoe O. Brady, MD
[+] Article, Author, and Disclosure Information

From Children's Hospital Medical Center, Cincinnati, Ohio; Mt. Sinai School of Medicine, New York, New York; and the National Institute of Neurological Disorders and Strokes, Bethesda, Maryland. Requests for Reprints: Gregory A. Grabowski, MD, Children's Hospital Medical Center, Division of Human Genetics, Pavilion 3-52, 3333 Burnet Avenue, Cincinnati, OH 45229-3039. Grant Support: A grant to Dr. Grabowski supported only and in toto the patient-related study costs. Other grant support was provided by the General Clinical Research Center at Mt. Sinai Medical Center (grant 2 M01 00071) and the Markey Center for Pediatric Molecular Genetics at the Children's Hospital Medical Center, Cincinnati, Ohio.

Copyright ©2004 by the American College of Physicians

Ann Intern Med. 1995;122(1):33-39. doi:10.7326/0003-4819-122-1-199501010-00005
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Objective: To compare the efficacy of mannose-terminated glucocerebrosidase prepared from natural (alglucerase; Ceredase, Genzyme Corp., Cambridge, Massachusetts) and recombinant (imiglucerase; Cere-zyme, 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.


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Figure 1.
Decreases in splenic volume from initial volume measured at 6 months in the Cerezyme (Group 1) and Ceredase (Group 2) treatment groups.horizontal line in boxesboxes

The values are presented as the percentage change from initial volume. The distribution of individual values, median value ( ), and interquartile range ( ), are shown. The outer quartile range is indicated by the vertical capped lines.

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