Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources.
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.


METHODS
Double-blind, randomized, parallel trial.


METHODS
University medical center and clinical research hospital.


METHODS
15 patients (4 children and 11 adults) randomly assigned to receive Ceredase and 15 patients (3 children and 12 adults) assigned to receive Cerezyme.


METHODS
Ceredase and Cerezyme were infused every 2 weeks for 9 months at a dose of 60 U/kg body weight.


METHODS
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.
Grabowski
G A
GA
Children's Hospital Medical Center, Cincinnati, Ohio.
Barton
N W
NW
Pastores
G
G
Dambrosia
J M
JM
Banerjee
T K
TK
McKee
M A
MA
Parker
C
C
Schiffmann
R
R
Hill
S C
SC
Brady
R O
RO
D000293:Adolescent; D000328:Adult; D002648:Child; D004311:Double-Blind Method; D005260:Female; D005776:Gaucher Disease / Q000097:blood / Q000188:drug therapy* / Q000201:enzymology / Q000473:pathology; D005962:Glucosylceramidase / Q000009:adverse effects / Q000276:immunology / Q000627:therapeutic use*; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007074:Immunoglobulin G / Q000096:biosynthesis; D008099:Liver / Q000473:pathology; D008297:Male; D009929:Organ Size / Q000187:drug effects; D010976:Platelet Count / Q000187:drug effects; D013154:Spleen / Q000473:pathology
D006454:Hemoglobins; D007074:Immunoglobulin G; C070292:alglucerase; D005962:Glucosylceramidase; C090568:imiglucerase