Imiglucerase and its use for the treatment of Gaucher's disease.
BACKGROUND
Gaucher's disease is caused by deficient lysosomal glucocerebrosidase activity. Intravenous enzyme replacement therapy with imiglucerase (Cerezyme, Genzyme Corporation, Cambridge, MA), a recombinant human glucocerebrosidase, ameliorates systemic manifestations such as hepatosplenomegaly, anemia, thrombocytopenia and skeletal abnormalities in patients with type 1 (non-neuronopathic) and type 3 (chronic neuronopathic) Gaucher's disease.


OBJECTIVE
The aim of this study was to identify and comment on the current issues related to imiglucerase for Gaucher's disease based on a review of published English language literature and personal clinical experience.


RESULTS
The following topics were covered with respect to imiglucerase: development, pharmacokinetics, preparation and administration, efficacy, pediatrics, pregnancy, type 3 Gaucher's disease, dosing, treatment interruptions, safety and alternative pharmacological therapies.


CONCLUSIONS
Imiglucerase is safe and well tolerated. In addition, it corrects the hepatic, splenic, hematologic and bone abnormalities observed with types 1 and 3 Gaucher's disease effectively and enhances health-related quality of life.
Weinreb
Neal J
NJ
University Research Foundation for Lysosomal Storage Diseases, Inc., Northwest Oncology Hematology Associates PA, 8170 Royal Palm Boulevard, Coral Springs, FL 33065, USA. boneal@winning.com
D000328:Adult; D000818:Animals; D002648:Child; D002986:Clinical Trials as Topic; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000503:physiopathology; D005962:Glucosylceramidase / Q000008:administration & dosage* / Q000009:adverse effects / Q000378:metabolism / Q000493:pharmacokinetics; D006801:Humans; D007275:Injections, Intravenous; D011247:Pregnancy; D011788:Quality of Life
D005962:Glucosylceramidase; C090568:imiglucerase