Neal J Weinreb1. 1. 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
Abstract
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/ METHODS: 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. CONCLUSION: 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.
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/ METHODS: 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. CONCLUSION: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.
Authors: David Aviezer; Einat Brill-Almon; Yoseph Shaaltiel; Sharon Hashmueli; Daniel Bartfeld; Sarah Mizrachi; Yael Liberman; Arnold Freeman; Ari Zimran; Eithan Galun Journal: PLoS One Date: 2009-03-11 Impact factor: 3.240