期刊
EXPERT OPINION ON PHARMACOTHERAPY
卷 9, 期 11, 页码 1987-2000出版社
TAYLOR & FRANCIS LTD
DOI: 10.1517/14656566.9.11.1987
关键词
enzyme replacement therapy; Gaucher's disease; imiglucerase; lysosomal storage disorders
Background: Gaucher's disease is caused by deficient lysosomal glucocerebrosidase activity. Intravenous enzyme replacement therapy with imiglucerase (Cerezyme (R), 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.
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