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Ⅰ型戈谢病药物velaglucerase alfa的药理作用及临床研究

Pharmacology and clinical study of velaglucerase alfa for treatment of type 1 Gaucher disease
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摘要 Velaglucerase alfa(VprivTM)是一种水解性溶酶体葡糖脑苷脂专一性酶,用于儿童和成人Ⅰ型戈谢病的长期酶替代疗法,于2010年2月由美国FDA批准上市。文中就VprivTM的作用机制、药动学、临床研究以及不良反应等方面进行了综述。 VprivTM is a hydrolytic lysosomal glucocerebroside-specific enzyme,and used as long-term enzyme replacement therapy（ERT） for pediatric and adult patients with type 1 Gaucher disease.It was approved by the United States Food and Drug Administration on February,2010.The mechanism of action,pharmacokinetics,clinical studies,and adverse reactions of this drug were reviewed in this article.
作者 孙韬华 王唯红 曹国颖 傅得兴 胡欣
机构地区 山东大学药学院药物分析研究所 卫生部北京医院药学部
出处 《中国新药杂志》 CAS CSCD 北大核心 2011年第4期297-299,共3页 Chinese Journal of New Drugs
基金 重大新药创制科技重大专项(2008ZX09312)
关键词 VprivTM velaglucerase ALFA 戈谢病 临床研究 VprivTM velaglucerase alfa Gaucher disease clinical study
分类号 R973 [医药卫生—药品]
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参考文献8

  • 1Lable approved on 02/26/2010 for VPRIVTM , NDA no. 022575 [ EB/OL ]. [ 2010 - 02 - 26 ] http ://www. accessdata, fda. gov/ drugsatfda_docs/label/2010/022575 lbl. pdf.
  • 2石志鸿,张本恕.戈谢病与帕金森病研究进展[J].医学综述,2008,14(4):583-585. 被引量：3
  • 3ZIMRAN A,ALTARESCU A,PHILIPS M, et al. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience [ J]. Blood, 2010,115(23) :4651 -4656.
  • 4ZIMRAN A, LOWEDAY K, FRATAZZI C,et al. A pharmacokinetie analysis of a novel enzyme replacement therapy with Gene- Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease[ J ]. Blood Cell Mol Dis, 2007,39:115 - 118.
  • 5ZIMRANA A,GONZALEZB D, LUKINAC EA,et al. Enzyme replacement therapy with velaglucerase alfa significantly improves key clinical paramelers in type 1 Gaucher disease: positive results from a randomized, double-blind, global, phase llI study [ J ]. Mol Genet Metab,2010,99( 2 ) : S41.
  • 6ZIMRANA A,GONZALEZB D, CROMBEZC E,et al. Enzyme replacement therapy with velaglucerase alfa improves key clinical parameters in a pediatric subgroup with type 1 Gaucher disease [ J ] Mol Genet Metab, 2010,99(2) : S40 -S41.
  • 7ELSTEINA D,ZIMRANA A, COHNB GM, et al. Five-year safety and efficacy of velaglucerase alfa in Gaucher disease type 1 : experience in clinic and home settings[ J]. Mol Genet Metab, 2010,99 (2) : S18.
  • 8RUIZ J, BARZEGAR S, CLARKE A, et al. Antigenic differences in patients receiving velaglucerase alfa or imiglucerase treatment [J]. Mol Genet Metab, 2010,99(2): S33.

二级参考文献16

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中国新药杂志
2011年 第4期

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