Imiglucerase in the management of Gaucher disease type 1: an evidence-based review of its place in therapy

Core Evidence Pub Date : 2016-10-14 DOI:10.2147/CE.S93717

C. Serratrice, S. Carballo, J. Serratrice, J. Stirnemann

引用次数: 21

Abstract

Introduction Gaucher disease is the first lysosomal disease to benefit from enzyme replacement therapy, thus serving as model for numerous other lysosomal diseases. Alglucerase was the first glucocerebrosidase purified from placental extracts, and this was then replaced by imiglucerase – a Chinese hamster ovary cell-derived glucocerebrosidase. Aim The aim was to review the evidence underlying the use of imiglucerase in Gaucher disease type 1 Evidence review Data from clinical trials and Gaucher Registries were analyzed. Conclusion Imiglucerase has been prescribed and found to have an excellent efficacy and safety profile. We report herein the evidence-based data published for 26 years justifying the use of imiglucerase.

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imigluc酶在戈谢病1型治疗中的应用:基于证据的评价

戈谢病是第一个受益于酶替代治疗的溶酶体疾病，因此可以作为许多其他溶酶体疾病的模型。醛葡萄糖苷酶是从胎盘提取物中纯化出来的第一个糖脑苷酶，随后被一种中国仓鼠卵巢细胞衍生的糖脑苷酶——半氨基葡萄糖苷酶所取代。本研究的目的是回顾来自临床试验和戈歇注册中心的数据分析，以支持在戈歇病1型中使用伊米格鲁糖酶。结论Imiglucerase具有良好的疗效和安全性。我们在此报告26年来发表的基于证据的数据，证明了imiglucerase的使用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Core Evidence PHARMACOLOGY & PHARMACY-

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期刊介绍： Core Evidence evaluates the evidence underlying the potential place in therapy of drugs throughout their development lifecycle from preclinical to postlaunch. The focus of each review is to evaluate the case for a new drug or class in outcome terms in specific indications and patient groups The emerging evidence on new drugs is reviewed at key stages of development and evaluated against unmet needs