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伊米苷酶用于1型戈谢病的治疗：基于证据对其在治疗中地位的综述

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

作者信息

Serratrice Christine, Carballo Sebastian, Serratrice Jacques, Stirnemann Jérome

机构信息

Department of Internal Medicine and Rehabilitation, Geneva University Hospital, Thonex, Switzerland.

Department of General Internal Medicine, Geneva University Hospital, Geneva, Switzerland.

出版信息

Core Evid. 2016 Oct 14;11:37-47. doi: 10.2147/CE.S93717. eCollection 2016.

DOI:10.2147/CE.S93717
PMID:27790078
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5072572/
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.

摘要

引言

戈谢病是首个受益于酶替代疗法的溶酶体疾病，因此成为众多其他溶酶体疾病的模型。阿糖苷酶是首个从胎盘提取物中纯化得到的葡萄糖脑苷脂酶，随后被伊米苷酶所取代，后者是一种源自中国仓鼠卵巢细胞的葡萄糖脑苷脂酶。

目的

目的是回顾伊米苷酶用于1型戈谢病的相关证据。

证据综述

分析了来自临床试验和戈谢病登记处的数据。

结论

伊米苷酶已被处方使用，且具有出色的疗效和安全性。我们在此报告26年来发表的基于证据的数据，以证明伊米苷酶的使用合理性。

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本文引用的文献

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Glucosylsphingosine is a key biomarker of Gaucher disease.葡萄糖神经酰胺是戈谢病的关键生物标志物。
Am J Hematol. 2016 Nov;91(11):1082-1089. doi: 10.1002/ajh.24491. Epub 2016 Aug 8.
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Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase.用他利糖酶α进行酶替代疗法：对先前接受过伊米苷酶治疗的戈谢病成年患者的36个月安全性和疗效结果。
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Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.1型戈谢病患者对底物还原疗法与酶替代疗法的生化反应。
Orphanet J Rare Dis. 2016 Mar 24;11:28. doi: 10.1186/s13023-016-0413-3.
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Clonal Immunoglobulin against Lysolipids in the Origin of Myeloma.骨髓瘤起源中针对溶血磷脂的克隆性免疫球蛋白。
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Gaucher disease. Unusual presentation and mini-review.戈谢病。不寻常的表现及简要综述。
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Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase.长期使用维拉苷酶α治疗1型戈谢病初治或曾接受伊米苷酶治疗的儿童。
Mol Genet Metab. 2016 Feb;117(2):164-71. doi: 10.1016/j.ymgme.2015.05.012. Epub 2015 Jun 1.
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Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.依利格鲁司他与伊米苷酶治疗稳定的 1 型戈谢病患者的比较：一项 3 期、随机、开放标签、非劣效性试验。
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Am J Hematol. 2015 Jul;90(7):592-7. doi: 10.1002/ajh.24007.
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Achievement of therapeutic goals with low-dose imiglucerase in Gaucher disease: a single-center experience.低剂量伊米苷酶治疗戈谢病实现治疗目标：单中心经验
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Glucosylsphingosine is a highly sensitive and specific biomarker for primary diagnostic and follow-up monitoring in Gaucher disease in a non-Jewish, Caucasian cohort of Gaucher disease patients.葡糖神经酰胺是一种高度敏感且特异的生物标志物，用于非犹太白种人戈谢病患者队列中戈谢病的初步诊断及随访监测。
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