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遗传性血管性水肿的新型疗法。

Novel therapies for hereditary angioedema.

作者信息

Zuraw Bruce L

机构信息

University of California San Diego, La Jolla, CA 92093-0732, USA.

出版信息

Immunol Allergy Clin North Am. 2006 Nov;26(4):691-708. doi: 10.1016/j.iac.2006.09.007.

DOI:10.1016/j.iac.2006.09.007
PMID:17085285
Abstract

Advances in our understanding of the molecular mechanisms underlying hereditary angioedema (HAE) have led to the development of new treatment modalities. Five new drugs for the treatment of HAE are currently undergoing clinical testing in the United States. These novel therapeutics can be divided into two groups: drugs that replace C1 inhibitor (C1INH) functional activity and drugs that abrogate the bradykinin-mediated increase in vascular permeability associated with HAE attacks. The first group includes two plasma-derived C1INH concentrates as well as a recombinant transgenic human C1INH protein, and the second group includes an engineered plasma kallikrein inhibitor as well as a B2 bradykinin receptor antagonist. This article reviews the rationale, development, and potential use of these novel therapeutics.

摘要

我们对遗传性血管性水肿(HAE)潜在分子机制认识的进展,促成了新治疗方式的发展。目前有五种治疗HAE的新药正在美国进行临床试验。这些新型疗法可分为两类:替代C1抑制剂(C1INH)功能活性的药物,以及消除缓激肽介导的与HAE发作相关的血管通透性增加的药物。第一类包括两种血浆源性C1INH浓缩物以及一种重组转基因人C1INH蛋白,第二类包括一种工程化血浆激肽释放酶抑制剂以及一种B2缓激肽受体拮抗剂。本文综述了这些新型疗法的理论依据、研发情况及潜在用途。

相似文献

1
Novel therapies for hereditary angioedema.遗传性血管性水肿的新型疗法。
Immunol Allergy Clin North Am. 2006 Nov;26(4):691-708. doi: 10.1016/j.iac.2006.09.007.
2
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Nonallergic angioedema: role of bradykinin.非过敏性血管性水肿:缓激肽的作用
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Recombinant human C1 esterase inhibitor for the treatment of hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE).重组人C1酯酶抑制剂用于治疗因C1抑制剂缺乏所致的遗传性血管性水肿(C1-INH-HAE)。
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DX-88 and HAE: a developmental perspective.
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Critical role of kallikrein in hereditary angioedema pathogenesis: a clinical trial of ecallantide, a novel kallikrein inhibitor.激肽释放酶在遗传性血管性水肿发病机制中的关键作用:新型激肽释放酶抑制剂依库珠单抗的一项临床试验
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Current and future therapy for hereditary angioedema.
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J Health Econ Outcomes Res. 2025 Jul 9;12(2):1-10. doi: 10.36469/001c.141171. eCollection 2025.
2
Development of a disease-specific quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency (HAE-QoL): Spanish multi-centre research project.遗传性血管性水肿患者生活质量问卷的制定:西班牙多中心研究项目(HAE-QoL)。
Health Qual Life Outcomes. 2012 Jul 20;10:82. doi: 10.1186/1477-7525-10-82.
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Induction of selective blood-tumor barrier permeability and macromolecular transport by a biostable kinin B1 receptor agonist in a glioma rat model.
生物稳定激肽 B1 受体激动剂在胶质瘤大鼠模型中诱导选择性血-肿瘤屏障通透性和大分子转运。
PLoS One. 2012;7(5):e37485. doi: 10.1371/journal.pone.0037485. Epub 2012 May 21.
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The first probable case of hereditary angioedema in Vietnam.越南首例遗传性血管性水肿疑似病例。
Allergy Asthma Immunol Res. 2012 May;4(3):165-7. doi: 10.4168/aair.2012.4.3.165. Epub 2012 Feb 10.
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Complement-targeted therapeutics.补体靶向疗法。
Nat Biotechnol. 2007 Nov;25(11):1265-75. doi: 10.1038/nbt1342.