Recombinant human C1 esterase inhibitor (Conestat alfa) for prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema

被引：9

作者：

Valerieva, Anna ^{[1
]}

Caccia, Sonia ^{[2
]}

Cicardi, Marco ^{[2
]}

机构：

[1] Med Univ Sofia, Univ Hosp Alexandrovska, Clin Ctr Allergol, Sofia, Bulgaria

[2] Univ Milan, Luigi Sacco Hosp, ASST Fatebenefratelli Sacco, Dept Biomed & Clin Sci, Milan, Italy

来源：

EXPERT REVIEW OF CLINICAL IMMUNOLOGY | 2018年 / 14卷 / 09期

关键词：

C1-inhibitor; C1-inhibitor deficiency; conestat alfa; glycosylation; hereditary angioedema; long-term prophylactic treatment; recombinant C1 inhibitor; rhC1-INH; prophylaxis serpin; BRADYKININ-MEDIATED ANGIOEDEMA; MANNAN-BINDING LECTIN; ISCHEMIA-REPERFUSION INJURY; INTERNATIONAL WORKING GROUP; HUMAN C1-ESTERASE INHIBITOR; N-TERMINAL DOMAIN; HUMAN C1-INHIBITOR; COMPLEMENT ACTIVATION; REPLACEMENT THERAPY; POPULATION PHARMACOKINETICS;

D O I：

10.1080/1744666X.2018.1503055

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

Introduction: Hereditary angioedema (HAE) due to C1 inhibitor (C1-INH) deficiency is a debilitating and potentially lethal disease. Management includes on-demand treatment of angioedema and their prophylaxis. Plasma derived C1-INH is an established treatment for both on demand and prophylaxis of HAE. Conestat alfa is a recombinant form of human C1-INH (rhC1-INH) produced in transgenic rabbits. It has granted drug's registration as treatment option for acute HAE attacks in adults and adolescents in Europe, America, and other countries. Long-term prophylaxis with rhC1-INH received recent consideration in clinical trials. Areas covered: This review will critically appraise available information about rhC1-INH (conestat alfa) prophylactic treatment in adult and adolescent patients with congenital C1-INH deficiency. Results from a phase II randomized placebo-controlled trial for prophylaxis of severe HAE evidenced positive treatment outcomes for its application, both twice or once weekly. Expert commentary: Phase II clinical studies suggest that rhC1-INH is a viable option for prophylaxis of HAE. Safety and tolerability data are comparable to other available HAE specific drugs, zeroing the possibility for blood-born viral transmission. Sustainability of modern technologies is granting a practically stable and continuous recombinant production process. With other available options, rhC1-INH facilitates tailoring HAE treatment to patients' needs.

引用

页码：707 / 718

页数：12

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