Li Huamin Henry
Institute for Asthma and Allergy, Chevy Chase, MD, USA.
Patient Prefer Adherence. 2016 Sep 7;10:1727-37. doi: 10.2147/PPA.S86379. eCollection 2016.
Hereditary angioedema (HAE) is a rare genetic disease characterized by episodic subcutaneous or submucosal swelling. The primary cause for the most common form of HAE is a deficiency in functional C1 esterase inhibitor (C1-INH). The swelling caused by HAE can be painful, disfiguring, and life-threatening. It reduces daily function and compromises the quality of life of affected individuals and their caregivers. Among different treatment strategies, replacement with C1-INH concentrates is employed for on-demand treatment of acute attacks and long-term prophylaxis. Three human plasma-derived C1-INH preparations are approved for HAE treatment in the US, the European Union, or both regions: Cinryze(®), Berinert(®), and Cetor(®); however, only Cinryze is approved for long-term prophylaxis. Postmarketing studies have shown that home therapy (self-administered or administered by a caregiver) is a convenient and safe option preferred by many HAE patients. In this review, we summarize the role of self-administered plasma-derived C1-INH concentrate therapy with Cinryze at home in the prophylaxis of HAE.
遗传性血管性水肿（HAE）是一种罕见的遗传性疾病，其特征为发作性皮下或黏膜下肿胀。最常见形式的HAE的主要病因是功能性C1酯酶抑制剂（C1-INH）缺乏。HAE引起的肿胀可能会疼痛、毁容且危及生命。它会降低日常功能，损害患者及其护理人员的生活质量。在不同的治疗策略中，使用C1-INH浓缩物进行替代治疗可用于急性发作的按需治疗和长期预防。三种源自人血浆的C1-INH制剂在美国、欧盟或这两个地区均被批准用于HAE治疗：Cinryze（®）、Berinert（®）和Cetor（®）；然而，只有Cinryze被批准用于长期预防。上市后研究表明，家庭治疗（自我给药或由护理人员给药）是许多HAE患者首选的方便且安全的选择。在本综述中，我们总结了在家中使用Cinryze进行自我给药的源自人血浆的C1-INH浓缩物治疗在HAE预防中的作用。
