Clinical Immunology and Allergy, Department of Medicine, UCLA David Geffen School of Medicine, 10833 Le Conte Avenue, 37-131 CHS, Los Angeles, CA 90095, USA.
Curr Allergy Asthma Rep. 2011 Aug;11(4):300-8. doi: 10.1007/s11882-011-0203-x.
Hereditary angioedema (HAE) is a genetic condition causing a significant burden of illness for affected individuals. Episodes of angioedema involving the skin, gastrointestinal tract, as well as the larynx and oropharynx are often unpredictable and cause significant morbidity and mortality. Isolation of the underlying protein deficiency, specifically the serine protease C1 inhibitor, and further description of its role in multiple physiologic cascades has led to the development of several specific therapies for HAE. This report provides a brief overview of HAE but focuses primarily on reviewing recently published clinical studies of therapeutics developed for medical management of the condition.
遗传性血管性水肿(HAE)是一种遗传性疾病,给患者带来了很大的疾病负担。皮肤、胃肠道以及喉部和口咽部的血管性水肿发作常常是不可预测的,并导致严重的发病率和死亡率。对潜在蛋白缺乏症,特别是丝氨酸蛋白酶 C1 抑制剂的分离,以及对其在多种生理级联反应中作用的进一步描述,导致了针对 HAE 的几种特定治疗方法的发展。本报告简要概述了 HAE,但主要侧重于审查最近发表的针对该疾病的医学治疗开发的治疗药物的临床研究。