Hereditary angioedema (HAE) is a rare disease that causes episodic attacks of subcutaneous and submucosal edema, which can be painful, incapacitating, and potentially fatal. These attacks are mediated by excessive bradykinin production, as a result of uncontrolled activation of the plasma kallikrein/kinin system, which is caused by a C1 esterase inhibitor deficiency or dysfunction in HAE types 1 and 2, respectively. For many years, treatment options were limited to therapies with substantial adverse effects, insufficient efficacy, or difficult routes of administration. Increased insights in the pathophysiology of HAE have paved the way for the development of new therapies with fewer side effects. In the last two decades, several targeted novel therapeutic strategies for HAE have been developed, for both long-term prophylaxis and on demand treatment of acute attacks. This article reviews the advances in the development of more effective and convenient treatment options for HAE and their anticipated effects on morbidity, mortality, and quality of life. The emergence of these improved treatment options will presumably change current HAE guidelines, but adherence to these recommendations may become restricted by high treatment costs. It will therefore be essential to determine the indications and identify the patients that will benefit most from these newest treatment generations. Ultimately, current preclinical research into gene therapies may eventually lead the way towards curative treatment options for HAE. In conclusion, an increasing shift towards the use of highly effective long-term prophylaxis is anticipated, which should drastically abate the burden on patients with hereditary angioedema.

遗传性血管性水肿 (HAE) 是一种罕见的疾病，会导致皮下和黏膜下水肿的发作性发作，这可能是痛苦的、失能的，甚至可能致命。这些攻击是由过度的缓激肽产生介导的，这是由于血浆激肽释放酶/激肽系统失控激活的结果，这分别是由 1 型和 2 型 HAE 中的 C1 酯酶抑制剂缺乏或功能障碍引起的。多年来，治疗选择仅限于具有严重副作用、疗效不足或给药途径困难的疗法。对 HAE 病理生理学的深入了解为开发副作用更少的新疗法铺平了道路。在过去的二十年中，已经开发了几种针对 HAE 的新型治疗策略，用于急性发作的长期预防和按需治疗。本文回顾了在开发更有效和方便的 HAE 治疗方案方面取得的进展，以及它们对发病率、死亡率和生活质量的预期影响。这些改进的治疗方案的出现可能会改变当前的 HAE 指南，但遵守这些建议可能会受到高额治疗费用的限制。因此，必须确定适应症并确定将从这些最新一代治疗中受益最多的患者。最终，目前对基因疗法的临床前研究可能最终会引领 HAE 的治愈性治疗选择。总之，预计将越来越多地转向使用高效的长期预防措施，