Allergy Associates, Boston, Massachusetts, USA.
Allergy Asthma Proc. 2010 Sep-Oct;31(5):398-406. doi: 10.2500/aap.2010.31.3387.
Hereditary angioedema (HAE) is a rare, potentially life-threatening disease that manifests as recurrent episodes of nonpruritic swelling that may affect the extremities, face, genitalia, gastrointestinal tract, and/or larynx. HAE is the result of a deficiency of functional C1-esterase inhibitor (C1-INH), a key regulator of the complement, coagulation, and kallikrein-kinin cascades. In HAE patients, overactivation of the kallikrein-kinin cascade results in excessive release of bradykinin, the mediator of the pain and swelling that is characteristic of HAE. Historically, treatment options for HAE have been limited, but newly approved and emerging therapies, such as C1-INH replacement products, a plasma kallikrein inhibitor, and a bradykinin B₂-receptor antagonist, appear to provide safe and effective relief for a significant proportion of patients with HAE. Because they may have therapeutic and practical advantages over existing HAE therapies, the new agents have the potential to improve the overall management of patients with HAE. This article reviews the results from recent clinical trials of these drugs and considers their role in clinical practice.
遗传性血管性水肿（HAE）是一种罕见的、可能危及生命的疾病，表现为反复发作的非瘙痒性肿胀，可能影响四肢、面部、生殖器、胃肠道和/或喉部。HAE 是功能性 C1-酯酶抑制剂（C1-INH）缺乏的结果，C1-INH 是补体、凝血和激肽释放酶-激肽系统的关键调节剂。在 HAE 患者中，激肽释放酶-激肽系统过度激活会导致缓激肽过度释放，这是 HAE 的疼痛和肿胀的特征介质。在历史上，HAE 的治疗选择有限，但新批准和新兴的疗法，如 C1-INH 替代产品、血浆激肽释放酶抑制剂和缓激肽 B₂受体拮抗剂，似乎为很大一部分 HAE 患者提供了安全有效的缓解。由于它们可能具有优于现有 HAE 疗法的治疗和实际优势，这些新药物有可能改善 HAE 患者的整体管理。本文综述了这些药物最近临床试验的结果，并考虑了它们在临床实践中的作用。
