Department of Allergology, Medical University of Sofia, Sofia, Bulgaria.
Department of Medicine, University of Padua, Padova, Italy.
Expert Rev Clin Immunol. 2019 Dec;15(12):1239-1248. doi: 10.1080/1744666X.2020.1693261. Epub 2019 Nov 19.
: Hereditary angioedema (HAE) with C1 esterase inhibitor deficiency (C1-INH-HAE) is a rare disease that manifests with cutaneous and/or submucosal swellings due to uncontrolled activation of the contact/kinin system. Attacks recur with unpredictable frequency and severity, laryngeal edema is potentially lethal, and the disease burden may severely disrupt patients' lives.: This review provides an overview of lanadelumab, a human monoclonal antibody targeted against plasma kallikrein that was recently approved for prevention of symptoms in C1-INH-HAE.: The phase III HELP Study demonstrated the efficacy of lanadelumab in reducing HAE attacks. These positive results are being further confirmed in the open-label extension study. This agent addresses some of the limitations of existing prophylactic options as tolerability issues, the need for intravenous administration and frequent dosing. Therefore, lanadelumab can profoundly improve the quality of life of patients with C1-INH-HAE.
遗传性血管性水肿（HAE）伴 C1 酯酶抑制剂缺乏症（C1-INH-HAE）是一种罕见疾病，其特征为接触/激肽系统不受控制激活导致皮肤和/或黏膜下肿胀。发作具有不可预测的频率和严重程度，喉头水肿可能是致命的，疾病负担可能严重扰乱患者的生活。
本文综述了 lanadelumab，一种针对血浆激肽释放酶的人源单克隆抗体，最近被批准用于预防 C1-INH-HAE 症状。III 期 HELP 研究证明了 lanadelumab 在减少 HAE 发作方面的疗效。这些积极的结果正在开放标签扩展研究中进一步证实。该药物解决了一些现有预防方案的局限性，如耐受性问题、需要静脉给药和频繁给药。因此，lanadelumab 可以显著改善 C1-INH-HAE 患者的生活质量。
