Diaz-Menindez Maximiliano, Morgenstern-Kaplan Dan, Cuervo-Pardo Lyda, Alvarez-Arango Santiago, Gonzalez-Estrada Alexei
Division of Pulmonary, Allergy, and Sleep Medicine, Department of Medicine, Mayo Clinic, Jacksonville, FL, USA.
Department of Internal Medicine, University of Miami/Jackson Health System, Miami, FL, USA.
Ther Clin Risk Manag. 2023 Mar 30;19:313-317. doi: 10.2147/TCRM.S310376. eCollection 2023.
Hereditary angioedema (HAE) is a condition characterized by episodes of cutaneous and submucosal edema. Angioedema of the extremities and abdominal attacks are the most common manifestations of the disease. It can also affect the upper airways with the potential of becoming life-threatening. The two most common causes of HAE are a deficiency of C1 inhibitor (classified as type 1 HAE) or a dysfunction of C1 inhibitor (type 2 HAE). A malfunction or deficiency of C1 inhibitor leads to an overactivated plasma kallikrein (an inflammatory vasoactive peptide), that increases bradykinin, mediating the angioedema episodes in patients with HAE. To minimize the difficulties of this pathology and to improve patients' quality of life, prevention of this condition is essential. Berotralstat is a unique option for oral administration for routine prophylaxis. This drug acts by binding to kallikrein and reducing its plasma activity, lowering bradykinin levels. Open-label studies have demonstrated the effectiveness of a single daily dose of berotralstat 150 mg in preventing HAE attacks. This review aims to examine studies performed to elucidate the efficacy, safety, and tolerability of berotralstat.
遗传性血管性水肿（HAE）是一种以皮肤和黏膜下水肿发作为特征的疾病。四肢血管性水肿和腹部发作是该疾病最常见的表现。它也可影响上呼吸道，有危及生命的可能。HAE最常见的两个病因是C1抑制剂缺乏（归类为1型HAE）或C1抑制剂功能障碍（2型HAE）。C1抑制剂功能异常或缺乏会导致血浆激肽释放酶过度激活（一种炎性血管活性肽），进而增加缓激肽，介导HAE患者的血管性水肿发作。为了尽量减少这种病症带来的困难并提高患者的生活质量，预防这种疾病至关重要。贝罗他司他是用于常规预防的口服给药的独特选择。该药物通过与激肽释放酶结合并降低其血浆活性，从而降低缓激肽水平发挥作用。开放标签研究已证明每日单次服用150 mg贝罗他司他预防HAE发作的有效性。本综述旨在研究为阐明贝罗他司他的疗效、安全性和耐受性而开展的研究。
