Gupta Ratika, Balduzzi Jeffrey, Davis-Lorton Mark
NYU-Winthrop University Hospital, Dept of Allergy and Immunology, Mineola, NY 11501, USA.
NYU-Winthrop University Hospital, Dept of Internal Medicine, Mineola, NY 11501, USA.
Immunotherapy. 2018 Jun;10(8):635-642. doi: 10.2217/imt-2017-0049. Epub 2018 Mar 23.
Hereditary angioedema (HAE) is a rare genetic disease resulting in unpredictable and potentially life-threatening subcutaneous and submucosal attacks mediated by the vasoactive peptide, bradykinin. HAE often presents within the first or second decade of life, with attacks increasing in both frequency and severity over time. First-line therapies exert their action by replacing C1 inhibitor (C1-INH) or via blocking the production or function of bradykinin. Cinryze is a nanofiltered C1-INH, approved in Europe for the acute treatment, preprocedure prevention and routine prophylaxis of HAE attacks, and for routine prophylaxis of attacks in the USA. Of the current C1-INH preparations available, Cinryze shows particular promise in the safe and effective treatment and prophylaxis of HAE attacks in pediatric age patients.
遗传性血管性水肿（HAE）是一种罕见的遗传性疾病，由血管活性肽缓激肽介导，导致不可预测且可能危及生命的皮下和黏膜下发作。HAE通常在生命的第一个或第二个十年内出现，随着时间的推移，发作的频率和严重程度都会增加。一线治疗通过替代C1抑制剂（C1-INH）或通过阻断缓激肽的产生或功能来发挥作用。Cinryze是一种经过纳米过滤的C1-INH，在欧洲被批准用于HAE发作的急性治疗、术前预防和常规预防，在美国则用于发作的常规预防。在目前可用的C1-INH制剂中，Cinryze在安全有效地治疗和预防儿童年龄患者的HAE发作方面显示出特别的前景。
