Institute of Allergology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.
Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Allergology and Immunology, Berlin, Germany.
Clin Exp Allergy. 2022 Sep;52(9):1048-1058. doi: 10.1111/cea.14206. Epub 2022 Aug 7.
In patients with hereditary angioedema (HAE), bradykinin causes swelling episodes by activating bradykinin B receptors. Icatibant, a selective bradykinin B receptor antagonist, is approved for on-demand treatment of HAE attacks. The Icatibant Outcome Survey (IOS; NCT01034969) is an ongoing observational registry initiated in 2009 to monitor the effectiveness/safety of icatibant in routine clinical practice. As of March 2019, 549 patients with HAE type 1 or 2 from the IOS registry had been treated of 5995 total attacks. This article reviews data published from IOS over time which have demonstrated that the effectiveness of icatibant in a real-world setting is comparable to efficacy in clinical trials; one dose is effective for the majority of attacks; early treatment (facilitated by self-administration) leads to faster resolution and shorter attack duration; effectiveness/safety of icatibant has been shown across a broad range of patient subgroups, including children/adolescents and patients with HAE with normal C1 inhibitor levels; and tolerability has been demonstrated in patients aged ≥65 years. Additionally, this review highlights how IOS data have provided valuable insights into patients' diagnostic journeys and treatment behaviours across individual countries. Such findings have helped to inform clinical strategies and guidelines to optimise HAE management and limit disease burden. This research was sponsored by Takeda Development Center Americas, Inc. Takeda Development Center Americas, Inc., provided funding to Excel Medical Affairs for support in writing and editing this manuscript.
在遗传性血管性水肿（HAE）患者中，缓激肽通过激活缓激肽 B 受体引起肿胀发作。依卡替班，一种选择性缓激肽 B 受体拮抗剂，被批准用于按需治疗 HAE 发作。依卡替班结局调查（IOS；NCT01034969）是一项正在进行的观察性登记研究，于 2009 年启动，旨在监测依卡替班在常规临床实践中的有效性/安全性。截至 2019 年 3 月，IOS 登记处的 549 名 HAE 1 型或 2 型患者已接受了 5995 次总发作的治疗。本文回顾了 IOS 随时间发布的已发表数据，这些数据表明，依卡替班在真实环境中的有效性与临床试验中的疗效相当；大多数发作使用一剂即可有效；早期治疗（通过自我给药来促进）可导致更快的缓解和更短的发作持续时间；依卡替班的有效性/安全性已在广泛的患者亚组中得到证实，包括儿童/青少年和 C1 抑制剂水平正常的 HAE 患者；并且在≥65 岁的患者中已证实具有耐受性。此外，本综述强调了 IOS 数据如何为各国患者的诊断过程和治疗行为提供了有价值的见解。这些发现有助于为优化 HAE 管理和减轻疾病负担提供临床策略和指南。这项研究由武田开发中心美洲公司赞助。武田开发中心美洲公司向 Excel Medical Affairs 提供资金，以支持本文的撰写和编辑。
