Department of Dermatology, Graduate School of Biomedical & Health Sciences, Hiroshima University, Hiroshima, Japan.
Department of Internal Medicine, Kyushu University Beppu Hospital, Oita, Japan.
Allergol Int. 2021 Jan;70(1):45-54. doi: 10.1016/j.alit.2020.07.008. Epub 2020 Sep 9.
Hereditary angioedema (HAE) is characterized by unpredictable, recurring and painful swelling episodes that can be disabling or even life-threatening. Awareness of HAE has progressively grown worldwide, and options for treatment of acute attacks and prevention of future attacks continue to expand; however, unmet needs in diagnosis and treatment remain. In Japan, recognition of HAE within the medical community remains low, and numerous obstacles complicate diagnosis and access to treatment. Importance of timely treatment of HAE attacks with on-demand therapies is continually demonstrated; recommended agents per the WAO/EAACI treatment guidelines published in 2018 include C1 inhibitor (C1-INH) concentrate, ecallantide, and icatibant. In Japan, multiple factors contribute to delayed HAE treatment (potentially leading to life-threatening consequences), including difficulties in finding facilities at which C1-INH agents are readily available. Recognition of challenges faced in Japan can help promote efforts to address current needs and expand access to effective therapies. Icatibant, a potent, selective bradykinin B receptor antagonist, has demonstrated inhibition of various bradykinin-induced biological effects in preclinical studies and has shown efficacy in treating attacks in various clinical settings (e.g. clinical trials, real-world studies), and HAE patient populations (e.g. with C1-INH deficiency, normal C1-INH). Icatibant was approved in Japan for the treatment of HAE attacks in September 2018; its addition to the HAE treatment armamentarium contributes to improved patient care. In Japan, disease awareness and education campaigns are warranted to further advance the management of HAE patients in light of the unmet needs and the emerging availability of modern diagnostic approaches and therapies.
遗传性血管性水肿（HAE）的特征是反复发作、不可预测且伴有疼痛的肿胀发作，可能导致残疾甚至危及生命。HAE 在全球范围内的认知度逐渐提高，治疗急性发作和预防未来发作的选择也在不断增加；然而，在诊断和治疗方面仍存在未满足的需求。在日本，医学界对 HAE 的认识仍然较低，诊断和获得治疗的诸多障碍也较为复杂。及时使用按需治疗治疗 HAE 发作的重要性不断得到证明；根据 2018 年发布的 WAO/EAACI 治疗指南推荐的药物包括 C1 抑制剂（C1-INH）浓缩物、艾卡替班和依卡替班。在日本，多种因素导致 HAE 治疗延迟（可能导致危及生命的后果），包括难以找到 C1-INH 药物易于获得的医疗机构。认识到日本面临的挑战有助于促进努力满足当前需求并扩大有效治疗方法的可及性。依卡替班是一种强效、选择性缓激肽 B 受体拮抗剂，在临床前研究中已证明可抑制多种缓激肽诱导的生物学效应，在各种临床环境（例如临床试验、真实世界研究）和 HAE 患者人群（例如 C1-INH 缺乏症、正常 C1-INH）中治疗发作均显示出疗效。依卡替班于 2018 年 9 月在日本获批用于治疗 HAE 发作；它的加入为 HAE 治疗提供了更多选择，有助于改善患者的治疗效果。在日本，鉴于存在未满足的需求以及现代诊断方法和治疗手段的出现，有必要开展疾病宣传和教育活动，进一步推进 HAE 患者的管理。
