Horváth Hanga Réka, Visy Beáta, Kőhalmi Kinga Viktória, Balla Zsuzsanna, Andrási Noémi, Czaller Ibolya, Zotter Zsuzsanna, Farkas Henriette
Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Szentkirályi Street 46, Budapest HU-1088, Hungary.
Hungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Szentkirályi Street 46, Budapest HU-1088, Hungary; Heim Pál National Institute of Paediatrics, Üllői Street 86, Budapest HU-1089, Hungary.
Clin Immunol. 2025 Oct;279:110542. doi: 10.1016/j.clim.2025.110542. Epub 2025 Jun 11.
Hereditary angioedema (HAE) imposes a significant burden on patients due to the unpredictability of attacks. Long-term prophylaxis (LTP) aims to prevent these episodes and improve quality of life. Over the past five decades, LTP options have evolved from non-specific treatments (fresh frozen plasma, attenuated androgens, antifibrinolytics) to more targeted therapies (intravenous or subcutaneous C1 inhibitor (C1INH), lanadelumab, berotralstat).
To assess the use, efficacy, and safety of various LTP therapies among Hungarian HAE patients.
Data from the Hungarian HAE Registry (1979-2023) were analysed, including LTP usage, attack rates, side effects, and laboratory parameters.
Danazol and tranexamic acid use increased over the first 30 years but has declined in the last decade. Conversely, the use of modern LTP therapies and the proportion of patients relying solely on on-demand medication have significantly increased over the past decade. Danazol and tranexamic acid reduced attack rates in 60 % of patients without significant changes in the laboratory parameters at the lowest effective doses. Furthermore, 100 % of patients on subcutaneous C1INH, lanadelumab, and berotralstat experienced fewer attacks and improved quality of life compared to pre-treatment periods, with no serious side effects or significant laboratory abnormalities.
Modern prophylaxis usage is increasing among Hungarian HAE-C1INH patients, following the global trends. Both modern and traditional LTP options proved safe and effective in our patient population when used with appropriate monitoring.
遗传性血管性水肿（HAE）因发作不可预测，给患者带来了沉重负担。长期预防（LTP）旨在预防这些发作并改善生活质量。在过去的五十年中，LTP方案已从非特异性治疗（新鲜冷冻血浆、减毒雄激素、抗纤维蛋白溶解剂）发展为更具针对性的疗法（静脉内或皮下注射C1抑制剂（C1INH）、拉那单抗、贝罗司他）。
评估匈牙利HAE患者中各种LTP疗法的使用情况、疗效和安全性。
分析了匈牙利HAE注册中心（1979 - 2023年）的数据，包括LTP使用情况、发作率、副作用和实验室参数。
达那唑和氨甲环酸的使用在前30年有所增加，但在过去十年中有所下降。相反，在过去十年中，现代LTP疗法的使用以及仅依赖按需用药的患者比例显著增加。达那唑和氨甲环酸使60%的患者发作率降低，在最低有效剂量下实验室参数无显著变化。此外，与治疗前相比，接受皮下注射C1INH、拉那单抗和贝罗司他治疗的患者中有100%发作次数减少且生活质量得到改善，没有严重副作用或显著的实验室异常。
遵循全球趋势，匈牙利HAE - C1INH患者中现代预防疗法的使用正在增加。在我们的患者群体中，当进行适当监测时，现代和传统的LTP方案都证明是安全有效的。
