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在患有遗传性血管的青少年中使用兰阿德鲁马布有效的长期预防:EMPOWER/ENABLE

Raffi Tachdjian^1,2, Aleena Banerji³, Paula J Busse⁴

¹Division of Allergy, Immunology and Rheumatology, UCLA School of Medicine, Los Angeles, California, USA.

Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology

|April 2, 2025

概括

拉纳德鲁马布有效且安全地预防青少年遗传性血管 (HAE) 发作. 这项研究显示,在接受兰德鲁马布治疗的年轻HAE患者中,发作频率和严重程度显著降低.

科学领域:

免疫学免疫学免疫学
遗传学遗传学是一种遗传学.
药理学药理学是指药理学的学科.

背景情况:

遗传性血管 (HAE) 的症状通常在童年开始,在青春期的频率和严重程度增加.
有限的现实世界数据存在于青少年的长期HAE预防.
针对血kallikrein的单克隆抗体lanadelumab正在评估HAE攻击的预防.

研究的目的:

评估lanadelumab在青少年患者HAE预防中的有效性和安全性.
分析 EMPOWER 和 ENABLE 4 期研究中青少年参与者的数据.
评估lanadelumab对年轻人群中HAE发作率和严重程度的影响.

主要方法:

患有HAE-C1INH的青少年患者 (12至18岁以下) 接受了开放标签的lanadelumab (300毫克每两周).
通过患者在治疗期间报告的HAE发作来衡量有效性.
通过治疗出现的不良事件 (TEAE) 和严重不良事件来监测安全性.

主要成果:

新患者每月HAE发作率显著降低 (84.2%的平均值,92.9%的中位数).
已确定的患者在治疗期间的攻击率非常低 (0.04次攻击/月).
大多数HAE发作和TEAE是轻度/中度;没有TEAE与lanadelumab相关.

结论:

兰德鲁马布在青少年HAE患者中显示出有效性和良好的安全性.
这些发现与混合成人和青少年群体的第三阶段研究结果一致.
拉纳德鲁马布为患有HAE的青少年提供了一种可行的预防方案.

关键词:

青少年青少年青少年有效性有效性.遗传性血管功能拉纳德卢马布拉姆是什么意思预防预防预防预防在安全方面,安全是安全的.

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