2岁至12岁以下遗传性血管患者的兰德鲁马布:第三期春季研究的结果
Marcus Maurer1, William R Lumry2, H Henry Li3
1Institute 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, Immunology and Allergology, Berlin, Germany.
The journal of allergy and clinical immunology. In practice
|September 20, 2023
概括
兰德鲁马布显著减少了遗传性血管 (HAE) 发作,并改善了2至12岁儿童的生活质量. 治疗表明,发作率下降了94.8%,大多数患者仍然没有发作.
科学领域:
- 免疫学 免疫学 免疫学
- 儿科 儿科 儿科
- 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,严重的发作会影响生活质量.
- 对于6岁以下的儿童,没有批准长期预防治疗.
研究的目的:
- 评估2岁至12岁以下患者的兰阿德鲁马布的安全性,药理动力学和疗效.
- 评估拉纳德卢马布对儿科HAE患者与健康相关的生活质量 (HRQoL) 的影响.
主要方法:
- 春季研究招募了21名患有HAE的儿科患者.
- 2-6岁的患者每4周服用150毫克兰阿德鲁马布 (Q4W);6-12岁的患者每2周服用150毫克兰阿德鲁马布 (Q2W),如果在26周内没有发作,可以选择切换到Q4W.
- 治疗持续时间为52周.
主要成果:
- 没有报告任何严重的治疗出现的不良事件 (TEAE) 或因TEAE而中止治疗.
- 从基线观察到HAE攻击率减少了94.8% (1.84到0.08次/月).
- 21名患者中有16名 (76.2%) 没有发作,HRQoL有显著改善.
结论:
- 拉纳德鲁马布 (150毫克Q2W和Q4W) 是安全有效的,用于预防2至12岁儿童的HAE发作.
- 治疗lanadelumab导致在这个儿科群体的健康相关的生活质量大幅改善.
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