口服贝罗特拉斯塔特用于2至12岁以下患者的遗传性血管预防:APeX-P临时结果
Jolanta Bernatoniene1, Mélisande Bourgoin-Heck2, Mauro Cancian3
1Paediatric Immunology and Infectious Disease Department, Bristol Royal Hospital for Children, Bristol, United Kingdom.
概括
口服贝罗特拉斯塔特为幼儿遗传性血管 (HAE) 提供了耐受性良好,有效的长期预防. 这项研究显示,在12岁以下的患者中,HAE发作率显著降低.
科学领域:
- 药理学和治疗学 药理学和治疗学
- 遗传学和罕见疾病
- 儿科医学 儿科医学
背景情况:
- 遗传性血管 (HAE) 是一种罕见的遗传性疾病,通常在儿童时期出现,目前对12岁以下的患者有长期预防 (LTP) 选择,需要通过肠道给药.
- 贝罗特拉斯塔特是一种口服小分子血卡利克林抑制剂,已被批准用于12岁及以上患者的HAE预防.
研究的目的:
- 评估口服贝罗特拉斯塔的安全性和有效性作为遗传性血管 (HAE) 的长期预防 (LTP) 在2岁至12岁以下的儿科患者中.
- APeX-P试验 (NCT05453968) 代表了在这个特定的儿科HAE人群中对LTP的最大研究.
主要方法:
- 患者在开始治疗前接受了12周的标准护理期.
- 通过计划的中间分析,评估了口服贝罗特拉斯塔特的药理动力学,安全性和疗效.
- 这项研究包括29名患有HAE的儿科患者.
主要成果:
- 参与者的平均年龄为8.0岁,82.8%的人在6岁之前出现症状.
- 贝罗特拉斯塔特表现出有利的药理动力学参数,中位数Tmax为3.9小时.
- 没有报告任何与药物相关的严重不良事件,死亡或因不良事件而停止治疗. HAE发作率显著降低,从标准护理期间每4周的平均0.96次发作到48周的每4周的0次发作.
结论:
- 口服贝罗特拉斯塔特在2岁至12岁的儿科患者中耐受良好.
- 治疗导致遗传性血管发作发作率的早期和持续减少.
- 贝罗特拉斯塔特为年轻的HAE患者提供了一个有前途的口服预防方案.
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