Blood:Emicizumab预防用于儿童A型血友病

2019-10-14 QQY MedSci原创

Emicizumab是一种双特异性的人源化单克隆抗体,通过桥接激活因子(F) IX和FX来恢复A型血友病的FVIII激活功能缺陷。Guy Young等人开展了一项3期临床试验,评估emicizumab预防用于儿童A型血友病的疗效和安全性。招募既往接受过间歇性/预防性旁通药物(BPAs)治疗的受试者,予以皮下emicizumab治疗:A组:1.5mg/kg·周;B组:3mg/kg·2周;C组:6mg

Emicizumab是一种双特异性的人源化单克隆抗体,通过桥接激活因子(F) IX和FX来恢复A型血友病的FVIII激活功能缺陷。Guy Young等人开展了一项3期临床试验,评估emicizumab预防用于儿童A型血友病的疗效和安全性。

招募既往接受过间歇性/预防性旁通药物(BPAs)治疗的受试者,予以皮下emicizumab治疗:A组:1.5mg/kg·周;B组:3mg/kg·2周;C组:6mg/kg·4周。评估药代动力学、安全性和效果。

共招募了85位12岁以下的患者。A组65人,年化需处理的出血事件率(ABR)为0.3(95% CI 0.17-0.50),77%的患儿未发生需处理的出血事件。对15位既往服用过BPA预防的受试者进行单独对比,结果显示emicizumab预防可将ABR降低99%。B组和C组,分别有10为患儿,ABRs分别是0.2(95% CI 0.03-1.72)和2.2(95% CI 0.69-6.81)。最常见的副反应事件有鼻咽炎和注射部位反应;无血栓事件发生。88位受试者中有2位产生了具有中和特性的抗药抗体(ADAs),与emicizumab血药浓度降低有关;1位表现为无效果,另1位,随时间的推移,在未干预也没出血的情况下,ADAs自行消失。无论采用了何种治疗方案,所有其他受试者的emicizumab血药浓度均达到了有效水平。

本研究表明emicizumab预防是一种非常有效的治疗儿童A型血友病的新药物和抑制剂。

原始出处:

Guy Young, et al.A multicenter, open-label, phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors.blood.2019001869.https://doi.org/10.1182/blood.2019001869

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    2020-08-03 snf701207
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    2019-10-16 by2011

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