Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials

Blood Vessels, Thrombosis & Hemostasis Pub Date : 2025-03-27 DOI:10.1016/j.bvth.2025.100069

Guy Young , Johnny Mahlangu , Lisa N. Boggio , Manuel Carcao , Yesim Dargaud , Miguel Escobar , Adam Giermasz , Cédric Hermans , Philip Kuriakose , Wolfgang Miesbach , Danielle Nance , Amina Rafique , Robert F. Sidonio Jr. , Kateryna V. Vilchevska , Michael Wang , Steven W. Pipe

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Abstract

Severe bleeding episodes (BEs) in persons with hemophilia A or B and inhibitors (PwHABIs) represent challenging clinical situations and can require treatment regimens lasting days or weeks before hemostatic control is achieved. Eptacog beta is a recombinant activated human factor VII bypassing agent approved for treating and controlling bleeding in PwHABIs aged ≥12 years. The aim of this study is to assess the efficacy and safety of eptacog beta for severe bleed treatment in PwHABIs during 2 phase 3 trials (PERSEPT 1 and PERSEPT 2). Patients could treat severe BEs with initial doses of 75 or 225 μg/kg eptacog beta at home, followed by subsequent 75 μg/kg eptacog beta infusions administered at predefined intervals in a hospital or hemophilia treatment center. Satisfactory treatment responses to eptacog beta were typically defined in this post hoc analysis by physician- and patient-reported hemostasis evaluations of “excellent” or “good.” Hemostatic control of an intracranial hemorrhage (ICH) in 1 patient was assessed by computed tomography. Seven PwHABIs (aged 1-50 years) treated 8 BEs considered severe or otherwise life threatening with eptacog beta during PERSEPT 1 and PERSEPT 2. Hemostatic control of 7 of these BEs (including 3 ICH events) was achieved. Eptacog beta treatment durations ranged from 25 minutes to 96 hours. No thrombotic events were reported, and eptacog beta was well tolerated. Most severe BEs resolved with eptacog beta treatment during PERSEPT 1 and PERSEPT 2. The PERSEPT 1 and PERSEPT 2 trials were registered at www.clinicaltrials.gov as #NCT02020369 and #NCT02448680, respectively.

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用抑制剂治疗血友病A或B患者的严重出血：PERSEPT 1和2试验的事后分析

血友病A或B患者的严重出血发作（BEs）和抑制剂（PwHABIs）代表着具有挑战性的临床情况，可能需要持续数天或数周的治疗方案才能达到止血控制。Eptacog β是一种重组活化的人因子7旁路药物，被批准用于治疗和控制年龄≥12岁的PwHABIs出血。本研究的目的是在2个3期试验（PERSEPT 1和PERSEPT 2）中评估eptacog β治疗PwHABIs严重出血的疗效和安全性。患者可以在家中以75或225 μg/kg的β β β初始剂量治疗严重的BEs，随后在医院或血友病治疗中心以预定的间隔注射75 μg/kg β β。对eptacog β满意的治疗反应通常在事后分析中由医生和患者报告的止血评价“优秀”或“良好”来定义。通过计算机断层扫描评估1例颅内出血患者的止血控制。7名PwHABIs（年龄1-50岁）在PERSEPT 1和PERSEPT 2期间使用eptacog β治疗8名被认为严重或危及生命的BEs。其中7例出血事件（包括3例脑出血事件）止血得到控制。Eptacog β治疗持续时间从25分钟到96小时不等。没有血栓事件的报道，eptacog β耐受性良好。大多数严重的BEs在PERSEPT 1和PERSEPT 2期间通过eptacog治疗得到缓解。PERSEPT 1和PERSEPT 2试验分别在www.clinicaltrials.gov注册为#NCT02020369和#NCT02448680。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Blood Vessels, Thrombosis & Hemostasis

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