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

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.

Authors

Institutions

Publication Details

Journal
Blood Vessels Thrombosis & Hemostasis
Published
2025-03-27
DOI
https://doi.org/10.1016/j.bvth.2025.100069
Primary Topic
Hemophilia Treatment and Research
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

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

Guy A. Young, Cedric R.J.R. Hermans, Philip Kuriakose, Steven W. Pipe et al.
Blood Vessels Thrombosis & Hemostasis
Hemophilia Treatment and Research
article

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

Guy A. Young, Cedric R.J.R. Hermans, Philip Kuriakose, Steven W. Pipe, Yesim G. Dargaud, Lisa Boggio, Kateryna Vilchevska, Danielle Nance, Manuel Carção, Robert F. Sidonio, Amina Rafique, Adam Giermasz, Wolfgang Miesbach, Johnny Ndoni Mahlangu, Miguel A. Escobar, Michael Wang
article en

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.

Blood Vessels Thrombosis & HemostasisVol. 2(3)
National Health Laboratory Service (ZA), Goethe University Frankfurt (DE), University of Southern California (US), Rush University Medical Center (US), Cliniques Universitaires Saint-Luc (BE), Children's Hospital of Los Angeles (US), University of Michigan (US), Hospital for Sick Children (CA), Banner Health (US), Henry Ford Hospital (US), Levine Cancer Institute (US), Hospices Civils de Lyon (FR), Banner - University Medical Center Phoenix (US), University Hospital Frankfurt (DE), Thrombosis Research Institute (GB), Children's Healthcare of Atlanta (US), Indiana Hemophilia and Thrombosis Center (US), Children's Specialized Hospital (US), University of Colorado Anschutz Medical Campus (US), University of California, Davis (US), The University of Texas Health Science Center at Houston (US)
Zero hunger
Openalex Percentile: Top 92%
Hemophilia Treatment and Research
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.