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. 2025 Mar 27;2(3):100069.
doi: 10.1016/j.bvth.2025.100069. eCollection 2025 Aug.

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 Young  1   2 , Johnny Mahlangu  3 , Lisa N Boggio  4 , Manuel Carcao  5 , Yesim Dargaud  6 , Miguel Escobar  7 , Adam Giermasz  8 , Cédric Hermans  9 , Philip Kuriakose  10 , Wolfgang Miesbach  11 , Danielle Nance  12 , Amina Rafique  13 , Robert F Sidonio Jr  14 , Kateryna V Vilchevska  15 , Michael Wang  16 , Steven W Pipe  17
Affiliations

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 Young et al. Blood Vessel Thromb Hemost. .

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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Conflict of interest statement

Conflict-of-interest disclosure: G.Y. has received consulting fees from ASC Biotherapeutics, BioMarin, Centessa, CSL Behring, Genentech/Roche, HEMA Biologics/LFB, Novo Nordisk, Octapharma, Pfizer, Sanofi, Genzyme, Spark, and Takeda; and funds for research support from Sanofi. J.M. has received research grants from BioMarin, Catalyst Biosciences, CSL Behring, Novartis, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, and uniQure; served as a consultant or member of the scientific board for BioMarin, CSL Behring, Catalyst Biosciences, Novo Nordisk, Roche, Sanofi, Spark, and Takeda; and received speaker bureau fees from the International Society on Thrombosis and Haemostasis (ISTH), Novo Nordisk, Pfizer, Roche, Sanofi, Takeda, and the World Federation of Hemophilia (WFH). L.N.B. has received research support from Pfizer, Bayer, Octapharma, Sanofi, Principia Biopharma, LFB, and HEMA Biologics; and consulting fees from Genentech, Genzyme, Novo Nordisk, and Octapharma. M.C. has received research support from Novartis, Novo Nordisk, Pfizer, Roche, Sanofi, and Takeda; and honoraria for speaking/participating in advisory boards from Bayer, LFB, Novo Nordisk, Pfizer, Roche, Sanofi, and Takeda. Y.D. has received funding for research from CSL Behring, Novo Nordisk, LFB, Bayer, Centessa, Pfizer, and Octapharma; and acted as a paid consultant to Sobi, CSL Behring, Pfizer, Roche-Chugai, LFB, Takeda, Novo Nordisk, and Octapharma. M.E. has received grants and research funding from the American Thrombosis and Hemostasis Network (ATHN), uniQure, Takeda, Bayer, Sanofi Regeneron, Novo Nordisk, Pfizer, and LFB; and honoraria and consulting fees from Takeda, Bayer, LFB, HEMA Biologics, BioMarin, Regeneron, Genentech/Roche, Sanofi, Novo Nordisk, CSL Behring, Pfizer, Kedrion, the National Hemophilia Foundation (NHF; now known as the National Bleeding Disorders Foundation), and Magellan. A.G. has served as a consultant or advisory board member for Sanofi, HEMA Biologics, Vega Therapeutics, Alexion, and Genzyme; has been a member of Data and Safety Monitoring Committee for Adrenas; and has acted as a speaker for Sanofi, Alexion, and Genzyme. C.H. has received research funding from Bayer, BioMarin, CSL Behring, Novo Nordisk, Pfizer, Shire/Takeda, and Sobi; and honoraria and speaker bureau fees from Bayer, Belgium Red Cross Central Fractionation Facility (CAF-DCF), CSL Behring, Hoffmann-La Roche, LFB, Novo Nordisk, Octapharma, Pfizer, Shire/Takeda, Sobi, and uniQure. W.M. reports consultancy and research funding from Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, SOBI, and Takeda. D.N. has received consulting fees from HEMA Biologics. R.F.S. Jr has acted as a paid consultant for HEMA Biologics, LFB, Octapharma, Genentech/Roche, Bayer, Pfizer, Novo Nordisk, Guardian, Vega, Takeda, and Hemab; and has investigator-initiated grant funding from HEMA Biologics/LFB, Takeda, and Octapharma. M.W. has served on advisory boards for Bioverativ/Sanofi, Takeda, CSL Behring, Novo Nordisk, Bayer, Genentech, HEMA Biologics, Vega Therapeutics, and BioMarin; and served as a past investigator for HEMA Biologics and Novo Nordisk. S.W.P. has received grants/research support from Siemens and YewSavin; and served as a consultant to Apcintex, ASC Therapeutics, Bayer, BioMarin, CSL Behring, Equilibra Bioscience, GeneVentiv, HEMA Biologics, Freeline, LFB, Novo Nordisk, Pfizer, Poseida Therapeutics, Regeneron/Intellia, Roche/Genentech, Sanofi, Takeda, Spark Therapeutics, and uniQure. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Dosing schedules for 75 and 225 μg/kg severe BE dosing regimens in PERSEPT 1 and PERSEPT 2. Intervals between eptacog beta infusions and total severe BE treatment duration could be extended at the discretion of the treating physician, depending on response to treatment.
Figure 2.
Figure 2.
Treatment of patients experiencing severe BEs in PERSEPT 1 and PERSEPT 2 with eptacog beta. Time points for the first recorded “good” or excellent” hemostasis evaluation obtained from the treating physician and from the patient/caregiver for each BE are noted by asterisks. Treatment outcomes of “bleed control” or “insufficient response” are indicated.
Figure 3.
Figure 3.
Eptacog beta treatment durations and number of infusions by patient and bleed type. (A) Eptacog beta treatment durations for severe BEs in PERSEPT 1 and PERSEPT 2. (B) Number of infusions received by PERSEPT 1 and PERSEPT 2 patients for severe BEs.
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