Eptacog Beta (Factor VIIa, Recombinant) Provides Rapid Bleed Resolution and Pain Relief in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

W. Miesbach¹, C. Hermans², S.W. Pipe³, J. Journeycake⁴, J. Ducore⁵, M. Escobar⁶, M. Wang⁷, D. Quon⁸, L. Boggio⁹, N. Chambron¹⁰, A. AL-Sabbagh¹¹, D. Bonzo¹¹, C. Macie¹², I.S. Mitchell¹³, J. Mahlangu¹⁴

¹Goethe University Hospital, Frankfurt, Germany, ²Saint-Luc University Hospital, Brussels, Belgium, ³University of Michigan, Ann Arbor, MI, United States, ⁴University of Oklahoma Health Sciences Center, Oklahoma City, OK, United States, ⁵University of California Davis, Sacramento, CA, United States, ⁶University of Texas Health Science Center, Houston, TX, United States, ⁷University of Colorado Denver, Aurora, CO, United States, ⁸Orthopaedic Hemophilia Treatment Center, Los Angeles, CA, United States, ⁹Rush University Medical Center, Chicago, IL, United States, ¹⁰LFB, Les Ulis, France, ¹¹LFB-USA, Framingham, MA, United States, ¹²HEMA Biologics, Louisville, KY, United States, ¹³Gloval, Broomfield, CO, United States, ¹⁴University of the Witwatersrand, Johannesburg, South Africa

Abstract Number: PB0544

Meeting: ISTH 2021 Congress

Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical

Background: Rapid bleed control in patients with hemophilia A/B with inhibitors (PwHABI) is critical to maintain normal musculoskeletal structure and function.

Aims: We evaluated the efficacy and safety of eptacog beta (EB; LFB/HEMA Biologics) for the treatment of bleeding episodes (BEs) in adult/adolescent (≥12 years of age) PwHABI.

Methods: PERSEPT 1 was a randomized, crossover, phase 3 study. Patients received either an initial dose (ID) of 75 or 225µg/kg EB followed by per protocol dosing of 75µg/kg at prespecified intervals (determined by clinical response). The primary efficacy endpoint (PEE) using the EMA definition was the proportion of BEs of all severity (mild, moderate and severe) with ‘good’ or ‘excellent’ responses at 12h. Other efficacy endpoints were time to response, number of administrations, bleeding recurrence, responses at 24h and pain assessments at 12h (Visual Analogue Scale).

Results: 465 mild/moderate and 3 severe BEs were treated in 27 PwHABI. Hemostatic response (PEE) was achieved in 94% of BEs in the 225µg/kg ID regimen (IDR; n=216) and 86% in the 75µg/kg IDR (n=252) within 12h. The median time to response was 3h and 6h respectively in mild/moderate BEs. Hemostasis was achieved at 12h for the 3 severe BEs. A single dose of 225µg/kg achieved response in 81.3% of BEs compared to 29% with the 75µg/kg dose regardless of bleed severity (Figure 1).

Figure 1. Successfully Treated Bleeding Episodes (Includes Mild/Moderate and Severe Bleeds) by Number of Infusions and Treatment Regimen
Pain was relieved in >89% of cases by 12h for both IDRs. The success proportion at 24h was >98%; most BEs had no recurrence or need for alternative treatment. Other efficacy results are detailed in Table 1. No thromboembolic, allergic, or anti-EB antibodies were observed.

Table 1. Primary, Secondary and Tertiary Efficacy Endpoints (N=468 BEs, Regardless of Severity*)

Conclusions: EB is an effective and safe bypassing agent that provides a new treatment option for BEs in adult/adolescent PwHABI. A single administration of 225µg/kg EB offers rapid BE resolution in the vast majority of patients.

To cite this abstract in AMA style:

Miesbach W, Hermans C, Pipe SW, Journeycake J, Ducore J, Escobar M, Wang M, Quon D, Boggio L, Chambron N, AL-Sabbagh A, Bonzo D, Macie C, Mitchell IS, Mahlangu J. Eptacog Beta (Factor VIIa, Recombinant) Provides Rapid Bleed Resolution and Pain Relief in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/eptacog-beta-factor-viia-recombinant-provides-rapid-bleed-resolution-and-pain-relief-in-adult-and-adolescent-patients-with-hemophilia-a-or-b-with-inhibitors/. Accessed November 28, 2023.

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