PB0693 - Efficacy and Safety of Eptacog Beta (Recombinant Activated FVII) in Patients with Hemophilia A or B with Inhibitors According to Time to Initial Infusion: a Post-Hoc Analysis from PERSEPT-1

Monday, June 26, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Cedric Hermans, MD, PhD (he/him/his)

Head Haemostasis and Thrombosis Unit
St-Luc University Hospital
Brussels, Brussels Hoofdstedelijk Gewest, Belgium

Co-Author(s)

Miguel Escobar, MD

Professor of Medicine and Pediatrics
University of Texas Health Science Center of Houston, McGovern Medical School, Gulf States Hemophilia and Thrombophilia Center, Houston, Texas
Houston, Texas, United States
YA

Yasmina Abajas

Clinical Assistant Professor
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Suchitra Acharya, MD

Director Hemostasis and Thrombosis Center
Cohen Children’s Medical Center, Northwell Health
New Hyde Park, New York, United States
Maria Teresa Alvarez-Román, MD, PhD

Associate Professor
Department of Hematology, University Hospital La Paz, Autonoma University of Madrid, Madrid, Spain
Madrid, Madrid, Spain
GB

Glaivy Batsuli

Assistant Professor of Pediatrics
Emory University, Department of Pediatrics
Atlanta, Georgia, United States
YD

Yesim Dargaud

Director Hemostasis Unit
Hôpital Cardiologique Louis Pradel, Université Lyon 1
Lyon, Rhone-Alpes, France
MJ

Maissaa Janbain

Associate Director
Louisiana Center for Bleeding and Clotting Disorders, Tulane University Medical Center, LA, USA
New Orleans, Louisiana, United States
PK

Philip Kuriakose

Assistant professor
Wayne State University Medical School, Henry Ford Health System
Detroit, Michigan, United States
Johnny Mahlangu, MD (he/him/his)

Professor and Director
Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
Johannesburg, Gauteng, South Africa
Maria Elisa Mancuso, MD, PhD (she/her/hers)

Senior Consultant in Haematology
IRCCS Humanitas Research Hospital
Milan, Lombardia, Italy
Wolfgang Miesbach, MD

Professor and Head of Coagulation Disorders and Comprehensive Care Centre
University Hospital Frankfurt, Frankfurt, Germany
Frankfurt, Hessen, Germany
SY

Shenghua Yuan

Senior Biostatistician
LFB-USA
Framingham, Massachusetts, United States
IM

Ian Mitchell

Medical Science Liaison
HEMA Biologics
Louisville, Kentucky, United States
Manuel Carcao, MD, FRCPC

Professor
Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada
Toronto, Ontario, Canada

Background: Early resolution of bleeding episodes (BEs) may reduce BE-related complications for patients with hemophilia A/B with inhibitors (PwHABI), including pain, musculoskeletal complications, absenteeism from work/school and hospitalization. The impact of time to initial dose of rFVIIa on bleed resolution is poorly understood/documented.

Aims: We evaluated the impact of time to initial infusion and dosing regimen on bleed resolution and safety of eptacog beta.

Methods: PERSEPT-1 was a Phase 3 trial of eptacog beta for the treatment of BEs in PwHABI (NCT02020369). Patients received an initial eptacog beta dose regimen (IDR) of either 75 or 225μg/kg, followed by 75μg/kg at predefined intervals over 24 hours until hemostasis was achieved. Primary endpoint was the proportion of successfully treated BEs (i.e. ‘good’/’excellent’ response) 12 hours after initial infusion. Safety assessments included physical examination, laboratory and immunogenicity testing, and assessment of adverse events (AEs). This post-hoc analysis evaluated the efficacy and safety of eptacog beta according to time between start of bleed and first infusion ( < 30 (N=228), 30-60 (N=105) and ≥60 (N=73) min).

Results: Across 468 BEs treated, the overall success proportion for the primary endpoint was 85.3% (95% CI 77.0-93.5) with 81.0% (70.9-91.0) and 90.3% (82.9-97.7) in the 75 and 225μg/kg IDRs, respectively. Initial infusion at < 30min was associated with numerically higher success proportion for both IDRs, compared with an initial infusion at 30-60 and ≥60min; but the differences did not reach statistical significance. No significant differences were found when comparing both IDRs (Figure 1). Seven treatment-related AEs occurred in 2 patients, independently of the time between bleed start and first infusion. There were no thromboembolic events, hypersensitivity reactions, or deaths (Table 1). No neutralizing antibodies to eptacog beta were detected.

Conclusion(s): Eptacog beta resulted in successful resolution of BEs with both IDRs with no difference in safety, regardless of the time to initial infusion.