PB1188 - Efficacy and safety of eptacog beta for resolution of spontaneous versus traumatic bleeding episodes in patients with hemophilia A or B with inhibitors

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

Jerzy Windyga, MD, PhD

Professor
Department of Hemostasis Disorders and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
Warsaw, Mazowieckie, Poland

Co-Author(s)

Shannon Meeks, MD, MBA

Professor of Pediatrics
Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Department of Pediatrics, Emory University School of Medicine
Atlanta, Georgia, United States
SA

Suchitra Acharya

Director Hemostasis and Thrombosis Center
Cohen Children’s Medical Center, Northwell Health
New Hyde Park, New York, United States
LB

Lisa Boggio

Adult Hematologist
Rush University Medical Center
Chicago, Illinois, United States
SB

Santiago Bonanad

Head of Thrombosis and Hemostasis Unit
Haemostasis, Thrombosis, Arteriosclerosis and Vascular Biology Research Group, Medical Research Institute Hospital La Fe, Valencia, Spain; Thrombosis and Haemostasis Unit, Haematology Service, La Fe University and Polytechnic Hospital, Valencia, Spain
Valencia, Comunidad Valenciana, Spain
GC

Giancarlo Castaman

Medical Director
Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy
Florence, Toscana, Italy
AH

Annie Harroche

Pediatric Hematologist
CRC-MHC Hôpital Necker-Enfants malades, Paris, France
Paris, Ile-de-France, France
JJ

Janna Journeycake

Pediatric Hematologist
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Johnny Mahlangu, MD, MMed

Professor in Haematology
University of the Witwatersrand and NHLS
Johannesburg, South Africa
Lynn Malec, MD, MSc

Associate Investigator
Versiti Blood Research Institute
Milwaukee, Wisconsin, United States
Catherine McGuinn, MD

HTC Director; Pediatric Hematologist
New York Weill Cornell Center; Reg Comp Hemophilia Diag & Trmt Center
New York, New York, United States
UN

Ulrike Nowak-Göttl

MD, Head of Institute of Clinical Chemistry,
Thrombosis & Haemostasis Treatment Centre, University Hospital Schleswig-Holstein, Germany
Kiel, Schleswig-Holstein, Germany
AR

Amina Rafique

Adult Hematologist
Louisiana Comprehensive Hemophilia Center; Tulane University Medical Center
New Orleans, Louisiana, United States
MR

Mark T. Reding

Professor and Director
Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, MN, USA
Minneapolis, Minnesota, United States
CM

Christopher Macie

Senior Medical Director
HEMA Biologics
Louisville, Kentucky, United States
HW

Hongying Wang

Director Biostatistics
LFB-USA
Framingham, Massachusetts, United States
Cédric Hermans, Professor

Professor, Head of Hematology
Cliniques Universitaires Saint-Luc
Brussels, Brussels Hoofdstedelijk Gewest, Belgium

Background: Prophylaxis with non-factor replacement therapy has significantly reduced the incidence of spontaneous bleeds in patients with hemophilia. However, bypassing agents remain the first line to treat traumatic bleeding episodes (BEs). Eptacog beta is a new bypassing agent approved in the US for BE treatment in patients with hemophilia A or B with inhibitors (PwHABI) aged ≥12 years.

Aims: To evaluate eptacog beta efficacy and safety for treatment of spontaneous versus traumatic BEs in PwHABI.

Methods: PERSEPT 1 was a randomized, cross-over, phase 3 trial (NCT02020369, LFB funded) in which PwHABI aged ≥12-75 years received eptacog beta for treatment of BEs using an initial dose regimen (IDR) of either 75 or 225μg/kg followed by 75μg/kg at predefined intervals over 24 hours. The primary endpoint, per EMA definition, was the proportion of successfully treated BEs (i.e., ‘good’/‘excellent’ response), irrespective of BE severity, 12 hours after the initial dose. IEC/IRB approval was granted, and all patients (or parents/legal guardians) provided informed consent.

Results: Among the 27 patients in PERSEPT 1, 27 had spontaneous BEs (197 BEs treated with 75µg/kg IDR and 184 BEs treated with 225µg/kg IDR) and 21 had traumatic BEs (53 BEs treated with 75µg/kg IDR and 32 BEs treated with 225µg/kg IDR). The location of BEs is presented in Figure 1. A similarly high proportion of spontaneous and traumatic BEs achieved successful resolution at 12 and 24 hours (Table 1). Two patients experienced seven treatment-related adverse events [infusion site discomfort (n=4), infusion site hematoma (n=2) and body temperature increase (n=1)] during 2 spontaneous and 3 traumatic BEs. There were no thromboembolic events, hypersensitivity reactions, or deaths; no neutralizing antibodies to eptacog beta were detected.

Conclusion(s): Both eptacog beta IDRs achieved successful and safe resolution of spontaneous and traumatic BEs at 12 and 24 hours in adolescent and adult PwHABI.