The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors

Gulf States Hemophilia and Thrombophilia Center, Houston, Texas, USA. Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy. University and Polytechnic Hospital La Fe, Valencia, Spain. Central Michigan University, Detroit, Michigan, USA. Faculty of Medicine, University of Geneva, Geneva, Switzerland. Hematology/Oncology Clinic, University of California at Davis, Sacramento, California, USA. Cliniques Saint-Luc, Université Catholique de Louvain, Brussels, Belgium. Oklahoma Bleeding and Clotting Disorders Center at OU Health, Oklahoma City, Oklahoma, USA. Section of Hematology/Oncology, Tulane University School of Medicine, New Orleans, Louisiana, USA. Orthopaedic Hemophilia Treatment Center, Los Angeles, California, USA. Hemophilia Comprehensive Care Center, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa. Goethe University Hospital, Frankfurt, Germany. Lakeview Hospital, Benoni, Gauteng, South Africa. Edouard Herriot University Hospital, Lyon, France. American Thrombosis and Hemostasis Network, Rochester, New York, USA. Oregon Health & Science University, Portland, Oregon, USA. Haemophilia Treatment Centre, University Hospital Montpellier, Montpellier, France. Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA. Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, Georgia, USA. Christian Medical College, Vellore, Tamil Nadu, India. Institute of Blood Pathology and Transfusion Medicine, Lviv, Ukraine. National Specialized Children's Hospital Okhmatdyt, Kyiv, Ukraine. Hemophilia and Thrombosis Center, University of Colorado, Aurora, Colorado, USA. Children's Hospital Los Angeles, Los Angeles, California, USA. Keck School of Medicine, University of Southern California, Los Angeles, California, USA. Aoede Associates, Athens, Texas, USA. LFB-USA, Inc., Framingham, Massachusetts, USA. HEMA Biologics, LLC, Louisville, Kentucky, USA. GLOVAL LLC, Broomfield, Colorado, USA. Lombardi Comprehensive Cancer Center, Georgetown University Medical Center, Washington, District of Columbia, USA.

Haemophilia : the official journal of the World Federation of Hemophilia. 2021
INTRODUCTION Haemophilia patients with inhibitors often require a bypassing agent (BPA) for bleeding episode management. Eptacog beta (EB) is a new FDA-approved recombinant activated human factor VII BPA for the treatment and control of bleeding in haemophilia A or B patients with inhibitors (≥12 years of age). We describe here the EB safety profile from the three prospective Phase 3 clinical trials performed to date. AIM: To assess EB safety, immunogenicity and thrombotic potential in children and adults who received EB for treatment of bleeding and perioperative care. METHODS Using a randomized crossover design, 27 subjects in PERSEPT 1 (12-54 years) and 25 subjects in PERSEPT 2 (1-11 years) treated bleeding episodes with 75 or 225 μg/kg EB initially followed by 75 μg/kg dosing at predefined intervals as determined by clinical response. Twelve PERSEPT 3 subjects (2-56 years) received an initial preoperative infusion of 75 μg/kg (minor procedures) or 200 μg/kg EB (major surgeries) with subsequent 75 μg/kg doses administered intraoperatively and post-operatively as indicated. Descriptive statistics were used for data analyses. RESULTS Sixty subjects who received 3388 EB doses in three trials were evaluated. EB was well tolerated, with no allergic, hypersensitivity, anaphylactic or thrombotic events reported and no neutralizing anti-EB antibodies detected. A death occurred during PERSEPT 3 and was determined to be unlikely related to EB treatment by the data monitoring committee. CONCLUSION Results from all three Phase 3 trials establish an excellent safety profile of EB in haemophilia A or B patients with inhibitors for treatment of bleeding and perioperative use.
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Language : eng
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