The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors
Year: 2021
Type: article
Abstract: 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.
Source: Haemophilia
DOI: 10.1111/hae.14419
Authors: Miguel Escobar, Giancarlo Castaman, Santiago Bonanad Boix, Michael Callaghan, Philippe de Moerloose, Jonathan Ducore, Cédric Hermans, Janna Journeycake, Cindy Leissinger, James Luck, Johnny Mahlangu, Wolfgang Miesbach, Ismail Haroon Mitha, Claude Négrier, Doris Quon, Michael Recht, Jean François Schved, Amy D. Shapiro, Robert Sidonio, Alok Srivastava, Oleksandra Stasyshyn, Kateryna V. Vilchevska, Michael Wang, Guy Young, W. Allan Alexander, Ahmad Al‐Sabbagh, Daniel Bonzo, Christopher Macie, Thomas A. Wilkinson, Craig Kessler
Institutions: Azienda Ospedaliero-Universitaria Careggi, Hospital Universitari i Politècnic La Fe, Central Michigan University, University of Geneva, University of California, Davis, Cliniques Universitaires Saint-Luc, UCLouvain, OU Health, University of Oklahoma Health Sciences Center, Tulane University, National Health Laboratory Service, University of the Witwatersrand, University Hospital Frankfurt, Hôpital Edouard Herriot, Oregon Health & Science University, American Thrombosis and Hemostasis Network, Centre Hospitalier Universitaire de Montpellier, Indiana Hemophilia and Thrombosis Center, Emory University, Christian Medical College, Vellore, Institute of Blood Pathology and Transfusion Medicine of the National Academy of Medical Sciences of Ukraine, University of Colorado Anschutz, University of Southern California, Children's Hospital Los Angeles, LFB (United States), Kentucky Educational Television, Georgetown University Medical Center, Georgetown Lombardi Comprehensive Cancer Center
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W3207597418