ASH 2021 | Eptacog beta efficacy in children and adolescents with hemophilia A or B with inhibitors

Eptacog beta is a recombinant coagulation factor VIIa molecule recently approved as an on-demand treatment to control bleeds in patients aged 12 or older with hemophilia A or B, with inhibitors. Data from the pivotal Phase III PERSEPT 1 study (NCT02020369) led to the approval after showing eptacog beta to effectively control most mild to moderate bleeding episodes. The PERSEPT 2 study (NCT02448680) conducted in pediatric patients suggested eptacog beta may be less efficacious in children under age 12. Guy Young, MD, Children’s Hospital Los Angeles & Keck School of Medicine, University of Southern California, Los Angeles, CA, discusses an evaluation of the clinical responses to eptacog beta in children across three age subgroups within PERSEPT 1 and 2. Treatment was shown to be highly successful, with bleeding event treatment success of 58%, 72%, and 93%, in the 0–6-, 6–12-, and 12–18-year subgroups, respectively. It was shown that the number of doses used and the duration of treatment increased with decreasing age. These observations are consistent with the recognized challenges in determining bleeding episode resolution in children by parents or caregivers. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.