biostudies-literatureUnknown28(4)Pipe SWLFB SA<h4>Introduction</h4>Eptacog beta is a new recombinant activated human factor VII bypassing agent approved in the United States for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors 12 years of age or older.<h4>Aim</h4>To prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children <12 years of age with haemophilia A or B with inhibitors.<h4>Methods</h4>Using a randomised crossover design, subjects received initial doses of 75 or 225 μg/kg eptacog beta followed by 75 μg/kg dosing at predefined intervals (as determined by clinical response) to treat bleeding episodes (BEs). Treatment success criteria included a haemostasis evaluation of 'excellent' or 'good' without use of additionHaemophilia : the official journal of the World Federation of Hemophilia548-556https://www.ebi.ac.uk/biostudies/studies/S-EPMC9542908biostudies-literatureEptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.PMC9542908Macie CMeeks SLWilkinson TAYoung GCastaman GPipe SWNowak-Gottl UShapiro ADMartinez LVWang MRecht MHermans CEscobar MMahlangu JWindyga JCarcao MSauty ENegrier CChrisentery-Singleton TVilchevska KVMitchell ISChitlur MJourneycake JMitha IHAlexander WADavis JADucore JDunn ALBonzo DKhan OStasyshyn OfalseEptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors.<h4>Introduction</h4>Eptacog beta is a new recombinant activated human factor VII bypassing agent approved in the United States for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors 12 years of age or older.<h4>Aim</h4>To prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children <12 years of age with haemophilia A or B with inhibitors.<h4>Methods</h4>Using a randomised crossover design, subjects received initial doses of 75 or 225 μg/kg eptacog beta followed by 75 μg/kg dosing at predefined intervals (as determined by clinical response) to treat bleeding episodes (BEs). Treatment success criteria included a haemostasis evaluation of 'excellent' or 'good' without use of addition2022-01-01T00:00:00Z2022 Jul2026-05-31T16:08:11.946Z2025-02-19T01:28:29.656ZS-EPMC95429083547530810.1111/hae.14563