Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors. Issue 4 (27th April 2022)

• Record Type: Journal Article
• Authors: Pipe, Steven W.; Hermans, Cédric; Chitlur, Meera; Carcao, Manuel; Castaman, Giancarlo; Davis, Joanna A.; Ducore, Jonathan; Dunn, Amy L.; Escobar, Miguel; Journeycake, Janna; Khan, Osman; Mahlangu, Johnny; Meeks, Shannon L.; Mitha, Ismail Haroon; Négrier, Claude; Nowak‐Göttl, Ulrike; Recht, Michael; Chrisentery‐Singleton, Tammuella; Stasyshyn, Oleksandra; Vilchevska, Kateryna V.; Martinez, Laura Villarreal; Wang, Michael; Windyga, Jerzy; Young, Guy; Alexander, W. Allan; Bonzo, Daniel; Macie, Christopher; Mitchell, Ian S.; Sauty, Evelyne; Wilkinson, Thomas A.; Shapiro, Amy D.;
• Is Part Of: Haemophilia. Volume 28:Issue 4(2022)
• Page Start: 548
• Page End: 556
• Publication Date: 2022-04-27
• Subjects: eptacog beta -- haemophilia -- inhibitors -- PERSEPT -- paediatric -- recombinant FVIIa; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.14563
• Languages: English
• ISSNs: 1351-8216

Abstract

Abstract: Introduction: 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. Aim: To prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children &lt;12 years of age with haemophilia A or B with inhibitors. Methods: 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 &#39;excellent&#39; or &#39;good&#39; without use of additional eptacog beta, alternative haemostatic agent or blood product, and no increase in pain following the first &#39;excellent&#39; or &#39;good&#39; assessment. Results: Treatment success proportions in 25 subjects (1–11 years) who experienced 546 mild or moderate BEs were 65% in the 75 μg/kg initial dose regimen (IDR) and 60% in the 225 μg/kg IDR 12 h following initial eptacog beta infusion. By 24 h, the treatment success proportions were 97% for the 75 μg/kg IDR and 98% for the 225 μg/kg IDR. No thrombotic events, allergic reactions, neutralising antibodies or treatment‐related adverse events were reported. Conclusion: Both 75 and 225 μg/kg eptacog beta IDRs provided safe and effective treatment and control of bleeding in children &lt;12 years of age.