Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children: XTEND-ed Second Interim Analysis

Hämostaseologie 2026

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Authors: Lynn M. Malec, Christoph Königs, Amy L. Dunn, M Carcao, Bülent Zülfikar, Rubén Berrueco, Simon A Brown, Umer Khan, Sriya W. Gunawardena, Graham Neill, Lydia Abad‐Franch, Linda Bystrická
+1 more Sophie Susen
DOI: 10.1055/s-0045-1814564
Cited by: 0

Abstract

Introduction Efanesoctocog alfa is a first-in-class high sustained factor VIII replacement therapy that overcomes the von Willebrand factor-imposed half-life ceiling. In XTEND-Kids (NCT04759131), once-weekly efanesoctocog alfa (50 IU/kg) was well tolerated and provided highly effective bleed protection in children with previously treated severe hemophilia A. Participants completing XTEND-Kids could enter XTEND-ed (NCT04644575). Here, we report on the efficacy of efanesoctocog alfa for treating bleeding episodes (BEs) in children at interim analysis 2 of XTEND-ed (datacut: February 22, 2024). Method Participants provided informed consent; the study was approved by applicable review boards. Children aged

Fields of Study & Subjects

Hemophilia Treatment and Research Platelet Disorders and Treatments Coagulation Bradykinin Polyphosphates and Angioedema

Publication details

Journal / source
Hämostaseologie
Volume
46
Issue
S 01
Pages
S47–S48
Publication date
1 February 2026
Language
DE