Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A.
Lynn MalecFlora PeyvandiAnthony K C ChanChristoph KönigsBulent ZulfikarHuixing YuanMindy SimpsonMaria Teresa Álvarez RománManuel CarcaoJanice M StaberAmy L DunnSheng-Chieh ChouRoseline d'OironManuela AlbisettiMarek DemissieElena SantagostinoAbhimanyu YarramaneniNancy WongLydia Abad-FranchSriya GunawardenaKarin Fijnvandraatnull nullPublished in: The New England journal of medicine (2024)
In children with severe hemophilia A, once-weekly prophylaxis with efanesoctocog alfa provided high sustained factor VIII activity in the normal to near-normal range (>40 IU per deciliter) for 3 days and more than 10 IU per deciliter for almost 7 days after administration, leading to effective bleeding prevention. Efanesoctocog alfa was associated with mainly nonserious adverse events. (Funded by Sanofi and Sobi; XTEND-Kids ClinicalTrials.gov number, NCT04759131.).