Key clinical point: Emicizumab (Hemlibra) is well tolerated and has substantial, clinically meaningful efficacy in pediatric patients with hemophilia A and factor VIII inhibitors.
Major finding: Among those receiving once-weekly emicizumab prophylaxis, 77% had no treated bleeding events and 100% of evaluable target joints resolved. Emicizumab resulted in a 99% reduction in bleeding rate versus previous bypassing agent prophylaxis.
Study details: Analysis of 88 male pediatric patients with congenital hemophilia A enrolled in HAVEN 2, an ongoing phase 3 multicenter, nonrandomized, open-label study.
Disclosures: Study authors reported disclosures related to Alnylam, Bayer, Bioverativ, CSL Behring, Genentech/Roche, Grifols, Kedrion, Novo Nordisk, Shire, Spark, and uniQure, among others.
Young G, et al. Blood. 2019 Oct 10; doi: 10.1182/blood.2019001869