Infant and toddlers with Severe Hemophilia A with Inhibitor on
Prophylaxis with Emicizumab
Emicizumab is a recombinant, humanized, and bispecific monoclonal
antibody that bridges activated factor (F) IX and FX in place of FVIII
to restore hemostasis in persons with hemophilia A (PHA). Data on the
efficacy and safety of emicizumab in young children is limited.
Immunologic naivety, physiologically decreased production of vitamin K
dependent proteins, specifically FIX, and enhanced clearance of
emicizumab in infants may support decreased emicizumab effectiveness. We
report on the facilitation of care rendered by using emicizumab in young
PHA with inhibitors and extend data on the efficacy and safety in PHA
< 3 years.