Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity \< 1%, bleeding may frequently occur spontaneously, most commonly in joints, leading to painful hemophilic arthropathy and loss of joint function. Patients with moderate or mild hemophilia A, defined as FVIII clotting activity between 1-\<5% and 5-40% respectively, are less likely to have spontaneous bleeding however can have significant bleeding with trauma or surgery. Perioperative management by a hematologist who specializes in hemophilia is needed to ensure hemostasis during surgery. Hemophilia is an X-linked recessive disorder affecting 1 in 5000 to 10,000 males.