Recombinant Factor VIIa in the Treatment of Bleeding in Hemophilic Children with Inhibitors

Information contained in this review of the use of recombinant factor VIIa (rFVIIa, NovoSeven) in the pediatric population was obtained through review of literature and abstraction of data available at Novo Nordisk. Pharmacokinetic investigations in hemophilic children suggest that rFVIIa may have a shorter half-life and more rapid clearance in the pediatric population than in adults, with resultant implications for dosing. Analysis of pediatric data from various studies and case reports indicates that rFVIIa is safe and effective for the control of spontaneous or surgical bleeding in hemophilic children with inhibitors, including central nervous system bleeds, and bleeding episodes during immune tolerance induction therapy. The very young pediatric population (less than 1 year of age) poses specific difficulties in treatment, and rFVIIa provides an effective alternative to other therapeutic modalities. In some hemophilia B patients with inhibitors, anaphylactic reaction to FIX infusions is a potentially life-threatening problem, for which rFVIIa may provide the only safe alternative therapy for the control of bleeding episodes. Continuous infusion of rFVIIa has also been used effectively in individual cases. Optimal dosing regimens, both intermittent and continuous, still need to be determined in children.