Treatment of Inhibitors in Hemophilia

Recent Topics in Inhibitor Bypassing Coagulation Products

Abstract

Marked advances have been made in hemostatic treatment for hemophilia during the last20 years, owing to a stable supply of safe and effective coagulation factor concentrates. On the other hand, the treatment results in patients who developed inhibitors, particularly high responder type inhibitors after the initiation of the injection of coagulation factor concentrates are considerably poorer than those in inhibitor-negative patients. Therefore, hemostatic treatment in inhibitor-positive patients is a majorproblem in the treatment of hemophilia. To improvethissituation, various approaches have been attempted. We introduce recent topics on inhibitor bypassing agents as follows. 1. Long-term safety and efficacy of rFVIIa (NovoSeven®) in hemophilia patients with inhibitors : Interim marketing study in Japan. 2. Clinical studies for single/highdose treatment of rFVIIa. 3. Clinical studies comparingrFVIIa to APCC. 4. Prophylactic treatment with rFVIIa or APCC. 5. New inhibitor bypassing drugs under clinical investigation.