title: Prophylactic recombinant factor VIIa in haemophilia patients with inhibitors.
abstract: Prevention of bleeding, especially into joints, with prophylactic factor infusions is the most effective treatment for severe haemophilia patients. Approximately 15-30% of patients with factor VIII deficiency and 3-5% of patients with factor IX deficiency develop neutralizing antibodies (inhibitors) to factor precluding their use. Such patients often have significant bleeding complications including life- and limb-threatening bleeds and severe joint disease. Prophylaxis for such patients is not generally considered because of the fact that the standard (bypassing) agents for such patients are not as effective as natural factor replacement, because of concerns for thrombotic complications and also because of the very high cost of bypassing agents. We treated two patients with high titre inhibitors with prophylactic recombinant factor VIIa (rFVIIa). The first patient was treated as a result of development of a target joint and to reduce the use of agents that can lead to anamnesis of his inhibitor. The second patient had multiple severe bleeds and was hospitalized 20% of the time over a 2-year period. He had a very poor quality of life. Both patients had shown good responses previously to rFVIIa for treatment of bleeds. Both patients had an outstanding response to prophylaxis albeit at a very high cost. Prophylaxis with rFVIIa can be an effective approach in select inhibitor patients with severe complications related to bleeding.
authors.affiliation: Department of Pediatric Hematology, Children's Hospital of Orange County, Orange, CA 92868, USA. gyoung@choc.org
authors.forename: G
authors.initials: G
authors.lastname: Young
authors.forename: M
authors.initials: M
authors.lastname: McDaniel
authors.forename: D J
authors.initials: DJ
authors.lastname: Nugent
mesh_terms: D000293:Adolescent; D001323:Autoantibodies / Q000276:immunology; D002675:Child, Preschool; D005167:Factor VII / Q000627:therapeutic use*; D005169:Factor VIII / Q000032:analysis; D015942:Factor VIIa; D006395:Hemarthrosis / Q000517:prevention & control; D006467:Hemophilia A / Q000188:drug therapy* / Q000276:immunology; D002836:Hemophilia B / Q000188:drug therapy* / Q000276:immunology; D006470:Hemorrhage / Q000276:immunology / Q000517:prevention & control*; D006801:Humans; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use*; D012189:Retrospective Studies
chemical_list: D001323:Autoantibodies; D011994:Recombinant Proteins; D005167:Factor VII; D005169:Factor VIII; C103587:recombinant FVIIa; D015942:Factor VIIa