Evidence-based use of recombinant FVIIa (NovoSeven, NiaStase) for the treatment of hemophilia with inhibitors in children and adolescents.
Children and adolescents comprise a significant proportion of the hemophilia population, including those patients who have developed inhibitors to factor VIII or FIX. We examine the use of rFVIIa for the treatment of bleeding episodes and the prevention of bleeding in children and adolescents with hemophilia A and B with inhibitors, focusing on registry data and recent clinical trial results. Based on this review of the literature, we conclude that recombinant FVIIa is safe and effective for use in controlling bleeding in these patient populations.
Goldstein
Brahm
B
Novo Nordisk Inc., 100 College Road West, Princeton, NJ, United States. bgol@novonordisk.com
Geldziler
Brian
B
Bjerre
Jens
J
Seremetis
Stephanie
S
D000293:Adolescent; D002648:Child; D019317:Evidence-Based Medicine; D005164:Factor IX / Q000627:therapeutic use*; D005169:Factor VIII / Q000627:therapeutic use*; D015942:Factor VIIa / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D002836:Hemophilia B / Q000188:drug therapy*; D006470:Hemorrhage / Q000188:drug therapy / Q000517:prevention & control; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use
D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX; C103587:recombinant FVIIa; D015942:Factor VIIa