title: Safety, efficacy and cost-effectiveness of home therapy with recombinant activated factor VII in a patient with severe haemophilia A and an anti-factor VIII inhibitor.
abstract: We report the use of recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) in a patient with severe haemophilia A and a high-titre anti-factor VIII inhibitor. A home therapy protocol was devised and its cost and efficacy were assessed after 2 years. rFVIIa (90 microg/kg) was self-administered at the onset of symptoms of bleeding. Doses were subsequently repeated at 2-h intervals until adequate haemostasis was achieved. The patient experienced a total of 25 bleeding episodes over the 2-year period, of which 23 were treated with up to three doses of rFVIIa. On two occasions, there was a recurrence of bleeding in the same area within 24 h, necessitating retreatment. There was a reduction in the number of inpatient hospital days compared with the 2 years before the use of rFVIIa and, overall, the cost of rFVIIa treatment was less than that of the previously used activated prothrombin complex concentrate (FEIBA; Immuno Ltd, Vienna, Austria). Our experience suggests that in selected haemophiliacs with inhibitors, home treatment with rFVIIa may be safe and cost-effective.
authors.affiliation: Department of Haematology, Royal Infirmary of Edinburgh, UK.
authors.forename: A J
authors.initials: AJ
authors.lastname: Stewart
authors.forename: J P
authors.initials: JP
authors.lastname: Hanley
authors.forename: C A
authors.initials: CA
authors.lastname: Ludlam
mesh_terms: D003362:Cost-Benefit Analysis; D003718:Denmark; D004305:Dose-Response Relationship, Drug; D005169:Factor VIII / Q000037:antagonists & inhibitors*; D015942:Factor VIIa / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006699:Home Care Services; D006801:Humans; D007223:Infant; D008297:Male; D011994:Recombinant Proteins / Q000009:adverse effects / Q000191:economics / Q000627:therapeutic use
chemical_list: D011994:Recombinant Proteins; D005169:Factor VIII; D015942:Factor VIIa