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.