Early treatment with recombinant factor VIIa results in greater efficacy with less product.

Abstract

Early treatment of bleeding episodes in haemophilia patients offers advantages over later treatment, including minimizing the damage caused by the haemorrhage and reducing the amount of product needed to control it. Self-administration at home also offers greater convenience and time and cost savings for both patient and physician. We compared the amount of recombinant activated factor VII (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark) used in the treatment of peripheral intramuscular haemorrhages in people with haemophilia A and B with inhibitors and in those with acquired haemophilia, in the compassionate use, dose-finding and US home treatment studies. We also compared the response rates in each of the 3 studies. In the compassionate use setting, in which the mean time from onset of bleeding to initiation of treatment with rFVIIa was 5 d, the mean number of doses given per bleeding episode was 13.6, with 63.1% of episodes without compartment syndrome and 73% with tense muscle/compartment syndrome having an excellent or effective response. In the dose-finding study, average time from onset of bleeding to treatment with rFVIIa was 9 h. The average number of doses given was 3.55, and 72% in the high-dose arm were judged to have an excellent or effective response. In the US home treatment study in which bleeding episodes were treated earlier (mean 1.2 h from onset of bleeding), the mean number of doses given was 2.3, and 92% had an effective response to treatment. These findings indicate that early treatment with rFVIIa has a greater success rate, with fewer doses being required. Home treatment results in cost savings, greater convenience, and less morbidity.