Home treatment with recombinant activated factor VII: results from one centre

Haemophilia patients developing an inhibitor against factor VIII (FVIII) or FIX require alternative treatment for the management of their bleeding, rather than standard procedures. In patients with low titre inhibitors, increased doses of FVIII or FIX may improve haemostasis. If a higher titre is present, a porcine FVIII concentrate may be efficacious in selected cases. 'Bypassing' agents, such as low purity FIX concentrates, or activated or unactivated concentrates of prothrombin complex may also be useful in inhibitor patients. An activated factor VII molecule (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark), has been produced by recombinant DNA cell technology. In June 1994, we established a home treatment programme with rFVIIa for five inhibitor patients to study its efficacy in the early intervention of bleeding episodes; our results from the first 3 years are presented. Self-treatment in the home took place in 50 instances of acute minor bleeding. A mean of 2.02 doses of rFVIIa (each dose of about 90 microg/kg bodyweight) was required to arrest bleeding (range 1-4 doses). Four bleeding episodes required in-hospital management either because the acute condition was caused by severe trauma, or because treatment had not been instituted in the early phase of bleeding. Here, the range of rFVIIa doses was 4-37. For comparison, we also report 36 minor bleeding episodes managed in-hospital in three of these five patients who participated in the Compassionate Use Study where home treatment was not permitted. These bleeding episodes required considerably more administrations of rFVIIa with a mean consumption of 8 doses in joint bleeds and 9.5 doses in muscle and soft tissue bleeds. In conclusion, we feel that our home treatment results strongly suggest that early intervention by home treatment with rFVIIa in acute minor bleeding is efficacious and cost effective.