HOME TREATMENT WITH RECOMBINANT ACTIVATED FACTOR-VII - RESULTS FROM ONE-CENTER

Haemophilia patients developing an inhibitor against factor VIII (FVII I) or FIX require alternative treatment for the management of their bl eeding, rather than standard procedures. In patients with low titre in hibitors, increased doses of FVIII or FIX may improve haemostasis. If a higher titre is present, a porcine FVIII concentrate may be efficaci ous in selected cases. 'Bypassing' agents, such as low purity FIX conc entrates, or activated or unactivated concentrates of prothrombin comp lex may also be useful in inhibitor patients. An activated factor VII molecule (rFVIIa; NovoSeven, Novo Nordisk, Bagsvaerd, Denmark), has be en produced by recombinant DNA cell technology. In June 1994, we estab lished a home treatment programme with rFVIIa for five inhibitor patie nts to study its efficacy in the early intervention of bleeding episod es; our results from the first 3 years are presented. Self-treatment i n the home took place in 50 instances of acute minor bleeding. A mean of 2.02 doses of rFVIIa (each dose of about 90 mu g/kg bodyweight) was required to arrest bleeding (range 1-4 doses). Four bleeding episodes required in-hospital management either because the acute condition wa s caused by severe trauma, or because treatment had not been institute d in the early phase of bleeding. Here, the range of rFVIIa doses was 4-37. For comparison, we also report 36 minor bleeding episodes manage d 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 m uscle and soft tissue bleeds. In conclusion, we feel that our home tre atment results strongly suggest that early intervention by home treatm ent with rFVIIa in acute minor bleeding is efficacious and cost effect ive. Blood Coag Fibrinol 9 (suppl 1):S107S110 (C) 1998 Lippincott-Rave n Publishers.