Approaches towards successful home treatment in patients with inhibitors

Significant advances have been achieved in prevention of haemophilic disabi lity through prophylactic administration of concentrates and early administ ration of coagulation factors to control new bleeding episodes, but there i s only limited experience with home treatment in haemophilia patients with inhibitors. A home treatment programme using recombinant activated factor V II (rFVIIa; NovoSeven(R), Novo Nordisk, Bagsvaerd, Denmark) in early interv ention against minor bleeds in patients with high responding inhibitors was initiated in Denmark in June 1994. Following careful education and instruc tion, 2-3 doses each giving 90-100 mu g/kg bodyweight of rFVIIa were stored in each patient's home. At the onset of a new bleeding episode patients we re instructed to inject 1 dose of rFVIIa, and to call the Haemophilia Centr e to discuss further management of the episode. If the drug was not complet ely effective after 1-2 h, a second dose was injected after 3 h. Patients w ere further instructed to contact us the following day for final efficacy r eporting. In total, 7 patients have been enrolled into the study, and to da te 114 bleeding episodes have been managed at home with a mean of 2.1 doses per bleed. On 4 occasions, recurrence of bleeding was noted within 24 h. H ospital admission was required in 9 cases, because of a serious injury, ins ufficient compliance or, in 2 cases, because bleeding required prolonged tr eatment. Management of these bleeding episodes required a mean of 18 doses. We propose and discuss key criteria for selection of patients for a home t reatment programme. The results of this study demonstrate that early interv ention in the home setting with rFVIIa is safe and effective in the managem ent of minor bleeding episodes in haemophilia patients with inhibitors.