Home treatment with recombinant activated factor VII in patients with factor VIII inhibitors: the advantages of early intervention

To evaluate the feasibility, efficacy and safety of home treatment with recombinant activated factor VII (rFVIIa), 10 inhibitor patients (all haemophiliacs except one acquired post-partum) self-administered up to four doses of 90 mu g/kg rFVIIa every 3 +/- 1 h. The response was rated by the patient as effective (haemorrhage stopped or decreased substantially), partially effective (reduced) or ineffective (unchanged or worsened). 45 haemarthroses and eight haematomas were treated within a median time of 1.0 h (range 0.3-11.9) from the onset of bleeding, with a median of two rFVIIa doses per course (range 1-4). rFVIIa was effective in 42 episodes (79%), partially effective in six (11%) and failed in five (10%). Compared with partially effective and ineffective treatments, effective treatments started earlier (median time: 0.6 v 2.7 h, P = 0.02) and required a smaller number of doses (median: 1.5 v 3, P = 0.007). The risk of a partially effective or ineffective treatment was smaller for treatments started within 6 h from the onset of bleeding than for those which started later (OR 0.24, 95% CI 0.09-0.63). Mild side-effects were reported only after 3/113 self-infusions (2.6%). Early home treatment with rFVIIa is safe, feasible and effective, inducing and maintaining haemostasis with a small number of doses.