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

Abstract

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 μg/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.