Long-term safety and efficacy of recombinant activated factor VII (NovoSeven^®) in haemophilia patients with inhibitors : interim post marketing study analysis of 5 patient exposure years

Abstract

This article reviews an interim analysis of the long-term safety and efficacy of recombinant activated factor VII (rFVIIa) over a 5-year period. Episodes of 1,580 bleedings were recorded in 102 patients with either congenital or acquired hemophilia. An overall efficacy rate in achieving excellent or good hemostasis within a 12-hour period was 69.6%. An excellent efficacy achieved within ≤ 8 hours was almost doubled as compared to the results in a previously reported Japanese clinical trial (60.9 vs. 31.2%).
Furthermore, the efficacy rate for rFVIIa was significantly higher in thepatient group, which met all three recommended practice requirements, i.e., initial dose ≥ 90 ug/kg; time from the onset of hemorrhage to initial dose ≤ 3 hours; and mean dosing interval ≤ 3 hours. This group of patients demonstrated an 82.4% efficacy rate compared with the non-optimal practice group, which had an efficacy rate of 44.4%. However, only 40% of bleeding episodes in the patients were treated according to the recommended practices. Compliance with treatment remains a challenge that needs to be addressed in order to achieve improved efficacy. Forty two adverse drug reactions were reported in 20 patients including 4 severe ones in 3 patients, but none of them were thought to be related to the treatment with rFVIIa.