Adaptation of recombinant activated factor VII in the treatment of acquired haemophilia A: Results from a prospective study (ACQUI-7) in France

Abstract

BackgroundThe efficacy and safety of recombinant activated factor VII (rFVIIa; NovoSeven®) in patients with acquired haemophilia A (AHA) are established; however, data on daily use in clinical practice for bleeding episodes are limited. The ACQUI-7 study aimed to provide additional data on managing bleeding episodes with rFVIIa in patients with AHA. MethodsACQUI-7, a prospective, observational, multicentre study in 20 sites in France, recruited patients from 2010 to 2013. Treatment was by physician’s judgement and therapeutic practice at each site. Inclusion criteria: anti-factor VIII (FVIII) auto-antibodies ​> ​1 Bethesda Unit, FVIII activity ​< ​50% and bleeding episodes treated with rFVIIa. Data collected included description of patient characteristics, bleeding episodes leading to haemostatic treatment and rFVIIa therapy. ResultsA total of 27 patients with 27 bleeding episodes (24 [88.9%] severe) were treated with first-line rFVIIa. Most (81.5%) were affected in more than 1 site (44.4% in muscle). Nineteen patients (70.4%) were treated for up to 5 days; 24 (88.9%) had their bleeding episode controlled (including 21 severe bleeds). The maximum daily dose for controlling severe bleeds decreased from 900 ​μg/kg/24 ​h on day 1–630 ​μg/kg/24 ​h on day 5, with a corresponding reduction in maximum number of daily injections from 10 to 7. The use of rFVIIa in this population did not raise any safety concerns. ConclusionsThese real-world data confirm the effectiveness and safety of rFVIIa and provide additional information on the daily use of rFVIIa to support the management of patients with AHA.