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

Abstract

Background: The 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. Methods: ACQUI-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. Results: A 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. Conclusions: These 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.

Keywords

• Acquired haemophilia A
• Bleeding episode
• Bypassing agents
• Daily-use rFVIIa
• Observational
• Prospective