Evidence Supporting the Use of Recombinant Activated Factor VII in Congenital Bleeding Disorders

Abstract

Recombinant activated factor VII ([rFVIIa] NovoSeven) was introduced in 1996 for the treatment of haemophilia patients with inhibitors (HPIs) to factor VIII or IX. This article reviews the evidence for the use of rFVIIa in congenital bleeding disorders. English-language databases were searched in September 2009 for reports of randomised controlled trials (RCTs) evaluating the effect of rFVIIa on haemostasis in congenital bleeding disorders. Eight RCTs comprising 256 HPIs were identified. The evidence for the use of rFVIIa in HPIs in terms of dose, clinical setting, modes of administration, efficacy and adverse events was weak, given the limited number of patients included and the heterogeneity of the RCTs. Overall, the haemostatic efficacy of rFVIIa varied from 25 to 100% in the studies reviewed; <1% of the patients receiving rFVIIa developed a thromboembolic adverse event. The authors suggest that the addition FVIIa therapy to HPIs should be based on the patient’s ability to generate thrombin and form a clot, rather than being based on weight alone. Assays reflecting thrombin generation, such as whole-blood thrombelastography, have the potential to significantly improve the treatment of these patients.