Recombinant Factor VIIa Concentrate versus Plasma-derived Concentrates for the Treatment of Acute Bleeding Episodes in Persons with Haemophilia and Inhibitors

The development of inhibitors remains the most challenging complication of treatment in persons with haemophilia, resulting in increased morbidity and a significant economic burden. The ultimate goal of treatment in patients with inhibitors is immune tolerance induction (ITI) therapy; however, during the induction phase of ITI, when ITI fails and where ITI is not affordable, the treatment of bleeding becomes a crucial issue. Recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) have been developed to bypass the inhibitor antibody effect and have been tested in several randomised controlled trials, including two crossover head-to-head comparisons. Two systematic reviews of the literature have appraised and synthesised the available evidence. The recombinant drug seems to provide a more favourable benefit–risk ratio and may be easily administered as a single front-loaded bolus, making it a good candidate for the role of first-line treatment for bleeding in patients with inhibitors. Aggressive treatment of acute bleeds should be considered, including the use of higher and repeated-dose regimens until complete resolution of the bleed.