Recombinant factor VIIa versus aPCCs in haemophiliacs with inhibitors: treatment and cost considerations

For haemophiliacs with factor VIII inhibitor titres greater than approximately 5 Bethesda Units, haemostasis is best achieved with activated prothrombin complex concentrates (aPCCs, AUTOPLEX®T and FEIBA®) and the recently approved recombinant factor VIIa (rFVIIa). The optimal treatment should be determined by consideration of efficacy, safety, availability, cost and convenience. The current evidence does not allow definitive conclusions to be drawn regarding the relative efficacy of aPCCs and rFVIIa. The principal safety concerns are transmission of viral or other pathogens, thrombogenicity and immunogenicity. The two aPCCs are derived from human plasma and therefore carry the potential for transmitting viruses. This risk is negligible for rFVIIa, although this product could theoretically transmit animal pathogens. The aPCCs are clinically safe in most applications. Likewise, rFVIIa should be considered potentially thrombogenic, though with a low risk. The aPCCs contain detectable factor VIII and can therefore be immunogenic, but in practice this rarely affects their efficacy. The costs of single doses of aPCCs and rFVIIa are similar, but a treatment course of rFVIIa typically requires more doses. Treatment with rFVIIa may be more cumbersome owing to the requirement for more frequent administration. In addition, as for any product for which there is only a single supplier, availability could be vulnerable in some circumstances.