Real-World Experience with Eptacog Beta for On-Label and Off-Label Indications: The Spanish Experience.

Background: Eptacog beta is a novel recombinant activated factor VII (rFVIIa). Preclinical studies have shown the product has a similar profile to eptacog alfa. The PERSEPT 1 and PERSEPT 3 trials proved the efficacy and safety of eptacog beta for surgical prophylaxis and treatment of bleed in patients with haemophilia A or B with inhibitors. This led to approval of the product by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Recently, real-world data have been published on the use of eptacog beta on this population which has provided positive outcomes. Given the similar in vitro and in vivo profile of eptacog beta and eptacog alfa positive outcomes could be expected not only in patients with haemophilia and inhibitors, but also in other situations where eptacog alfa is a therapeutic option. Methods: We report on the use of this product for on-label and off-label populations in two Spanish hospitals. Results: We describe four cases in an on-label population (haemophilia A with inhibitors) and four cases in another population (one with acquired haemophilia and three with mild factor VII deficiency). Conclusions: Our experience provides further evidence of the efficacy and safety of eptacog beta for surgical prophylaxis and treatment of bleeding in patients with haemophilia A with inhibitors, but also in those with factor VII deficiency. To our knowledge, this is the first report to describe the use of eptacog beta for factor VII deficiency and acquired haemophilia.