A prospective Crossover Triple-blind Controlled Trial on the Safety and Efficacy of Iranian Recombinant FVIII (Safacto) versus Plasma Derived FVIII A pilot study

Abstract

Background: Considering the increasing number of patients with hemophilia and infrastructure requirements for a comprehensive approach, development of a recombinant factor has become a milestone. The objective of this study was to assess the safety, efficacy and non inferiority of Safacto (Recombinant factor VIII) compared with plasma-derived factor in the treatment of hemophilia A. Methods: 10 patients with severe hemophilia A were enrolled in this study. Each patient was treated by a 40-50 IU/kg infusion of either plasma derived or recombinant factor VIII after initiation of each of 4 consecutive hemarthrosis episodes in a triple-blind prospective crossover permuted block randomizing method. Clinical efficacy scale score and in vivo recovery of factor VIII was assessed in each of the treated bleeding episodes. Any adverse event was also recorded. Results: The mean±SD level of factor VIII in the plasma versus recombinant groups was 111.5±39 and 115±39, respectively without any significant difference. Response scaling method which assessed pain and range of motion revealed equalized scores along with in vivo recovery, hence treatment success rate was comparable in both groups. One non-recurring, mild skin rash reaction occurred simultaneous with the administration of plasma derived factor. Conclusion: Safacto (r-FVIII) is safe and effective and non-inferior to plasma derived factor VIII in the treatment of hemophilia A related bleeding events.