Abstract
A multi-center post-marketing surveillance study was conducted in previously treated patients with hemophilia A in order to evaluate the safety and efficacy of a recombinant factor VIII (Recombinate) in the long term (2 years) management and prevention of bleeding episodes. One hundred thirty-four patients were enrolled into this study and 129 patients were found to be evaluable.In total, 4,171 bleeding episodes were assessed during the study period and, hemostatic efficacy was judged to be excellent in 1,769 and good in 2,031 episodes with an overall efficacy rate of 91.1%. Twenty-one patients received at least one regular replacement therapy per week with the recombinant factor VIII during this study. These patients manifested much less spontaneous bleeding episodes than the patients who had received on-demand treatments. Three adverse reactions were reported in three patients (2.3%): i.e., urticaria in one, and headache in two patients, each being only mild and transient.One patient developed an IgM antibody to recombinant the factor VIII, which was not associated with any clinical symptoms.No patients developed the inhibitor to factor VIII in Bethesda assay. These results indicate that the recombinant factor VIII, Recombinate, is safe and efficacious for the long-term management and prevention of bleeding episodes in patients with hemophilia A, who had previously received replacement treatments with factor VIII concentrates.
