A multi-center post-marketing surveillance study of recombinant factor VIII (Recombinate) in previously untreated patients with hemophilia A

Abstract

A multi-center post-marketing surveillance study was conducted in previously untreated patients with hemophilia A in order to evaluate the safety and efficacy of recombinant factor VIII (Recombinate) in the long term (≥2 years) management and prevention of bleeding episodes. Twenty-five patients were enrolled into this study and 22 patients were evaluable. A total of 378 bleeding episodes were assessed during the study period; hemostatic efficacy was judged to be excellent in 226 and good in 138 for an overall efficacy rate of 96.3%. Two patients received three prophylactic treatments per week with recombinant factor VIII during this study (primary and secondary prophylaxis). These patients had few spontaneous bleeding episodes and no joint bleeding episodes for over 2.5 years. Five adverse reactions in 5 patients (22.7%) were reported. Four patients (18.2%) developed evidence of an inhibitor to factor VIII(2 high responders, and 2 low responders, one of which was transient) and, one patient (4.5%) developed evidence of an anti-bovine serum albumin antibody. The incidence of anti-factor VIII inhibitor formation in this study is comparable to that reported in recent prospective studies of this and other factor VIII products. The patient who developed an anti-bovine serum albumin antibody showed no clinical symptoms. These results indicate that recombinant factor VIII (Recombinate) is safe and efficacious for the long-term managemant and prevention of bleeding episodes in previously untreated patients with hemophilia A.