Safety and Efficacy of B-domain Deleted Third Generation Recombinant Factor VIII (GreenGene F?) in Korean Patients with Hemophilia A: Data from a Post-marketing Surveillance Study

摘要

Background New B-domain deleted third generation recombinant factor VIII (FVIII; GreenGene F?, beroctocog alfa) was launched in 2010. We determined safety and efficacy of GreenGene F? during routine clinical practice in patients with hemophilia A over a period of 12 months. Methods From July 2010 to July 2014, a total of 136 hemophilia A patients were enrolled in a post-marketing surveillance (PMS) study. Among them, 134 patients were assessed for drug safety and 114 patients were analyzed for drug efficacy. Patients with differing hemophilia A severities and medical histories were monitored during 12 months of prophylactic and/or on-demand therapy. Results Among 134 patients evaluated, 85 (63.4%) had severe hemophilia. Ninety-two received a total of 1,266,077 units for prophylaxis, and 42 received 516,491 units for bleeding episodes. Three patients developed inhibitors. In 112 previously treated patients, one patient (0.9%) developed inhibitor after intensive FVIII treatment for surgery. Among 22 previously untreated patients, inhibitors were observed in 2 infants (9.1%). Overall, there were a total of 47 adverse events (other than inhibitors) of all types in 30 patients (22.4%), 11 in 10 patients (7.5%) of which were considered showing serious adverse events (SAEs); most of which were hemorrhages at different sites. None of the SAEs were judged as product related. An excellent/good efficacy rate of 91.3% for hemostasis and 89.4% for hemorrhage prevention was recorded. Conclusion The results of this PMS study support the use of GreenGene F? as safe and efficacious in hemorrhage prevention and treatment of hemophilia A. These results are consistent with the findings from previously published GreenGene F? studies. Go to: Graphical