Emicizumab in the treatment of hemophilia A

Abstract

Introduction: Despite substantial advances in the treatment of Hemophilia A with the use of concentrated factor VIII preparations during recent decades, bleeding episodes still occur from time to time. The development of inhibitors significantly reduces the efficacy of traditional replacement therapy, seriously increasing morbidity and mortality in these patients. Emicizumab (HEMLIBRA ®) is a chimeric bispecific humanized antibody that bridges activated FIX and FX and thus restores the function of missing activated FVIII. Aim: The aim of the study was to analyze the literature date of the effect of Emicizumab in the treatment of Hemophilia A. Results: Multicenter randomized studies called HAVEN have shown excellent results of this medication in the treatment of patients with Hemophilia A. FVIII inhibitors do not bind to or neutralize Emicizumab and therefore have no effect on the hemostatic activity of the drug. Emicizumab prophylaxis produced a significant reduction in treated bleedings of 79%, compared to with the group of patients on prophylaxis with bypassing agents, while the rate grew up to even 95% after longer observation. Other studies have also confirmed good treatment results and a favorable safety profile in both adults and children. In the cases of bleeding events or preparation for immediate surgical interventions, it is recommended to user FVII (NovoSeven) ® according to previous guidelines. Conclusion: The results of the prophylactic use of Emicizumabhave so far shown that it may be a revolutionary preparation that can significantly reduce bleeding episodes and improve the quality of life of patients with Hemophilia A. Nevertheless, further testing of this drug is required.

Keywords

• emicizumab
• hemophilia a
• treatment