Vaskulárna medicína 2/2019

Emicizumab, a new era in the treatment for patients with haemophilia A

Emicizumab is a humanized, bispecific monoclonal antibody that binds FIXa and FX, promotes the conversion of FX to FXa in the absence of FVIIIa, resulting in improved effective haemostasis in haemophilia A patients. decreased the annualized bleeding rate in adolescents, adults and children with haemophilia A with or without inhibitor. Emicizumab was well tolerated and had beneficial effects on the quality of life. It should also be stressed that emicizumab may not be effective enough to manage major bleeding or bleeding during major surgery. Combination therapy with FVIII, activated prothrombin complex concentrates or rFVIIa is also required in this case. A great advantage in treatment management is its convenient subcutaneous route of administration and various modified regimens (maintenance dose once a week, once every 2 weeks or once every 4 weeks), which provide an effective and generally well tolerated alternative to standard FVIII preparations for prophylaxis and treatment of bleeding episodes. in patients with haemophilia A, regardless of the presence or absence of inhibitor.

Keywords: haemophilia A, treatment, emicizumab