Vol 52, No 4 (2021)
Review article
Published online: 2021-08-31

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Emicizumab in severe hemophilia A

Jerzy Windyga1
DOI: 10.5603/AHP.2021.0079
Acta Haematol Pol 2021;52(4):422-428.

Abstract

Emicizumab is a recombinant, humanized, bispecific, asymmetric monoclonal antibody that bridges activated factor IX and factor X (FX) and leads to activation of FX, thus mimicking the hemostatic function of activated factor VIII (FVIIIa).

The clinical trial program showed that emicizumab prophylaxis maintains low bleed rates and is well tolerated by patients with hemophilia A of all ages with and without factor VIII (FVIII) inhibitors. Emicizumab prophylaxis in severe hemophilia A patients with high titer inhibitor against FVIII was launched in Poland in 2020. As of April 2021, 42 patients were receiving emicizumab in Poland, not including clinical trials. The aim of this paper was to review the most recent data on the role of emicizumab in the management of patients with severe hemophilia A.

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