Abstract

As emicizumab shares no sequence homology with FVIII, in patients with haemophilia A, haemostasis can be restored irrespectively of the presence of factor VIII inhibitors. Emicizumab therefore substantially improves upon current treatment options. This study aims at comparing the patients’ quality of life (QoL) and clinical outcomes of emicizumab prophylaxis therapy and recombinant FVIII (rFVIII) prophylaxis for the treatment of severe Haemophilia A, based on a Markov cohort simulation.

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