Blood Transfusion - 3 2019 (May-June)
Emicizumab for the treatment of haemophilia A: a narrative review
Authors:  Massimo Franchini, Giuseppe Marano, Ilaria Pati, Fabio Candura, Samantha Profili, Eva Veropalumbo, Francesca Masiello, Liviana Catalano, Vanessa Piccinini, Stefania Vaglio, Simonetta Pupella, Giancarlo M. Liumbruno
Pages:  223-228
To cite this article:  Blood Transfus 2019; 17: 223-8
Doi:  10.2450/2019.0026-19
Published online:  30/04/2019

One of the most serious complications of the treatment of severe haemophilia A is the development of alloantibodies against exogenous factor VIII (FVIII). Inhibitors render factor replacement therapy ineffective, exposing patients to a remarkably high risk of morbidity and mortality. Besides the well-known bypassing agents (i.e. activated prothrombin complex concentrate and recombinant activated factor VII) used to treat or prevent bleeding in haemophilia patients with inhibitors, there is growing interest in newer haemostatic therapies that are not based on the replacement of the deficient FVIII. This review will focus on the most interesting among these innovative therapies, emicizumab, and will provide an update on its current stage of clinical development.
Keywords: emicizumab, haemophilia A, inhibitors, bleeding.
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