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Anales de la Facultad de Medicina
On-line version ISSN 2301-1254
Abstract
BERRO, Maximiliano; INSAGARAY, Juan and RODRIGUEZ, Ismael. First report of emicizumab use in a patient with severe haemophilia A and high-titer inhibitor in Uruguay. Anfamed [online]. 2020, vol.7, n.2, e405. Epub Dec 01, 2020. ISSN 2301-1254. https://doi.org/10.25184/anfamed2020v7n2a9.
Haemophilia A is a congenital coagulopathy caused by a deficiency or malfunction of coagulation factor VIII. One of the most serious complications of haemophilia A treatment is the development of inhibitors that render FVIII replacement therapy ineffective, making it difficult to prevent and control bleeding. Emicizumab is a humanized bispecific monoclonal antibody directed against factors FIXa and FX, which mimics the cofactor function of FVIII. Emicizumab has been shown to be safe and effective as prophylaxis to prevent bleeding in haemophilia A patients with or without inhibitors to FVIII. We report the first patient treated with emicizumab in Uruguay.
Keywords : haemophilia A; inhibitors; bleeding; emicizumab.