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Aledort, Louis; Mannucci, Pier Mannuccio; Schramm, Wolfgang and Tarantino, Michael (2019): Factor VIII replacement is still the standard of care in haemophilia A. In: Blood Transfusion, Vol. 17, No. 6: pp. 479-486

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Abstract

Prophylactic factor VIII (FVIII) has dramatically improved haemophilia A treatment, preventing joint bleeding and halting the deterioration of joint status. FVIII products with an extended plasma half-life further improve patients' quality of life and increase therapeutic adherence. New licensed classes of non-replacement products include prophylactic emicizumab, which is administered subcutaneously up to every 4 weeks. However, this drug is not suitable for acute bleeding episodes or management of major surgery, and long-term data on the impact of emicizumab on joint health, FVIII inhibitor development and thrombotic risk are awaited. Prophylaxis with FVIII replacement remains the standard of care in haemophilia A, with the aim of achieving a level of haemostasis control that allows patients to meet their lifestyle goals.

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