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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Authors

Louis Aledort - Division of Hematology/Oncology, Icahn School of Medicine, Mount Sinai, New York, NY, United States of America

Pier Mannuccio Mannucci - Scientific Direction, IRCCS Foundation Ca' Granda Maggiore Policlinico Hospital, Milan, Italy

Wolfgang Schramm - Department of Transfusion Medicine and Haemostasis, University Hospital of Munich, Munich, Germany

Michael Tarantino - University of Illinois College of Medicine, and the Bleeding and Clotting Disorders Institute, Peoria, IL, United States of America

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