• Medicina clinica · Dec 2021

    Review

    Treatment of haemophilia: From replacement to gene therapy.

    • José A Páramo.
    • Servicio de Hematología y Hemoterapia, Clínica Universidad de Navarra, Instituto de Investigación Sanitaria de Navarra (IdiSNA), Pamplona, Navarra, España; CIBERCV. Electronic address: japaramo@unav.es.
    • Med Clin (Barc). 2021 Dec 24; 157 (12): 583-587.

    AbstractHaemophilia A and B are congenital bleeding disorders characterized by missing or defective factor VIII or factor IX, respectively. Factor replacement therapy has been the gold standard for prophylaxis and treatment of bleeding complications. However, the inconvenience of regular intravenous administration, along with progression of arthropathy and development of inhibitors has driven the need for alternative treatment options, such as extended half-life products, non-factor coagulation products, such as subcutaneous emicizumab, blocking natural anticoagulants (rebalancing haemostatic agents) and gene therapy, which have been useful to control bleeding or are currently under late-phase clinical investigation.Copyright © 2021 Elsevier España, S.L.U. All rights reserved.

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