Haemophilia care is evolving rapidly. Clotting factor concentrates (CFC) are increasingly available as either plasma derived or recombinant types. Recently, new recombinant CFC products have extended half-life technologies (e.g. Elocta, Alprolix). Examples of all these types of CFC have been part of recent WFH donations to the Bangladesh community and are suitable for on demand treatment, covering surgical procedures and/or consideration of low dose prophylaxis. Further treatment evolution now includes non-factor treatments, e.g. biphenotypic antibody (Emicizumab/Hemlibra). Emicizumab is an FDA approved prophylaxis agent for use in both the inhibitor and non-inhibitor setting of severe haemophilia A.
Dr Hart’s talk will discuss how the choice of agents from antifibrinolytics (e.g. Tranexamic acid), DDAVP, standard and extended half-life CFC through to emicizumab have important roles in the evolution of global haemophilia care. Capturing measurable outcome metrics in our patient communities will provide important data to continue to advocate for patient access to treatment.
Correspondence: Dr Daniel Hart, Senior Lecturer in Haematology, Immunobiology, Blizard Institute, Barts and The London School of Medicine & Dentistry, QMUL. Honorary Consultant. Royal London Haemophilia Centre, The Royal London Hospital, Barts Health NHS Trust, Whitechapel, London E1 1BB.