Haemophilia A and
haemophilia B are congenital X-linked
bleeding disorders caused by deficiency of
coagulation factor VIII (FVIII) and IX (FIX), respectively. The preferred treatment option for patients with
haemophilia is replacement
therapy. For patients with severe disease, prophylactic replacement of
coagulation factor is the treatment of choice; this has been shown to reduce
arthropathy significantly, reduce the frequency of bleeds and improve patients' quality of life. Prophylaxis with standard recombinant factor requires regular
intravenous infusion at least two (FIX) to three (FVIII) times a week. Recombinant FVIII and FIX products with an extended half-life are in development, or have been recently licensed. With reported mean half-life extensions of 1.5-1.8 times that of standard products for FVIII and 3-5 times that of standard products for FIX, these products have the potential to address many of the unmet needs of patients currently treated with standard factor concentrates. For example, they may encourage patients to switch from on-demand treatment to prophylaxis and improve the quality of life of patients receiving prophylaxis. Indeed, extended half-life products have the potential to reduce the burden of frequent
intravenous injections, reducing the need for central venous lines in children, promote adherence, improve outcomes, potentially allow for more active lifestyles and, depending on the dosing regimen, increase factor trough levels. Members of the Zürich
Haemophilia Forum convened for their 19th meeting to discuss the practicalities of incorporating new treatments into the management of people with
haemophilia. This review of extended half-life products considers their introduction in
haemophilia treatment, including the appropriate dose and schedule of infusions, laboratory monitoring, patient selection, safety considerations, and the economic aspects of care.