Prophylactic versus on-demand treatments for hemophilia: advantages and drawbacks

Early long-term prophylaxis is the standard of care to prevent joint bleeding and chronic arthropathy in patients with severe hemophilia. Areas covered: Despite the obvious prophylaxis advantages upon the clinical outcomes, there are still several drawbacks to be addressed for the optimal patients' compliance. Frequency of treatment due to short half-life of conventional FVIII and FIX concentrates, difficult venous access, adherence to the prescribed therapy and costs may represent significant critical issues. The development of inhibitors also makes replacement therapy ineffective, preventing patients from receiving long-term prophylaxis. This paper will review these drawbacks and the tools to overcome these limitations, mainly thanks to the use of extended half-life products and the development of novel non-conventional therapeutic approaches. Expert commentary: The use of extended half-life products may help in reducing the burden of the frequent intravenous administration and in better tailoring an individualized prophylaxis. The development of novel therapeutic approaches, like the bi-specific antibody mimicking the coagulation function of FVIII or inhibition of anticoagulant proteins may facilitate prophylaxis treatment not only in patients with inhibitors, but also in severe hemophilia patients without inhibitors. Exciting opportunities are emerging for improving prophylaxis in hemophilia.