Summary
The optimal treatment of hemophilia has been evolving since the advent of factor VIII
concentrates, continues to vary geographically, and remains a source of debate. There
now exists an extensive clinical literature that demonstrates clear benefits of prophylaxis
for patients with hemophilia A compared to on-demand treatment, including a reduction
in the number of bleeding episodes, improved joint function, and greater patient well-being.
However, the value of these benefits must be weighed against the heavier economic
burden of increased factor use. This paper reviews the literature that compares the
benefits of prophylaxis with that of on-demand treatment, compares varying prophylaxis
dose and administration frequency protocols, and considers the long-term cost-benefit
of prophylactic therapy.
Keywords
Hemophilia - prophylaxis - on-demand - factor VIII - arthropathy - cost-benefit