Future perspectives in haemophilia treatment

Current treatment of haemophilia provides excellent efficacy and safety albeit with a number of unresolved issues. The development of inhibitors following treatment with factor VIII (FVIII) is the most challenging complication of haemophilia and bears the highest economic burden for a chronic disease. Moreover, prophylactic therapy for haemophilia requires repeated infusions of FVIII, which can impact greatly on patients daily lives. As considerable room remains for further advancements in the management of this condition, the primary focus of this paper relates to issues regarding current treatment and strategies meant to resolve the various issues. It is important to know whether or not plasma-derived and recombinant products are associated with different risks of inhibitor development in previously untreated patients with severe haemophilia. Methods under investigation to prolong the half-life of factor concentrates offer new hope to reduce the burden of prophylaxis for patients with haemophilia, with early results suggesting greater benefits with FIX than with FVIII.