Personalized treatment for haemophilia

Abstract

Haemophilia treatment has been tremendously improved in the last decade with the development of extended half-life products, non-replacement therapies and finally the arrival of gene therapy on the scene. Several questions are still open to debate such as which molecules for which patients? Which dosage? Can we safely combine two procoagulant molecules in case of severe breakthrough bleeds? Answers to these questions include a personalized approach taking into account all individual variables such as genotype, clinical bleeding phenotype, individual pharmacokinetic characteristics with different products, personal pharmacodynamics, personal risk of inhibitors, joint status, physical activity, profession and adhesion to therapy. Scientific data supporting the importance of these variables as well as clinical, laboratory and radiological tools that may help to optimize treatment of patients with hemophilia are discussed in this manuscript.