New developments in haemophilia treatment
New developments and their impact on laboratory management raise many questions regarding laboratory diagnostics and that is why this topic deserves our attention.
The first section focuses on the clinical aspects. The first contribution gives an overview of new developments in the treatment of haemophilia. The updated management approaches (pharmacokinetics) and therapies which have been recently licensed or are currently in development are outlined. Next there follows an article focusing on the role of gene therapy in the future treatment of haemophilia patients. The third contribution describes personalising therapy in haemophilia; pharmacokinetically guided dosing of factor VIII and factor IX concentrates.
The second part of this special issue focuses on laboratory aspects and starts with a contribution about new developments in the treatment of haemophilia and the implications for laboratory testing. Are changes necessary in the near future when the new drugs, not based on the infusion of coagulation factors, will be widely used? An article about potency labelling of extended half-life FVIII and FIX products gives information about the importance of ensuring the consistency of production and the efficacy of a product, followed by a contribution about laboratory measurement of extended half-life FVIII and extended half-life FIX products. In the last contribution the quality assurance of replacement therapy and future perspectives are discussed.