Pharmacokinetics for haemophilia treaters: Meaning of PK parameters, interpretation pitfalls, and use in the clinic

Abstract

Replacement therapy with concentrates of factor VIII or IX remains the gold standard for severe haemophilia management. The recent development of clotting factor products with extended half-life, widely available on the market since 2 years, facilitates adherence, improves considerably the patients' quality of life, and simplifies the management of breakthrough bleedings or surgery. These molecules have also brought to the limelight the concepts of optimization and personalization of anti-haemophilic prophylaxis. Pharmacokinetics (PK) is one of the tools that can help haematologists to adapt in a more objective and precise manner the prophylaxis regimen to each individual patient's specific needs. For many years, clinicians at haemophilia centres have been using some simple PK parameters, such as recovery and residual level. However, recently, they have been confronted with an important number of new PK parameters they were not familiar with, but that can be used to improve patient management. Due to the accumulation of PK data and their relative complexity, it is now necessary to analyse the relevance of the different PK parameters relative to haemophilia specificities, and also to know their limits to better use them.

Keywords: Haemophilia A; Haemophilia B; Pharmacokinetic; Recombinant human factor IX; Recombinant human factor VIII.