Impact of pharmacokinetics to reduce bleeding in a cohort of patients with severe hemophilia A in a personalized comprehensive management program

Abstract

In recent decades, hemophilia A treatment has been focused on body weight, without taking pharmacokinetic parameters into account. Previous research has shown that the individual pharmacokinetic response is more effective in predicting the required dose of clotting factor. We want to evaluate the impact on reducing the frequency of bleeding in patients treated with recombinant factor VIII, based on a personalized comprehensive management program. Our aim was to compare the results of a standard comprehensive treatment program (stage I) vs. a personalized pharmacokinetic - based treatment program (stage II) in a cohort of 60 patients with severe hemophilia without inhibitors. The median age was 15.5 years (3-68). The annual bleeding rate (ABR) was 1.03 (62 episodes) in the first stage and 0.58 (35 episodes) in the second one, (p=0.004). By type of bleeding, the impact of the intervention differs significantly in spontaneous bleeding (p=0.007) and a 73% reduction in the first stage. There were no significant differences in traumatic bleeding. The use of pharmacokinetics (PK) for personalized dosing of patients with severe hemophilia A, significantly reduces ABR and spontaneous bleeding, improving the patient’s quality of life and costs for the health system.