Savings without changing: How to use the MyPKfit® device to improve treatment strategies in a cohort of patients with haemophilia A

Abstract

Background: The real goal on haemophilia treatment is to combine efficacy, safety, improvement in quality of life and cost-savings. Sometimes the choice for reaching this result is to switch the patients to an extended half-life (EHL) drug. In case of haemophilia A this goal is not always achieved due to the less pharmacokinetic (PK) differences among EHL and standard concentrates. A better and regular use of available tools, as MyPKfit®, can then optimize the treatment without distorting therapy or changing concentrate.

Methods: We now report our experience with a population of severe or moderate haemophilia A patients treated with octocog-alfa (Advate® -Shire Takeda) and in which a tailored prophylaxis with MyPKfit® has been assessed.

Results: PK evaluations of 14 patients were carried out. A Bayesian curve and a tailored prophylaxis were assessed individually employing PK data. The weekly frequency of infusions was reduced in three severe patients, it was increased in four while it remained the same in the others five patients. The annual consumption of concentrate was reduced in 81.8% of patients. A subsequent economic evaluation carried out for each of the twelve severe haemophilia A patients included in this analysis, in which we have compared the standard and the PK-driven prophylaxis, showed that an optimized treatment can lead to an annual average saving of € 20,525 (-15.8%).

Conclusions: The use of MyPKfit® for a tailored prophylaxis may lead to a more rational use of available resources through an easy correction of the treatment strategies without distorting the individual patient therapy.

Keywords: Bayesian model; Haemophilia; MyPKfit®; Tailored prophylaxis.