OBJECTIVE: The main objective of this prospective observational study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis with Advate® in Spain using myPKFiT®. Secondary objectives are (1) the characterization of the educational role of pharmacokinetics, (2) identifying whether there are any changes in attitude among the patients after receiving the educational information on pharmacokinetics and (3) evaluating the role of pharmacokinetics and other individual factors in patients with hemophilia A undergoing prophylactic treatment in Spain.
METHODS: This is an observational, prospective (EPA-SP) and multicenter study. It will be conducted in 14 centers in Spain and will include patients with diagnosis of hemophilia A undergoing prophylactic treatment with Advate® and will use myPKFiT®. The study will also include a cohort of patients who are already using myPKFiT® for the PK-guided prophylaxis dose adjustment.
DISCUSSION: Several studies have demonstrated the superiority of prophylactic treatment versus on-demand treatment. In order to make this prophylaxis more effective in preventing bleeding, it is necessary to adjust the treatment to several factors such as the physical activity that the patient performs, joint condition and the pharmacokinetic parameters of the infused factor VIII (clearance, life mean, area under the curve). Understanding the pharmacokinetic will help us personalize the treatment in our patients.
TRIAL REGISTRATION: ClinicalTrials.gov: NCT03006965.