Iannazzo S, et al. – The cost–effectiveness of pharmacokinetic–driven prophylaxis in severe haemophilia A patients, was evaluated. With savings of 31 205€ per bleed avoided, this study found pharmacokinetic–driven prophylaxis more preferable (i.e. more effective and less costly) than standard prophylaxis. Accounting for patients’ individual pharmacokinetic variability, the aforementioned prophylaxis seemed to be a promising strategy to improve outcomes with efficient use of available resources in these patients.

Methods

• This study developed a microsimulation model to evaluate the cost-effectiveness of pharmacokinetic-driven prophylaxis vs. standard prophylaxis and estimate cost, annual joint bleed rate (AJBR), and incremental cost-effectiveness ratio over a 1-year time horizon for a hypothetical population of 10 000 severe haemophilia A patients.
• For standard prophylaxis, a dose of 30 IU/kg per 48 h was assumed.
• In order to maintain trough levels at least 1 and 5 IU/dl or less, pharmacokinetic prophylaxis was individually adjusted.
• Experts evaluated AJBR on the relationship between factor VIII (FVIII) levels and bleeding rate reported in the literature.
• They regulated sensitivity analyses to assess the stability of the model and the reliability of results.

Results

• With a negligible impact on AJBR (+0.1 bleed/year), the FVIII dose was reduced in the 27.8% of patients with a trough level more than 5 IU/dl on standard prophylaxis.
• Results revealed increased FVIII dose in the 10.6% of patients with trough levels less than 1 IU/dl on standard prophylaxis, with a significant reduction of AJBR (-1.9 bleeds/year).
• With total saving of 5 197€ per patient-year, overall, pharmacokinetic-driven prophylaxis was shown to decrease the AJBR from 1.012 to 0.845 with a slight reduction of the infusion dose of 0.36 IU/kg.