What is the Role for Population Pharmacokinetics in Hemophilia?

Abstract

Prevention of bleeding in hemophilia requires that plasma levels of the deficient factor exceed the desired minimum target level. Large interindividual variability suggests that knowledge of individual pharmacokinetic (PK) would help to achieve this goal, simultaneously minimizing infusion frequency and the amount of concentrate used. Population PK (PopPK) allows for the incorporation of determinants of interpatient variability and eliminates the need for extensive postinfusion plasma sampling. Barriers to implementation of PopPK are the need for concentrate specific models, Bayesian calculation power, specific expertise for validation and appraisal of forecasted estimates. The Web Accessible Population Pharmacokinetic Service – Hemophilia ( www.wapps-hemo.org ), developed by an international research network of hemophilia centers will test if PK-guided dose individualization can improve patient important outcomes in hemophilia.