Longitudinal observations of TFPI levels in paediatric Haemophilia A patients

Abstract

AbstractIntroductionInhibition of tissue factor pathway inhibitor (TFPI) is a potential new mode of action to achieve haemostasis in haemophilia A and B patients.AimKnowledge about potential developmental changes of TFPI levels during childhood are a prerequisite to translate adult doses of TFPI inhibitors to doses in paediatric patients.MethodsIn this study we present longitudinal data for total TFPI concentrations (TFPI‐T) and TFPI activity (TFPI‐A) from 48 paediatric Haemophilia A patients in the age range from 3 to 18 years (2–12 observations per patient).ResultsTFPI‐T and TFPI‐A tend to decrease over age during childhood. Lowest values were observed between 12 and <18 years. On average, TFPI‐T and TFPI‐A were lower in adolescent haemophilia patients than in adult haemophilia patients.ConclusionIn summary, the presented information on TFPI levels in children adds to the current knowledge of developmental haemostasis and it can be helpful in evaluating how children respond to haemophilia treatment including the new class of anti‐TFPI compounds.