Pharmacokinetics and safety of ticagrelor in infants and toddlers with sickle cell disease aged <24 months

Pediatr Blood Cancer. 2021 May;68(5):e28977. doi: 10.1002/pbc.28977. Epub 2021 Feb 25.

Abstract

Inhibition of platelet activation may reduce vaso-occlusion rates in patients with sickle cell disease (SCD). In the HESTIA4 (NCT03492931) study, 21 children with SCD received a single oral dose of the antiplatelet agent ticagrelor (0.1 mg/kg <6 months; 0.2 mg/kg ≥6 to <24 months). All patients had measurable ticagrelor plasma concentrations. Ticagrelor and active metabolite (AR-C124910XX) exposure were comparable across all groups (<6 months, ≥6 to <12 months and ≥12 to <24 months). Ticagrelor was well tolerated. Palatability was generally acceptable. These data will be used to enable dose selection for further investigations of ticagrelor efficacy and safety in children with SCD.

Keywords: paediatric; pharmacokinetic; sickle cell disease; ticagrelor.

Publication types

  • Clinical Trial, Phase I
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Sickle Cell / drug therapy*
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Platelet Aggregation / drug effects
  • Platelet Aggregation Inhibitors / adverse effects*
  • Platelet Aggregation Inhibitors / pharmacokinetics*
  • Ticagrelor / adverse effects*
  • Ticagrelor / pharmacokinetics*

Substances

  • Platelet Aggregation Inhibitors
  • Ticagrelor

Associated data

  • ClinicalTrials.gov/NCT03492931