Ticagrelor was well-tolerated and palatable for children under 24 months old with sickle cell disease (SCD) in a recent study published in Pediatric Blood Cancer.

Prior studies have found that antiplatelet treatments reduce vaso-occlusive crises (VOCs) in patients with SCD. Ticagrelor is not currently indicated for pediatric patients, but it is a direct-acting antiplatelet agent used for adults with acute coronary syndromes. SCD symptoms can begin in children as young as 6 months, but treatments are limited to patients 9 months and older.

HESTIA2 is a phase 2b trial of ticagrelor for SCD patients aged 18 to 30 years. The drug was well-tolerated in the patient population. The current study, HESTIA4 (ClinicalTrials.gov identifier: NCT03492931) evaluated the safety and tolerability of ticagrelor in patients younger than 24 months.

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The phase 1, open-label study included 21 children with SCD. Patients received a weight-adjusted dose on day 1. Researchers collected pharmacokinetic (PK) blood samples up to 4 times: at 1, 2, 4, and 6 hours after dosing. The investigators did not identify any treatment-related adverse events.

Blood samples showed ticagrelor had similar antiplatelet activity in children as it does in adults, as confirmed in HESTIA1. Maximum plasma concentrations of ticagrelor occurred 1 to 2 hours after the single dose.

Previous trials in adults with heart disease have evaluated safety of ticagrelor. In this study, a single dose of ticagrelor was well-tolerated by the young population with SCD, and the drug continues to be studied in young children in the long-term HESTIA3 study.

The study is limited by the small number of participants, including only 2 patients younger than 6 months. Future studies can include more PK data and pharmacodynamic assessments in very young patients with SCD.

Disclosures: Some authors have declared affiliations with or received grant support from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.


Duniva Inusa BP, Inati A, Maes P, et al. Pharmacokinetics and safety of ticagrelor in infants and toddlers with sickle cell disease aged <24 months. Pediatr Blood Cancer. Published online February 25, 2021. doi:10.1002/pbc.28977