Ticagrelor may not yield additional benefits for patients with sickle cell disease (SCD), according to a study recently published in Blood.

“Despite clear biological rationale for antiplatelet therapy in SCD, and the greater platelet inhibition in HESTIA3 than in the previous DOVE study, there was no benefit of ticagrelor in this patient group,” the authors said.

This phase 3 study included a total of 193 pediatric patients previously diagnosed with SCD, aged 2 to 17 years, from 16 different countries. The participants underwent random assignment into either a group that received a placebo or a group that received ticagrelor in doses of 15 mg, 30 mg, or 45 mg twice daily in patients with a total body weight of 12 to 24 kg, 24 to 48 kg and more than 48 kg, respectively.

After 296.5 days of following this therapeutic scheme, the researchers did not observe any benefits. The incidence of vaso-occlusive crises remained similar among both groups, with 2.74 in the ticagrelor patients and 2.60 in the placebo group.

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Likewise, platelet inhibition did not seem to modify since the median was 34.9% and 55.7% prior to and 2 hours post-treatment, upon 6 months of the study. Furthermore, both groups had 9% of the participants experiencing 1 or more bleeding events.

These results led to the author’s decision to end the trial 4 months prior to what was initially planned, as explained by Heeney and colleagues.

Although unexpected, these findings do carry valuable information regarding SCD in children and potential therapeutic targets. Previous research also identified other drugs as potential therapies and ultimately deemed inappropriate for this disease, such as olinciguat, poloxamer, prasugrel, regadenoson, rivipansel, senicapoc, and sevuparin.

“One possible reason is that the degree of platelet activation and the cumulative endothelial damage is not as substantial in children as it is in adults living with SCD; thus, any possible benefit of platelet inhibition may be less evident,” the authors concluded.

Reference

Heeney M, Abboud M, Githanga J, et al. Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study. Blood. Published online July 18, 2022. doi:10.1182/blood.2021014095