Abstract
Sickle cell disease (SCD) is a hereditary, multi-systemic disorder characterized by hemolytic anemia and recurrent vaso-occlusive events (VOEs), significantly impacting patients’ health. Hydroxyurea (HU), the most widely prescribed disease-modifying therapy, shows variable efficacy due to differences in drug metabolism, pharmacokinetics, and adherence. This study evaluated HU blood metabolite levels in a large European sickle cell anemia (HbSS) cohort within the Genomed4all consortium, providing insights into HU response and personalized SCD management. The aim of this poster is to analyze association between HU blood metabolite levels, laboratory indices, and acute complications (VOE, acute chest syndrome [ACS]) in HbSS patients aged <10 and >10 years.