A Retrospective Analysis of Sociodemographic and Hematologic Characteristics Associated with Achieving Optimal Hydroxyurea Therapy in Children with Sickle Cell Disease

Hydroxyurea (HU) has proven hematologic and clinical benefits, especially when escalated to the maximum tolerated dose (MTD). We reviewed clinical data from patients with sickle cell disease (January 2011 to 2016) to determine baseline sociodemographic and laboratory parameters associated with reaching HU MTD without significant delays. In total, 210 patients (mean HU start age, 6.6 y) were included. Initial Kaplan-Meier event analysis showed 1 year to be an inflection point for reaching MTD. In total, 116 patients (55%) reached MTD in <1 year, with 56 (27%) taking >1 year to reach MTD and 38 (18%) patients not successfully reaching MTD during follow-up. In both crude and adjusted analyses, age at HU start was found to be significantly and inversely associated with reaching MTD within 1 year. The data presented, specifically the inflection point of reaching MTD at 1 year and the association of young HU start age with reaching MTD within a year, suggest that successful achievement of MTD may be facilitated by starting patients on HU at a young age and that older patients should receive additional intervention to attain MTD within 1 year. Patients who do not achieve MTD within a year may need the most extensive intervention.