Blood Adv

Long-term hydroxyurea therapy benefits children with sickle cell anemia

April 23, 2025

Study details: This quasi-experimental study analyzed the long-term effects of hydroxyurea on clinical and lab outcomes in 2,147 children with sickle cell anemia (SCA) over an 11-year period (2010-2021). The study utilized contemporary difference-in-differences and dynamic event study analyses to assess the impact of hydroxyurea on ED visits, hospital days, and hemoglobin concentration.

Results: Hydroxyurea use was associated with a significant reduction in ED visits (average treatment effect on the treated [ATT]; -0.36 visits/year, 95% confidence interval [CI], -0.57, -0.16) and hospital days (ATT, -0.84 days/year; 95% CI, -1.51, -0.17). Additionally, there was an increase in hemoglobin concentration (ATT, 0.56 g/dL; 95% CI, 0.39, 0.73), with sustained effects observed in patients with good adherence.

Clinical impact: Hydroxyurea provides sustained clinical benefits in reducing ED visits and hospital days in children with SCA. These findings underscore the importance of adherence to hydroxyurea therapy for optimizing long-term clinical outcomes in pediatric SCA management.

Source:

George PE, et al. (2025, April 16). Blood Adv. Evaluating the Long-Term Benefits of Hydroxyurea in Pediatric Sickle Cell Anemia. https://pubmed.ncbi.nlm.nih.gov/40238672/