Hydroxyurea Has Sustained Clinical Benefits in Children With Sickle Cell Anemia
TUESDAY, April 29, 2025 -- For children with sickle cell anemia (SCA), hydroxyurea has sustained clinical benefits in terms of reducing emergency department visits and hospital days, according to a study published online April 16 in Blood Advances.
Paul E. George, M.D., of Emory University in Atlanta, and colleagues conducted a quasi-experimental study using contemporary difference-in-differences and dynamic event study analyses on a longitudinal cohort of 2,147 children with SCA to quantify the effects of hydroxyurea on clinical and laboratory outcomes. The average follow-up time was 5.5 years, providing a total of 11,925 patient-years of data.
The researchers found that from 2010 to 2021, there was an association for hydroxyurea use with fewer emergency department visits per year and fewer hospital days per year (average treatment effect on the treated [ATT], −0.36 visits/year and −0.84 days/year, respectively), with effects sustained over time. Hemoglobin concentration increased with hydroxyurea use on average (ATT, 0.56 g/dL); only the subgroup with laboratory markers of good adherence had a sustained effect.
"These results underscore the potential for even greater clinical benefits through programmatic efforts to improve hydroxyurea adherence and optimize dosing strategies," the authors write. "These findings reinforce the importance of hydroxyurea as a key treatment modality in SCA and provide insights for clinicians and families regarding its long-term efficacy."
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