Long-term Effectiveness of Hydroxyurea in Children with Sickle Cell Disease: A Real-World Study

A recent extensive study published in Blood Advances highlights the sustained benefits of hydroxyurea in managing sickle cell disease (SCD) among children. The research reveals that hydroxyurea significantly decreases emergency department visits and hospital stays over prolonged use, confirming its safety and efficacy outside controlled clinical trial settings.

Conducted by a team including Dr. Paul George from Emory University, the study analyzed data from 2,147 children under 18 years old with the severe HbSS/HbSβ0 variant of SCD. Of these, 58% (1,240 children) had been treated with hydroxyurea for an average of 5.1 years. Importantly, the study found that children on hydroxyurea experienced 0.36 fewer emergency visits and 0.84 fewer hospital days per patient-year compared to those not on the medication.

The findings are particularly reassuring for pediatric patients and their families, emphasizing that hydroxyurea—initially used as a chemotherapy agent—continues to demonstrate benefits when used long-term for SCD. The research supports current guidelines recommending hydroxyurea for children with severe forms of sickle cell, beginning between nine and 12 months of age.

Despite its longstanding use, lingering concerns about safety have persisted due to its origins and traditional applications. However, Dr. Wilbur Lam from Emory University stresses that this study reinforces hydroxyurea’s safety profile and durable efficacy outside of clinical settings.

The study also showed that improvements in hemoglobin levels, which reflect reduced anemia, were only observed in children who adhered regularly to their medication regimen. This underscores the importance of medication compliance for optimal outcomes.

While the research provides encouraging evidence, it also acknowledges limitations inherent to real-world data, such as reliance on laboratory markers to assess adherence and lack of detailed information on transfusions or pain crises. Future studies aim to incorporate patient-reported outcomes to better understand hydroxyurea’s impact beyond hospital metrics.