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Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center
Kristine Karkoska1, Kevin Todd1, Omar Niss1,2
1Division of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Insights
Early hydroxyurea (hydroxyurea) use in children with sickle cell anemia (SCA) significantly reduced hospitalizations. Implementing guidelines for hydroxyurea treatment in pediatric SCA patients is feasible and improves outcomes.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Sickle cell anemia (SCA) complications begin in childhood without early disease-modifying therapy.
- Hydroxyurea is a safe and effective treatment for SCA complications.
- Current hydroxyurea (hydroxyurea) utilization in pediatric SCA patients remains below recommended levels.
Purpose of the Study:
- To evaluate the impact of updated guidelines on hydroxyurea (hydroxyurea) prescribing practices in pediatric SCA patients.
- To assess changes in clinical outcomes following increased hydroxyurea (hydroxyurea) utilization.
Main Methods:
- Retrospective review of hydroxyurea (hydroxyurea) prescribing practices and clinical outcomes over a 10-year period.
- Analysis of data before and after the 2014 NHLBI recommendations for hydroxyurea (hydroxyurea) use in SCA.
Main Results:
- Hydroxyurea (hydroxyurea) use in pediatric SCA patients increased from 43% to 95% between 2010 and 2019.
- The median age of hydroxyurea (hydroxyurea) initiation decreased significantly after 2014.
- SCA-related hospital admissions decreased by 42% during the study period.
Conclusions:
- Early and universal hydroxyurea (hydroxyurea) prescription for children with SCA is the standard of care.
- Implementing guidelines for hydroxyurea (hydroxyurea) treatment is feasible and leads to improved clinical outcomes.
- This approach can serve as a model for other institutions to increase hydroxyurea (hydroxyurea) use in pediatric SCA patients.
Background:
Without early initiation of disease-modifying therapy, the acute and chronic complications of sickle cell anemia (SCA) begin early in childhood and progress throughout life. Hydroxyurea is a safe and effective medication that reduces or prevents most SCA-related complications. Despite recommendations to prescribe hydroxyurea for all children with SCA as young as 9 months, utilization remains low.
Procedure:
We completed a retrospective review of hydroxyurea-prescribing practices and associated clinical outcomes at our institution over a 10-year period before and after the 2014 National Heart, Lung, and Blood Institute (NHLBI) recommendations to use hydroxyurea for all children with SCA.
Results:
Hydroxyurea use more than doubled within our pediatric SCA population from 43% in 2010 to 95% in 2019. The age of hydroxyurea initiation was significantly younger during 2014-2019 compared to 2010-2013 (median 2 years vs. 6 years, p ≤ .001). With this change in clinical practice, nearly all (69/71 = 97%) children born after 2013 received disease-modifying therapy by the end of 2019, primarily hydroxyurea (93%). Concurrently, the number of SCA-related admissions significantly decreased from 67/100 patient-years in 2010 to 39/100 patient-years in 2019 (p < .001).
Conclusion:
The early and universal prescription of hydroxyurea for children with SCA is the standard of care. Here, we demonstrate that a careful and deliberate commitment to follow this guideline in clinical practice is feasible and results in measurable improvements in clinical outcomes. Our approach and improved outcomes can serve as a model for other programs to expand their hydroxyurea use for more children with SCA.
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