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Hydroxyurea Use in Young Children With Sickle Cell Anemia in New York State
David G Anders1, Fei Tang2, Tatania Ledneva3
1Office of the Medical Director, Office of Quality and Patient Safety, New York State Department of Health, Albany, New York.
Insights
Hydroxyurea use is increasing in New York children with sickle cell anemia, but variations in treatment centers and adherence may affect its benefits.
Area of Science:
- Pediatric Hematology
- Pharmacology
- Public Health
Background:
- Sickle cell anemia is a serious inherited blood disorder affecting young children.
- Hydroxyurea is a disease-modifying therapy for sickle cell anemia.
- Understanding hydroxyurea usage patterns in children is crucial for optimizing treatment outcomes.
Purpose of the Study:
- To examine hydroxyurea usage patterns in a cohort of young children with sickle cell anemia in New York State.
- To analyze factors influencing hydroxyurea initiation and persistence.
- To identify regional and institutional variations in treatment delivery.
Main Methods:
- Retrospective analysis of Medicaid data for 273 children with sickle cell anemia born in NYS (2006-2009).
- Examined age at first prescription, persistence, region, treatment institution, and year of hydroxyurea use.
- Log-binomial regression models were used to assess treatment likelihood.
Main Results:
- Approximately 25% of children received hydroxyurea by age 5, rising to nearly 40% by study end.
- Mean adherence (proportion of days covered) was 56.3% in the first year; 34% achieved 80% coverage.
- Significant regional and institutional differences in hydroxyurea initiation were observed, but not in persistence.
Conclusions:
- Hydroxyurea use in young children with sickle cell anemia is widespread and increasing in NYS.
- Variations in treatment practices among centers and suboptimal adherence may limit hydroxyurea's full therapeutic potential.
- Further research into improving adherence and standardizing care is warranted.
Introduction:
This study examined hydroxyurea usage in young children with sickle cell anemia within New York State (NYS). The cohort was 273 children with sickle cell anemia born in NYS in 2006-2009 and enrolled essentially continuously in Medicaid for the first 4 years of life.
Methods:
Medicaid data were used to examine hydroxyurea usage in this group by age at first prescription fill, persistence, region, treatment institution, and year. Log-binomial regression models were used to estimate the likelihood of receiving hydroxyurea treatment. Data from birth through 2014 for all members of the study group were assembled and analyzed in 2015.
Results:
About 25% of the cohort had at least one filled hydroxyurea prescription by their fifth birthday, and nearly 40% by the end of the study period. The mean proportion of days covered for the first year of therapy was 56.3%. Adherence was also assessed by calculating medication possession ratios for individual treatment periods. Slightly more than one third of treated children showed 80% coverage by these measures. There was a consistent, but not statistically significant, trend toward younger age at first fill. Significant regional and treatment center differences in initiation of hydroxyurea use, but not in persistence after initiation, were noted among NYS centers.
Conclusions:
Subsequent to clinical studies demonstrating safety, current NYS-wide use of hydroxyurea in young children with sickle cell anemia appears to be widespread and increasing. However, practice differences between treatment centers and inadequate adherence may limit the full disease-modifying effects of hydroxyurea.
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