Hydroxyurea underused among children with sickle cell anemia despite guideline update
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Key takeaways:
- Researchers reported a mean annual days’ supply of hydroxyurea of 47.2 days per child in Michigan and 97.4 days per child in New York.
- The findings indicate a need for more interventions to increase uptake.
Despite having been established as the primary disease-modifying therapy for individuals with sickle cell anemia, hydroxyurea remains underused among children with the disease, according to data published in JAMA Network Open.
However, researchers did note “encouraging trends” in both Michigan and New York state Medicaid programs that point toward increased use of hydroxyurea since the implementation of revised guidelines, researchers wrote.
“Despite changes in clinical guidelines to recommend hydroxyurea for all children with sickle cell anemia starting in 2014, the majority of youths did not receive this disease-modifying therapy,” Sarah L. Reeves, PhD, MPH, assistant professor of pediatrics and epidemiology at University of Michigan School of Public Health, told Healio. “Although disappointing, this was not surprising. Our previous research showed that children with sickle cell anemia do not receive other essential, recommended preventive services at high rates. Specifically, less than one in 10 receive enough antibiotic prophylaxis in the year to protect against infection, and less than half receive screening for strokes.”
Background and methodology
Children diagnosed with sickle cell anemia are at an increased risk for pain crises, stroke and early death; however, such complications can be reduced with hydroxyurea, an oral disease-modifying medication that revised National Heart, Lung, and Blood Institute guidelines recommend for children aged 9 months and older, regardless of disease severity.
Reeves and colleagues conducted a cross-sectional study using 2010 to 2018 administrative data from Medicaid programs in Michigan and New York state.
The study included 4,302 youths with sickle cell anemia (2,236 males; 2,929 non-Hispanic Black) aged 1 to 17 years enrolled in the Medicaid programs for at least 1 year, for a total of 12,565 person-years of data.
Hydroxyurea use, characterized as mean annual counts of days’ supply of filled hydroxyurea prescriptions, served as the main outcome.
Results
Researchers reported a mean annual days’ supply of hydroxyurea of 47.2 days per child in Michigan and 97.4 days per child in New York.
Michigan saw an increase in the odds of having nonzero days’ supply of the agent following release of the updated guidelines (OR = 1.52; 95% CI, 1.07-2.14). Researchers did not note a change in New York.
Next steps
The findings indicate a need for additional interventions to increase hydroxyurea uptake among children with sickle cell anemia, Reeves said.
“Some modestly scaled interventions have been associated with some improvement in hydroxyurea use within the context of clinical trials designed to address adherence issues,” Reeves told Healio. “However, wide-scale implementation studies haven’t been conducted to improve uptake and sustainability of outcomes. It is important that family and patient voices and perspectives be integrated into any intervention. This is particularly true given the wider systemic issues often facing individuals living with sickle cell disease, such as structural and institutional racism.”
For more information:
Sarah L Reeves, PhD, MPH, can be reached at University of Michigan, 2800 Plymouth Road, NCRC Building 16 Room G027W, Ann Arbor, MI 48109-2800; email: sleasure@umich.edu.
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