Association of hydroxyurea adherence with transcranial Doppler screenings in children with sickle cell disease

Pediatr Blood Cancer. 2024 Jul;71(7):e31017. doi: 10.1002/pbc.31017. Epub 2024 May 5.


Background: National sickle cell disease (SCD) guidelines recommend oral hydroxyurea (HU) starting at 9 months of age, and annual transcranial Doppler (TCD) screenings to identify stroke risk in children aged 2-16 years. We examined prevalence and proportion of TCD screenings in North Carolina Medicaid enrollees to identify associations with sociodemographic factors and HU adherence over 3 years.

Study design: We conducted a longitudinal study with children ages 2-16 years with SCD enrolled in NC Medicaid from years 2016-2019. Prevalence of TCD screening claims was calculated for 3 years, and proportion was calculated for 12, 24, and 36 months of Medicaid enrollment. Enrollee HU adherence was categorized using HU proportion of days covered. Multivariable Poisson regression assessed for TCD screening rates by HU adherence, controlling for age, sex, and rurality.

Results: The prevalence of annual TCD screening was between 39.5% and 40.1%. Of those with 12-month enrollment, 77.8% had no TCD claims, compared to 22.2% who had one or higher TCD claims. Inversely, in children with 36 months of enrollment, 36.7% had no TCD claims compared to 63.3% who had one or higher TCD claims. The proportion of children with two or higher TCD claims increased with longer enrollment (10.5% at 12 months, 33.7% at 24 months, and 52.6% at 36 months). Children with good HU adherence were 2.48 (p < .0001) times more likely to have TCD claims than children with poor HU adherence.

Conclusion: While overall TCD screening prevalence was low, children with better HU adherence and longer Medicaid enrollment had more TCD screenings. Multilevel interventions are needed to engage healthcare providers and families to improve both evidence-based care and annual TCD screenings in children with SCD.

Keywords: Medicaid; big data; national guidelines; pediatrics; prevalence; sickle cell anemia; stroke.

MeSH terms

  • Adolescent
  • Anemia, Sickle Cell* / diagnostic imaging
  • Anemia, Sickle Cell* / drug therapy
  • Anemia, Sickle Cell* / epidemiology
  • Antisickling Agents* / therapeutic use
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Humans
  • Hydroxyurea* / therapeutic use
  • Longitudinal Studies
  • Male
  • Medicaid / statistics & numerical data
  • Medication Adherence / statistics & numerical data
  • North Carolina / epidemiology
  • Prognosis
  • Stroke / epidemiology
  • Stroke / prevention & control
  • Ultrasonography, Doppler, Transcranial*
  • United States / epidemiology


  • Hydroxyurea
  • Antisickling Agents