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Hydroxyurea promotes brain development in children with sickle cell anemia.
Yonca Cam1, Jingyi Zhang2, Igor Dedkov2
1Department of Neurology, Washington University in St. Louis School of Medicine, USA; Department of Pediatrics, Washington University in St. Louis School of Medicine, USA.
Brain Research Bulletin
|July 7, 2026
Summary
Early hydroxyurea (HU) treatment in children with sickle cell anemia (SCA) may protect brain development. Long-term HU use shows benefits, particularly when started before age nine, potentially mitigating brain volume loss and cortical thinning.
Area of Science:
- Neuroscience
- Pediatric Hematology
- Radiology
Background:
- Sickle cell anemia (SCA) is linked to chronic anemia and brain atrophy.
- Hydroxyurea (HU) is recommended for infants but is underused.
- Long-term effects of HU on pediatric brain development in SCA are not well understood.
Purpose of the Study:
- To investigate if childhood-initiated HU and long-term HU use mitigate brain volume and cortical thickness changes in children with SCA.
- To assess the neuroprotective effects of early HU initiation on brain development in pediatric SCA patients.
Main Methods:
- Analyzed 330 MRI scans from SCA patients and healthy controls (2004-2024).
- Utilized FreeSurfer for volumetric analysis of gray matter volume (GMV), white matter volume (WMV), and total brain volume.
- Employed linear mixed-effects models to compare brain metrics based on HU initiation age and duration.
Main Results:
- SCA patients, regardless of HU initiation age, showed reduced total brain volume, GMV, and WMV compared to controls.
- Early HU initiation (before age 9) was associated with higher GMV compared to SCA patients not on HU.
- Long-term HU use (>1 year) correlated with higher total brain volume and GMV; early HU showed diminishing benefits with age.
Conclusions:
- Early initiation of hydroxyurea (HU) before age nine may offer neuroprotective benefits in children with sickle cell anemia (SCA).
- Long-term HU treatment positively impacts brain volume, with greater advantages observed when treatment begins at younger ages.
- Findings support earlier HU initiation to potentially mitigate adverse brain changes associated with SCA.
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