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Effects of hydroxyurea on brain function in children with sickle cell anemia
Winfred C Wang1, Ping Zou2, Scott N Hwang2
1Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Pediatric Blood & Cancer
|July 31, 2021
Summary
Hydroxyurea treatment improved neurocognitive function and reduced brain blood flow velocity in children with sickle cell anemia (SCA). These findings suggest hydroxyurea can enhance brain health in SCA patients.
Area of Science:
- Neurology
- Pediatrics
- Hematology
Background:
- Sickle cell anemia (SCA) is a genetic blood disorder with significant neurological complications, including cognitive impairment.
- The long-term effects of hydroxyurea, a common SCA treatment, on brain function remain unclear.
Purpose of the Study:
- To prospectively evaluate the impact of one year of hydroxyurea treatment on brain function in children with SCA.
- To assess changes in neurocognitive performance, cerebral blood flow, and brain imaging markers.
Main Methods:
- A cohort of 19 children with SCA (HbSS/HbSβ0-thalassemia) underwent comprehensive neurocognitive testing, transcranial Doppler ultrasound (TCD), and brain MRI at baseline and after one year of hydroxyurea therapy.
- Evaluated parameters included Full-Scale IQ (FSIQ), silent cerebral infarcts (SCI), gray matter cerebral blood flow (GM-CBF), and BOLD signal response to visual stimulation.
Main Results:
- After one year, patients showed significant improvements in FSIQ and reading comprehension, alongside a marked decrease in TCD velocity.
- No significant changes were observed in GM-CBF, BOLD signal, or SCI frequency.
- Correlations were found between neurocognitive outcomes and levels of Hemoglobin F (HbF), hemoglobin, GM-CBF, and BOLD signal.
Conclusions:
- One year of hydroxyurea treatment demonstrates potential benefits for neurocognitive outcomes and cerebrovascular health in children with SCA.
- Further research is needed to elucidate the precise mechanisms underlying these improvements, particularly the relationships between HbF, hemoglobin, cerebral blood flow, and cognitive function.
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