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Neurocognitive gains among Ugandan children with sickle cell anemia on hydroxyurea: 18-month interim trial results

Shubaya Kasule Naggayi1,2, Dennis Kalibbala2, Vincent Mboizi2

  • 1Department of Paediatrics and Child Health, Makerere University College of Health Sciences, Kampala, Uganda.

Blood Advances
|March 14, 2025
PubMed

Insights

Hydroxyurea therapy improved neurocognitive function in children with sickle cell anemia (SCA). Early treatment is crucial for optimal effects on cognition, attention, and executive function in SCA patients.

Area of Science:

  • Pediatrics
  • Neurology
  • Hematology

Background:

  • Sickle cell anemia (SCA) is associated with progressive neurocognitive impairment in children.
  • Cognitive deficits in SCA can impact a child's development and quality of life.
  • Early identification and intervention are critical for managing SCA-related complications.

Purpose of the Study:

  • To evaluate the effects of hydroxyurea therapy on neurocognitive function in Ugandan children with SCA.
  • To compare neurocognitive outcomes at baseline and after 18 months of hydroxyurea treatment.
  • To assess the impact of hydroxyurea on transcranial doppler (TCD) blood flow velocity in SCA patients.

Main Methods:

  • A single-arm, open-label trial involving 264 Ugandan children (ages 3-9) with SCA treated with hydroxyurea.
  • Neurocognitive testing (cognition, attention, executive function) was performed at baseline and 18 months.
  • Sibling controls (N=110) without SCA underwent parallel neurocognitive testing for age-normalized z-score comparison.

Main Results:

  • Hydroxyurea treatment significantly improved z-scores in cognition, attention, and executive function from baseline to 18 months.
  • The proportion of children with cognitive impairment decreased from 8.7% at baseline to 4.0% at 18 months.
  • Improved neurocognitive scores were associated with higher baseline scores, better socioeconomic status, and hydroxyurea-induced TCD and hematological effects.

Conclusions:

  • Hydroxyurea therapy demonstrates a positive impact on neurocognitive function in sub-Saharan African children with SCA.
  • Early initiation of hydroxyurea treatment is recommended for maximizing neurocognitive benefits.
  • Ongoing research will further evaluate the long-term effects of hydroxyurea therapy on SCA-related neurocognitive outcomes.

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