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Hydroxyurea therapy for neurological and cognitive protection in pediatric sickle cell anemia in Uganda (BRAIN SAFE

Vincent Mboizi1, Catherine Nabaggala2, Deogratias Munube2

  • 1Global Health Uganda, Kampala, Uganda.

Contemporary Clinical Trials Communications
|December 24, 2024
PubMed

Insights

Hydroxyurea therapy may prevent sickle cerebrovascular injury (SCVI) in children with sickle cell anemia (SCA). This Ugandan trial investigates hydroxyurea

Area of Science:

  • Pediatric Neurology
  • Hematology
  • Vascular Medicine

Background:

  • Children with sickle cell anemia (SCA) in Sub-Saharan Africa face a high risk of sickle cerebrovascular injury (SCVI).
  • Hydroxyurea is a disease-modifying therapy that may reduce SCVI, potentially lowering stroke incidence and cognitive dysfunction.
  • The BRAIN SAFE II study addresses the impact of hydroxyurea on these outcomes in Ugandan children with SCA.

Purpose of the Study:

  • To test the impact of daily hydroxyurea therapy over 36 months on preventing, stabilizing, or improving SCVI in Ugandan children with SCA.
  • To assess the effects of hydroxyurea on stroke risk and neurocognitive dysfunction.
  • To provide critical insights into hydroxyurea's role in managing SCVI manifestations in pediatric SCA.

Main Methods:

  • An open-label, single-arm trial involving 270 children (ages 3-9) with SCA (HbSS) in Uganda.
  • Hydroxyurea therapy administered, with dosage escalated to the maximum tolerated dose (MTD).
  • Primary outcomes: cerebral arterial velocity (Doppler ultrasound) and neurocognitive testing. Secondary outcomes: MRI/MRA for structural SCVI, biomarkers of anemia, inflammation, and malnutrition.

Main Results:

  • This section is not available in the provided abstract.
  • Further analysis is required to determine the efficacy of hydroxyurea in preventing or ameliorating SCVI.
  • Longitudinal data collection at 18 and 36 months will allow for comprehensive outcome assessment.

Conclusions:

  • The trial will examine hydroxyurea's impact on preventing or ameliorating SCA-related SCVI in children.
  • Results will elucidate hydroxyurea's role in reducing stroke risk and neurocognitive dysfunction in pediatric SCA.
  • This study is crucial for understanding hydroxyurea's therapeutic potential in managing critical SCVI manifestations.
Abstract

Keywords:
HydroxyureaNeurological and cognitive protectionSickle cell anemiaTranscranial Doppler

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