Realizing Effectiveness Across Continents With Hydroxyurea

Korábbi kezelés

• Blood transfusion within 60 days before enrollment (temporary exclusion)
• In the Original Cohort, hydroxyurea use within 6 months before enrollment (temporary exclusion). In the New Cohort, the children should be hydroxyurea naïve, without any prior treatment exposure.

Más betegség

• Known medical condition making participation ill-advised (e.g., acute or chronic infectious disease, HIV, or malignancy)

Laborértékek

• Pre-existing severe hematological toxicity (temporary exclusions)
  • Anemia: Hb <4.0 g/dL
  • Anemia: Hb <6.0 g/dL with ARC <100 x 109/L
  • Reticulocytopenia: ARC <80 x 109/L with Hb <7.0 g/dL
  • Thrombocytopenia: Platelets <80 x 109/L
  • Neutropenia: ANC <1.0 x 109/L

Életkor

• Acute or chronic severe malnutrition determined by impaired growth parameters as defined by WHO (weight for length/height or height for age >3 z-scores below the median WHO growth standards.

• Pediatric patients with documented sickle cell anemia (typically HbSS supported by hemoglobin electrophoresis, complete blood count, and peripheral blood smear)
• In the Original Cohort, age range of 1.00-9.99 years, inclusive, at the time of enrollment (now age 5.5 - 17.5 years); age range 3.0-10.0 years for the New Cohort
• Weight at least 10.0 kg at the time of enrollment
• Parent or guardian willing and able to provide written informed consent, with child's verbal assent as per local IRB/Ethics Board requirements
• Willingness to comply with all study-related treatments, evaluations, and follow-up