Haematological Disorders
Conditions
Interventions
Hydroxyurea
None
Sponsors
Cincinnati Childrens Hospital Medical Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Pediatric patients with documented sickle cell anemia (typically HbSS supported by hemoglobin electrophoresis, complete blood count, and peripheral blood smear) 2. Age range of 1.00-9.99 years, inclusive, at the time of enrollment 3. Weight at least 10.0 kg at the time of enrollment 4. Parent or guardian willing and able to provide written informed consent, with child's verbal assent as per local IRB/Ethics Board requirements 5. Willingness to comply with all study-related treatments, evaluations, and follow-up
Exclusion criteria
Exclusion criteria: 1. Known medical condition making participation ill-advised, (e.g., acute or chronic infectious disease, HIV, or malignancy) 2. Acute or chronic severe malnutrition determined by impaired growth parameters as defined by WHO (weight for length/height or weight-for-length/height >3 z-scores below the median WHO growth standards, as defined in Appendix I) 3. Pre-existing severe hematological toxicity (temporary exclusions) a. Anemia: Hb <4.0 gm/dL b. Anemia: Hb <6.0 gm/dL with ARC <100 x 109/L c. Reticulocytopenia: ARC <80 x 109/L with Hb <7.0 gm/dL d. Thrombocytopenia: Platelets <80 x 109/L e. Neutropenia: ANC <1.0 x 109/L 4. Blood transfusion within 60 days before enrollment (temporary exclusion) 5. Hydroxyurea use within 6 months before enrollment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of Participants With Dose Limiting Toxic Events An expected toxicity rate of 20% and acceptable toxicity rate of 30% were used for statistical calculations. After 53 participants at each site complete 3 months of therapy, if = 15 participants have hematologic toxicity there is no early evidence against safety. If = 15 of the initial participants experience toxicity, this is early evidence against safety. Future participants will begin at a lower dose of hydroxyurea (10 ± 2.5 mg/kg), with another 53 participants recruited of the same safety analysis. Upon final analysis of 133 participants at the same starting dose, safety for fixed-dose hydroxyurea can be concluded | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy of Hydroxyurea The efficacy of hydroxyurea will be primarily assessed through fetal hemoglobin (HbF), comparing treatment with baseline values. Additional measures of laboratory efficacy will include changes in Hb, MCV, WBC, ANC, ARC, and bilirubin. Clinical events such as vaso-occlusive pain will be captured as secondary outcomes.;Medication Adherence and the Ability for Families to Adhere to Monthly Clinic Visits Hydroxyurea treatment will be dispensed only 35 days at a time, requiring a clinic visit every 4 ± 1 weeks. Medication adherence and the ability for families to adhere to monthly clinic visits are important feasibility outcomes | — |
Countries
Angola, Congo, Kenya, Uganda
Contacts
Public ContactSusan Stuber
Project Manager
Outcome results
None listed