Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
Condition: Hematologic Diseases; Anemia, Sickle Cell
MeSH terms: Anemia; Anemia, Sickle Cell; Hematologic Diseases
Intervention: Hydroxyurea; Placebo
Intervention description: Participants will receive hydroxyurea.; Participants will receive placebo.
Intervention MeSH terms: Hydroxyurea
Phase: Phase 3
Study type: Interventional
Primary purpose: Prevention
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 193.0
Sex: All
Minimum age: 9 Months
Maximum age: 18 Months
Healthy volunteers: No
Keywords: Blood Diseases; Sickle Cell Anemia

Summary:

      The purpose of this study is to determine if hydroxyurea therapy is effective in the
      prevention of chronic end organ damage in pediatric patients with sickle cell anemia.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by
             electrophoresis (screening may begin at 7 months of age)

        Exclusion Criteria:

          -  Chronic transfusion therapy

          -  Cancer

          -  Less than 5th percentile (10th percentile for the pilot study) height, weight, or head
             circumference for age

          -  Severe developmental delay (e.g., cerebral palsy or other mental retardation, Grade
             III/IV intraventricular hemorrhage)

          -  Stroke with neurological deficit

          -  Surgical splenectomy

          -  Participating in other clinical intervention trials

          -  Probable or known diagnosis of Hemoglobin S-Hereditary Persistence of Fetal Hemoglobin

          -  Known hemoglobin S-beta plus thalassemia (hemoglobin A present)

          -  Any condition or chronic illness, which in the opinion of the principal investigator,
             makes participation unadvised or unsafe

          -  Inability or unwillingness to complete baseline (pre-enrollment) studies, including
             blood or urine specimen collection, liver-spleen scan, abdominal sonogram,
             neurological examination, neuropsychological testing, or transcranial Doppler
             ultrasound (interpretable study not required, but confirmed velocity greater than 200
             cm/sec results in ineligibility)

          -  Previous or current treatment with hydroxyurea (HU) or another anti-sickling drug

          -  The following exclusion criteria are transient; patients can be re-evaluated for
             eligibility:

               1. Hemoglobin less than 6.0 gm/dL

               2. Reticulocyte count less than 80,000/cu mm if hemoglobin is less than 9 gm/dL

               3. Neutrophil count less than 2,000/cu mm

               4. Platelet count less than 130,000/cu mm

               5. Blood transfusion in the 2 months prior to study entry unless HbA is less than
                  10%

               6. ALT greater than twice the upper limit of normal

               7. Ferritin less than 10 ng/ml

               8. Serum creatinine greater than twice the upper limit of normal for age

               9. Bayley standardized mental score below 70