Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

• Status: Completed
• Trial end date: 2009-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: Hydroxyurea

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