Sickle Cell Anemia
Conditions
Keywords
sickle cell anemia, hydroxyurea
Brief summary
The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage.
Detailed description
Previous studies have shown that hydroxyurea therapy in adults and older children with SCA improves laboratory parameters and ameliorates the clinical severity of disease. Little is known, however, about the effects of hydroxyurea on the chronic organ damage that occurs in patients with SCA and leads to significant morbidity and mortality in young adults. The objectives of this study are to assess the safety and efficacy of HU in young children with SCA and to determine whether HU preserves renal function, reduces transcranial doppler ultrasound (TCD) values, and prevents development of brain ischemia as evidenced by MRI/MRA imaging. In addition, we will evaluate the effects of hydroxyurea on quality of life
Interventions
DRUGhydroxyurea
Sponsors
Duke University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Months to 5 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Magnetic resonance imaging/angiography | 2 years |
| Quality of Life | 2 years |
| Neurocognitive outcomes | 2 years |
| Transcranial doppler ultrasound velocity | 2 years |
| Glomerular Filtration Rate | 2 years |
Secondary
| Measure | Time frame |
|---|---|
| Hematological parameters | 2 years |
| Growth parameters | 2 years |
Countries
United States
Outcome results
None listed