Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia
Overview
- Phase
- Not Applicable
- Intervention
- hydroxyurea
- Conditions
- Sickle Cell Anemia
- Sponsor
- Duke University
- Enrollment
- 14
- Locations
- 1
- Primary Endpoint
- Transcranial doppler ultrasound velocity
- Status
- Completed
- Last Updated
- 18 years ago
Overview
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
Investigators
Eligibility Criteria
Inclusion Criteria
- •Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
Exclusion Criteria
- Not provided
Arms & Interventions
1
hydroxyurea
Intervention: hydroxyurea
Outcomes
Primary Outcomes
Transcranial doppler ultrasound velocity
Time Frame: 2 years
Magnetic resonance imaging/angiography
Time Frame: 2 years
Glomerular Filtration Rate
Time Frame: 2 years
Quality of Life
Time Frame: 2 years
Neurocognitive outcomes
Time Frame: 2 years
Secondary Outcomes
- Growth parameters(2 years)
- Hematological parameters(2 years)