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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.
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | hydroxyurea |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| hydroxyurea | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Transcranial doppler ultrasound velocity | 2 years | |
| Magnetic resonance imaging/angiography | 2 years | |
| Glomerular Filtration Rate | 2 years | |
| Quality of Life | 2 years | |
| Neurocognitive outcomes | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Growth parameters | 2 years | |
| Hematological parameters | 2 years |
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Inclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Courtney D Thornburg, MD mS | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medican Center | Durham | North Carolina | 27710 | United States |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D006918 | Hydroxyurea |
| ID | Term |
|---|---|
| D014508 | Urea |
| D000577 | Amides |
| D009930 | Organic Chemicals |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |