Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Transcranial doppler ultrasound velocity
研究概览
简要总结
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.
详细描述
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 18 Months 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)
排除标准
- 未提供
研究组 & 干预措施
1
hydroxyurea
干预措施: hydroxyurea (Drug)
结局指标
主要结局
Transcranial doppler ultrasound velocity
时间窗: 2 years
Magnetic resonance imaging/angiography
时间窗: 2 years
Glomerular Filtration Rate
时间窗: 2 years
Quality of Life
时间窗: 2 years
Neurocognitive outcomes
时间窗: 2 years
次要结局
- Growth parameters(2 years)
- Hematological parameters(2 years)