A Multicenter Study Comparing the Results of Allogeneic Stem Cell Genoidentical in Children With Sickle Cell Anemia and Cerebral Vascular Disease Detected by Transcranial Doppler
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 63
- 试验地点
- 1
- 主要终点
- Cerebral vasculopathy
研究概览
简要总结
The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients.
详细描述
The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD)are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized, prospective study of paired cohort will be conducted, with 2 groups of exposed (SCT) and non-exposed (TP) patients. Sixty-three patients will be enrolled, namely 21 sickle-cell patients in the "SCT" group and 42 in the "Transfusion Program (TP)" group
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Sickle cell anemia patients (SS/Sb0)
- •< 15 years old
- •History of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)
- •Siblings from the same parental couple
- •Parents amenable to Human Leucocyte Antigen (HLA) typing, SCT if an HLA-identical sibling is available or to long-term transfusion program
排除标准
- •Sickle cell patients older than 15 years
- •no history of abnormal transcranial doppler (TCD) (≥ 200 cm/sec)
- •no sibling from the same parental couple
- •parents averse to HLA typing, SCT if an HLA-identical sibling is available or to long-term transfusion program
结局指标
主要结局
Cerebral vasculopathy
时间窗: 1 year
velocity in the artery with highest velocity
次要结局
- ischemic lesions on magnetic resonance imaging (MRI)(1 year)
- stenoses on magnetic resonance imaging (MRI)(1 year)
- normalisation of arterial velocities(12 months)