Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
研究概览
简要总结
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood remains unclear. So we explore the ideal dosage of cyclophosphamide for the treatment of children with SAA.
详细描述
Tisdale et al. (2000,2002) attempted to compare immunosuppression using ATG/CSA with high-dose cyclophosphamide (50 mg/kg/d for 4 consecutive days) plus CSA in a randomized trial of newly diagnosed adults with SAA. Both groups received CSA as part of the treatment regimen. However, the trial was terminated prematurely due to excessive morbidity among the patients treated in the cyclophosphamide arm. They documented that invasive fungal infections were severe among the cyclophosphamide group. Between January 2008 through May 2009, in our department, nine pediatric patients with a diagnosis of SAA were enrolled a study with lower dose of cyclophosphamide with 30mg/kg/day for 4 consecutive days and combination with CSA, this study shows promise for children with severe aplastic anemia.Now we want explore the dosage of cyclophosphamide.
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
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Acquired Childhood Severe Aplastic Anemia (SAA)
排除标准
- •not Childhood and Acquired Severe Aplastic Anemia
研究组 & 干预措施
moderate-dose cyclophosphomide
干预措施: cyclophosphamide,cyclosporine A (Drug)
结局指标
主要结局
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
时间窗: 36 months
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count \>100×109/L, and ANC\>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count \>30×109/L, platelet count \>30×109/L, and ANC \>0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
次要结局
- The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia(36 months)
研究者
Xiaofan Zhu
chief physician
Chinese Academy of Medical Sciences