Antithymocyte Globulin (ATG) and Cyclosporine (CSA) to Treat Patients With Myelodysplastic Syndrome (MDS). A Randomized Trial Comparing ATG + CSA With Best Supportive Care
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 86
- 试验地点
- 1
- 主要终点
- Best response rate (CR + PR)
研究概览
简要总结
The main objective of this trial is to evaluate the efficacy and toxicity of intensive immuno-suppression with ATG + CSA versus best supportive care in patients with transfusion dependent low and intermediate risk myelodysplastic syndrome (MDS).
详细描述
This trial will answer the question whether immuno-suppression improves hematopoesis and reduces transfusion requirements analogous to patients with aplastic anemia as the short-term outcome, and whether immuno-suppression accelerates leukemic transformation and influences survival as the long-term outcome.
Primary endpoint: best response (CR + PR) rate at month 6
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Arm A: ATG + CSA
Treatment consists of 15 mg/kg ATG (Mérieux; horse antithymocyte globulin; i.e. 1.5 vial/10 kg of body weight/day) given over 8-12 hours for 5 consecutive days.
Cyclosporine A (CSA) will be administered orally in a dose of 2.5 mg/kg bid starting day 1 and continued through day 180.
干预措施: ATG + CSA (Drug)
Arm B: Supportive care
Patients randomized to this arm will be treated as outpatients.
干预措施: Supportive care (Behavioral)
结局指标
主要结局
Best response rate (CR + PR)
时间窗: 6 months
次要结局
- Leukemia-free survival(2 and 5 years)
- Response rate at month 3, time to response, quality and duration of response, and proportions of relapse/progression in responders(2 and 5 years after first response)
- Overall survival(2 and 5 years)
- Transformation-free survival(2 and 5 years)