Treatment of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 主要终点
- Event-free survival
研究概览
简要总结
RATIONALE: Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. Giving low-doses of cytarabine may be an effective treatment for Down syndrome and transient myeloproliferative disorder. Sometimes the disease may not need treatment until it progresses. In this case, observation may be sufficient.
PURPOSE: This phase III trial is studying low-dose cytarabine to see how well it works in treating infants with Down syndrome and transient myeloproliferative disorder.
详细描述
OBJECTIVES:
Primary
- Determine whether very low-dose cytarabine can improve event-free survival (EFS) rates in infants with high-risk transient myeloproliferative disorder (TMD), using high-risk TMD patients from clinical trial COG-A2971 for historic comparison, and in infants with intermediate-risk TMD, using intermediate-risk TMD patients from clinical trial COG-A2971 for historic comparison.
- Maintain the current high overall EFS rate in low-risk TMD patients.
Secondary
- Assess the toxicity of this regimen in these patients.
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
- 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
Group I
Patients receive very low-dose cytarabine subcutaneously twice daily on days 1-7. Treatment repeats every 14 days for up to 4 courses in the absence of disease progression or unacceptable toxicity. Patients achieving stable disease or complete or hepatic clinical remission undergo observation.
干预措施: cytarabine (Drug)
Group II
Patients are observed. If symptoms of intermediate- or high-risk disease develop, patients may crossover to group I.
干预措施: observation (Procedure)
结局指标
主要结局
Event-free survival
次要结局
- Percentage of patients experiencing grade 3-4 toxicity
- Incidence of subsequent leukemia in patients for whom transient myeloproliferative disorder is resolved
- Overall survival
- Disease-related mortality