Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Determine Probability of Long-term Disease Free Survival in JMML
研究概览
简要总结
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).
详细描述
Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation.
On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
- •Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)
- •The presence of immature myeloid cells in the peripheral blood
- •Less than 30% marrow blasts
- •Absence of t(9:22) or BCR-ABL transcript
- •Adequate major organ function including:
- •Cardiac: ejection fraction > 45%
- •Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- •Karnofsky performance status > 70% or Lansky score > 50%
- •Creatinine must be < 2 x normal for age
- •Written informed consent.
排除标准
- •Active uncontrolled infection within one week of HCT.
研究组 & 干预措施
Intent-to-Treat
Patients receiving study regimen.
干预措施: Preparative Regimen (Drug)
Intent-to-Treat
Patients receiving study regimen.
干预措施: Stem Cell Transplant (Biological)
结局指标
主要结局
Determine Probability of Long-term Disease Free Survival in JMML
时间窗: at 1 year after transplant
次要结局
- Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.(at 1 year after transplant)