Cytosine Arabinoside and Mitoxantrone for Patients With Juvenile Myelomonocytic Leukemia Receiving Repeat Stem Cell Transplantation
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Disease-free Survival
研究概览
简要总结
RATIONALE: Giving chemotherapy drugs, such as cytarabine and mitoxantrone, before a donor stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine, methotrexate, and methylprednisolone before or after transplant may stop this from happening.
PURPOSE: This phase I/II trial is studying the side effects and best way to give high-dose cytarabine together with mitoxantrone in treating patients with juvenile myelomonocytic leukemia undergoing a second donor stem cell transplant.
详细描述
OBJECTIVES:
Primary
- To determine the incidence of 1-year disease-free survival in patients with juvenile myelomonocytic leukemia and who is undergoing a repeat stem cell transplantation.
Secondary
- To evaluate the incidence of regimen-related toxicity.
- To evaluate the incidence of acute and chronic graft-versus-host-disease.
- To evaluate the incidence of relapse.
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients age 0-18 with juvenile myelomonocytic leukemia (JMML) who have relapsed or have residual disease after allogeneic HCT. Residual disease is defined as failure to eradicate original disease without prior documentation of remission. Relapse is defined as reappearance of i) leukocytosis with absolute monocytosis >1 x 10^8/L, ii) presence of immature myeloid cells in the peripheral circulation in two consecutive bone marrow specimens taken at least one month apart, or iii) presence of clonal cytogenetic abnormality. The diagnosis of relapse will be supported by the return of an abnormal cytogenetic marker (if present at diagnosis) or the presence of host cells by RFLP or other method.
- •Patients should be at least 6 months from first hematopoietic cell transplant (HCT) if clinically stable. (If JMML is rapidly progressive, second HCT may be performed earlier).
- •Adequate major organ function including:
- •Cardiac: ejection fraction ≥45%
- •Pulmonary: FEV >50%, DLCO >50%
- •Renal: creatinine clearance ≥40 mL/min
- •Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
- •Karnofsky performance status ≥70% or Lansky score ≥50%
- •Written informed consent.
排除标准
- •Active uncontrolled infection within one week of HCT.
研究组 & 干预措施
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: cyclosporine (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: cytarabine (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: filgrastim (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: methotrexate (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: methylprednisolone (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: mitoxantrone hydrochloride (Drug)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: allogeneic bone marrow transplantation (Procedure)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: umbilical cord blood transplantation (Procedure)
Cytarabine + Mitoxantrone
This is a phase I-II study designed to evaluate the efficacy of the administration of high dose cytosine arabinoside and mitoxantrone followed by HCT in patients with JMML who have residual disease or have relapsed after initial HCT.
干预措施: Cis-Retinoic acid (Drug)
结局指标
主要结局
Disease-free Survival
时间窗: 1 year
Number of patients who were free of disease and alive at 1 year.
次要结局
- Patients With Graft-Versus-Host-Disease(Up to 30 Days Post Study Treatment)
- Patients With Regimen-Related Toxicity(Up to 30 Days Post Study Treatment)
- Patients Who Relapsed(1 Year)