Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect).
PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.
研究概览
地位
详细说明
OBJECTIVES:
- To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell transplantation after reduced-intensity conditioning regimen comprising busulfan, fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure syndromes.
OUTLINE:
- Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7 and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30 minutes starting 30 minutes before ATG on days -4 to -1.
- HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.
- Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4 hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the absence of GVHD, cyclosporine is tapered starting between days 30 to 60.
After completion of study treatment, patients are followed periodically for 1 year.
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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-
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Seoul、大韩民国、138-736
- Asan Medical Center - University of Ulsan College of Medicine
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
DISEASE CHARACTERISTICS:
Diagnosis of any of the following bone marrow failure syndromes:
Severe aplastic anemia, meeting 1 of the following criteria:
- Not responsive to immunosuppressive therapy
- With recurrent cytopenia after immunosuppressive therapy or allogeneic hematopoietic cell transplantation
Low-risk myelodysplastic syndrome, including any of the following:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- Refractory cytopenia with multi-lineage dysplasia
Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
- With thrombotic episodes
- With severe cytopenia
No willing, suitable HLA-compatible donor in family or in donor registries
- Related donor with HLA-haploidentical mismatch at three or less of 6 loci
- Patients with very severe neutropenia (< 200/μL) or febrile episodes, who feel urgent need for allogeneic hematopoietic cell transplantation, are eligible without a search for HLA-matched unrelated donors
PATIENT CHARACTERISTICS:
- Karnofsky performance status 70-100%
- Bilirubin < 2.0 mg/dL
- AST < 3 times upper limit of normal
- Creatinine < 2.0 mg/dL
- Ejection fraction > 40% by MUGA scan
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Donor cell engraftment
大体时间：10-35 days after transplantation
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neutrophil count over 500/ul
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10-35 days after transplantation
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria
大体时间：0-60 months after transplantation
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various toxicities of treatment
|
0-60 months after transplantation
|
|
Acute and chronic GVHD
大体时间：15-100 days; 100 days to 4 years
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ocurrence of acute or chronic GVHD after transplantation
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15-100 days; 100 days to 4 years
|
|
overall survival
大体时间：0-60 months
|
patients surviving after transplantaion
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0-60 months
|
|
event-free survival
大体时间：0-60 months after transplatation
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patients undergoing transplantation and maintaining donor hematopoiesis
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0-60 months after transplatation
|
合作者和调查者
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (估计)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
关键字
其他相关的 MeSH 术语
其他研究编号
- CDR0000600351
- AMC-UUCM-2008-0038
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