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| ID | Type | Description | Link |
|---|---|---|---|
| AMC-UUCM-2008-0038 |
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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.
OBJECTIVES:
OUTLINE:
After completion of study treatment, patients are followed periodically for 1 year.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| nonmyeloablative allogeneic hematopoietic stem cell transplantation | Procedure | infusion of mobilized donor hematopoietic progenitor cells |
| Measure | Description | Time Frame |
|---|---|---|
| Donor cell engraftment | neutrophil count over 500/ul | 10-35 days after transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria | various toxicities of treatment | 0-60 months after transplantation |
| Acute and chronic GVHD | ocurrence of acute or chronic GVHD after transplantation |
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DISEASE CHARACTERISTICS:
Diagnosis of any of the following bone marrow failure syndromes:
Severe aplastic anemia, meeting 1 of the following criteria:
Low-risk myelodysplastic syndrome, including any of the following:
Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
No willing, suitable HLA-compatible donor in family or in donor registries
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
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| Name | Affiliation | Role |
|---|---|---|
| Kyoo H. Lee, MD | Asan Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Asan Medical Center - University of Ulsan College of Medicine | Seoul | 138-736 | South Korea |
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| 15-100 days; 100 days to 4 years |
| overall survival | patients surviving after transplantaion | 0-60 months |
| event-free survival | patients undergoing transplantation and maintaining donor hematopoiesis | 0-60 months after transplatation |
| ID | Term |
|---|---|
| D007938 | Leukemia |
| D009190 | Myelodysplastic Syndromes |
| D009369 | Neoplasms |
| D006457 | Hemoglobinuria, Paroxysmal |
| D000753 | Anemia, Refractory |
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D000080983 | Bone Marrow Failure Disorders |
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