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| ID | Type | Description | Link |
|---|---|---|---|
| UCSF-01152 | |||
| UCSF-H411-17802-06 |
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| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells. A donor peripheral blood, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving antithymocyte globulin before the transplant may stop this from happening.
PURPOSE: This phase I/II trial is studying the side effects of busulfan, antithymocyte globulin, and fludarabine when given together with a donor stem cell transplant in treating young patients with blood disorders, bone marrow disorders, chronic myelogenous leukemia in first chronic phase, or acute myeloid leukemia in first remission.
OBJECTIVES:
Primary
Secondary
OUTLINE: Patients receive one of the following cytoreductive regimens:
All patients undergo allogeneic bone marrow, umbilical cord blood, or peripheral blood stem cell transplantation on day 0.
After the completion of study treatment, patients are followed periodically for 20 years.
PROJECTED ACCRUAL: A total of 40 patients will be accrued for this study.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| anti-thymocyte globulin | Biological | |||
| busulfan | Drug | |||
| fludarabine phosphate | Drug | |||
| allogeneic bone marrow transplantation | Procedure | |||
| peripheral blood stem cell transplantation | Procedure | |||
| umbilical cord blood transplantation | Procedure | |||
| radiation therapy | Radiation |
| Measure | Description | Time Frame |
|---|---|---|
| Graft rejection measured by ANC < 500 with no evidence of donor cells in blood or marrow from transplantation to week 4 post transplantation |
| Measure | Description | Time Frame |
|---|---|---|
| Toxicity grades 3 or 4 assessed from conditioning through 1 year post transplantation | ||
| Engraftment at 1, 3, 6, 9, and 12 months post transplantation | ||
| Mixed chimerism at 1, 3, 6, 9, and 12 months post transplantation |
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DISEASE CHARACTERISTICS:
Diagnosis of one of the following hematopoietic disorders:
Severe aplastic anemia with marrow aplasia (i.e., absolute neutrophil count < 500/mm^3, platelet and/or red blood cell transfusion dependent), meeting 1 of the following criteria:
Congenital marrow failure syndrome, including any of the following:
Hemoglobinopathy including any of the following:
Severe immunodeficiency disease including any of the following:
Other stem cell defects (e.g., osteopetrosis)
Chronic myelogenous leukemia in first chronic phase
Acute myeloid leukemia in first remission
Inborn errors of metabolism
No severe combined immunodeficiency disorder
Available donor, meeting 1 of the following criteria:
Related donor matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch at the 4 HLA-A and -B alleles
Unrelated donor, meeting one of the following criteria:
PATIENT CHARACTERISTICS:
PRIOR CONCURRENT THERAPY:
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| Name | Affiliation | Role |
|---|---|---|
| Morton J. Cowan, MD | University of California, San Francisco | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Comprehensive Cancer Center | San Francisco | California | 94115 | United States |
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| Survival measured from the day of first dose of conditioning |
| Disease-free survival measured from the day of first dose of conditioning |
| ID | Term |
|---|---|
| C535982 | Congenital amegakaryocytic thrombocytopenia |
| D029503 | Anemia, Diamond-Blackfan |
| D005199 | Fanconi Anemia |
| D007938 | Leukemia |
| C537592 | Neutropenia, Severe Congenital, Autosomal Recessive 3 |
| D013921 | Thrombocytopenia |
| D015466 | Leukemia, Myeloid, Chronic-Phase |
| ID | Term |
|---|---|
| D029502 | Anemia, Hypoplastic, Congenital |
| D000741 | Anemia, Aplastic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D012010 | Red-Cell Aplasia, Pure |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D049914 | DNA Repair-Deficiency Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D007951 | Leukemia, Myeloid |
| D009196 | Myeloproliferative Disorders |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D000961 | Antilymphocyte Serum |
| D002066 | Busulfan |
| C042382 | fludarabine phosphate |
| D036102 | Peripheral Blood Stem Cell Transplantation |
| D036101 | Cord Blood Stem Cell Transplantation |
| D011878 | Radiotherapy |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
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