Busulfan, Antithymocyte Globulin, and Fludarabine Followed By 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 Firs

• Status: Completed
• Trial end date: 2004-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of California, San Francisco
• Collaborator: National Cancer Institute (NCI)
• Treatments: Antilymphocyte Serum; Busulfan; Fludarabine; Fludarabine phosphate

Criteria

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:

• Closely matched related donor

• Unresponsive to immunosuppressive therapy within 3 months after follow-up AND alternative matched unrelated donor available

• Congenital marrow failure syndrome, including any of the following:

• Primary red blood cell aplasia (Diamond-Blackfan syndrome)

• Congenital neutropenia (Kostmann's syndrome)

• Amegakaryocytic thrombocytopenia

• Hemoglobinopathy including any of the following:

• β-thalassemia major

• Sickle cell anemia

• Severe immunodeficiency disease including any of the following:

• Chediak-Higashi disease

• Wiskott-Aldrich syndrome

• Combined immunodeficiency disease (Nezelof's)

• Hyperimmunoglobulin M syndrome

• Bare lymphocyte syndrome

• Other stem cell defects (e.g., osteopetrosis)

• Chronic myelogenous leukemia in first chronic phase

• Not eligible for other ongoing phase II/III studies

• Acute myeloid leukemia in first remission

• Not eligible for other ongoing phase II/III studies

• 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:

• Bone marrow matched by high resolution DNA typing at both HLA Drβ1 alleles and ≤ 1 mismatch by high resolution DNA typing at the 4 HLA-A and -B alleles

• Umbilical cord blood matched at 4/6 HLA-A, -B, and Drβ1 alleles by high resolution typing with ≥ 1 Drβ1 match and ≥ 3 X 10^7 cells/kg body weight of recipient

PATIENT CHARACTERISTICS:

• See Disease Characteristics

• No active bacterial, viral, or fungal infection

• Cardiac shortening fraction ≥ 27%

• Creatinine clearance ≥ 60 mL/min

• DLCO ≥ 60% of predicted (corrected for anemia/lung volume)

PRIOR CONCURRENT THERAPY:

• See Disease Characteristics