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 First Remission
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia
About this trial
This is an interventional treatment trial for Congenital Amegakaryocytic Thrombocytopenia focused on measuring thrombocytopenia, childhood acute myeloid leukemia in remission, childhood chronic myelogenous leukemia, Diamond-Blackfan anemia, congenital amegakaryocytic thrombocytopenia, Fanconi anemia, severe congenital neutropenia, chronic phase chronic myelogenous leukemia
Eligibility 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
Sites / Locations
- UCSF Comprehensive Cancer Center