Stem Cell Transplant for Bone Marrow Failure Syndromes

Inclusion Criteria: * Patients eligible for transplantation under this protocol will be \<35 years of age, and will be diagnosed with: * a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia. * Diamond Blackfan Anemia: * Patients must show evidence of steroid resistance requiring equivalent of \>6 transfusions yearly despite steroid therapy. * Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation. * Kostmann's Neutropenia, Shwachman-Diamond syndrome: * Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia. * Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) \>750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (\<4 /year). Exclusion Criteria: * Patients \>35 years of age * Karnofsky score \<70% * Hepatic dysfunction as determined by bilirubin \>3.0, ALT \>150, or active hepatitis * Pulmonary function …