Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
Metabolism, Inborn Errors, Granulomatous Disease, Chronic
About this trial
This is an interventional treatment trial for Metabolism, Inborn Errors
Eligibility Criteria
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Inherited disorders treatable with allogeneic peripheral blood or bone marrow transplantation At high risk for regimen related toxicity with a conventional transplant No severe CNS involvement of disease, defined by IQ score less than 70 HLA matched donor Sibling donors must be a confirmed match at HLA-A, B, and DRB1 Other related and non-related donors must be matched at HLA-A, B, C, DRB1, and DQB1 A donor homozygous for one allele only at HLA-A, B, C, DRB1, or DQB1 allowed (1 antigen mismatch for graft-versus-host disease, 0 antigen mismatch for graft-rejection) --Prior/Concurrent Therapy-- No concurrent growth factors with mycophenolate mofetil --Patient Characteristics-- Age: Under 55 Performance status: Not specified Life expectancy: At least 100 days Hematopoietic: Not specified Hepatic: No evidence of synthetic dysfunction No severe cirrhosis Renal: Not specified Cardiovascular: LVEF at least 30% No poorly controlled hypertension on multiple antihypertensives Other: No organ dysfunction that would preclude survival Not pregnant or nursing Fertile patients must use effective contraception during and for 12 months following study
Sites / Locations
- Fred Hutchinson Cancer Research Center