This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Diagnosis of Fanconi anemia
Age <65 years of age
Severe aplastic anemia (SAA)
Myelodysplastic features
Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
Active, uncontrolled infection within 1 week prior to starting study therapy
Malignant solid tumor cancer within previous 2 years
an HLA-A, B, DRB1 matched sibling donor (matched sibling)