This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.
Considered appropriate candidate for allogeneic HCT following low dose (4Gy) TBI containing-conditioning and have one of the following diagnoses: A) BMF B)Hemoglobinopathies C)PID D) Autoimmune cytopenias E) Immune dysregulation F) HLH G) Other NMD treatable by HCT and NMD that is not clearly defined (a patient with a NMD for whom genetic testing has been done and a genetic mutation responsible for their NMD phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol PI
Patients aged 6 months- 5 years old (inclusive) at the time of informed consent
Patient with suitable HCT donor (see inclusion criteria below)
Recipient informed consent/assent (13 years and older), and/or legal guardian permission must be obtained
Patient with aplastic anemia
Patients with severe combined immunodeficiency (SCID)
Fanconi anemia
Dyskeratosis congenita
Parallel
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Biological/VaccineCD34-selected graft with CD45RA- depleted peripheral blood stem cells given to patients with Non-Malignant Diseases
GVHD-free Survival
Free of grade III-IV acute and NIH chronic (moderate-severe) GVHD requiring systemic immunosuppression
Overall survival
Transplant related mortality
Graft failure
Graft failure defined as failure to achieve an ANC ≥ 0.5 × 109/L before death or second HCT, or decrease to ANC \<0.1 × 109/L for 14 consecutive days (date of graft failure defined as the 14th day) after an established donor graft despite daily administration of G-CSF (SC or IV) and ≤ average 20% bone marrow cellularity on bone marrow aspirate or biopsy any time in the first 2 years following HCT. If a patient dies from organ toxicity and/or infection prior to day 28 without ANC ≥ 0.5 × 109/L this will not be considered graft failure. If the graft failure is attributed to viral infection, multi-organ failure or drug effect it will still be considered graft failure if it meets the definition of graft failure specified above.
Graft rejection
Graft rejection defined as \<5% donor CD3 T cell and CD33 myeloid chimerism
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