Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases (MiniClini)
Bone Marrow Failure Syndromes, Immunodeficiencies, Immune Dysregulation Syndromes
About this trial
This is an interventional treatment trial for Bone Marrow Failure Syndromes focused on measuring Transplant related mortality, Graft versus host disease, Bone marrow transplant
Eligibility Criteria
Inclusion Criteria:
- Bone marrow failure syndromes for which SCT is indicated, including severe aplastic anemia refractory to non transplant therapies congenital neutropenia, congenital thrombocytopenia, congenital red cell aplasia
- Immunodeficiencies for which allogeneic hematopoietic stem cell transplant is indicated, including severe combined immunodeficiencies, Wiskott-Aldrich syndrome, IPEX syndrome, X-linked lymphoproliferative disease
- Immune dysregulation syndromes, including refractory or recurrent hemophagocytic lymphohistiocytosis, HLH with genetic mutations, refractory multisystemic Langerhans cell histiocytosis, other MAS refractory to standard therapy
- Organ function clearance
Exclusion Criteria:
- Uncontrolled bacterial, viral or fungal infections
- HLA matched related or unrelated donor able to donate mobilized peripheral stem cells.
- Fanconi's syndrome, dyskeratosis congenita or other chromosomal fragility syndromes
- Pregnant Females
Sites / Locations
- The Children's Hospital of Philadelphia
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Bone Marrow Failure Syndrome
Immunodeficiency / Dysregulation
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.
Reduced intensity conditioning with chemotherapy followed by stem cell transplant using the CliniMACs device to deplete CD3+ CD19+ peripheral stem cells. Reduced intensity conditioning will include Busulfan, Fludarbine, Cyclophosphamide followed by stem cell infusion.