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Haploidentical donor bone marrow transplant
- Development stage
- Unknown
- Lead developer
- Blood and Marrow Transplant Clinical Trials Network
- Modality
- Stem Cell Therapies → Cell Therapies, Small Molecules, Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
This therapeutic approach involves a bone marrow transplant from a haploidentical (half-matched) related donor for the treatment of severe aplastic anemia (SAA). The regimen, specifically studied by the Medical College of Wisconsin in the BMT CTN 2207 (CureAA) trial, utilizes a non-myeloablative conditioning protocol consisting of antithymocyte globulin (ATG), fludarabine, low-dose cyclophosphamide, and total body irradiation (TBI). To prevent graft-versus-host disease (GVHD) and promote engraftment, the protocol incorporates post-transplantation high-dose cyclophosphamide (PTCy), tacrolimus, and mycophenolate mofetil. This strategy aims to provide a curative option for patients with newly diagnosed SAA who lack a matched sibling donor, leveraging the donor's hematopoietic stem cells to reconstitute the recipient's bone marrow function.
- Other names
- Haploidentical hematopoietic stem cell transplantHaplo-HSCTHaploidentical BMT
02
Targets
CD2 (T-cell Surface Antigen CD2)IL2RA (Interleukin-2 receptor alpha subunit)CD3 (T-cell surface glycoprotein CD3)CD4 (T lymphocyte surface antigen)CD44 (CD44 antigen)PTPRC (Receptor-type tyrosine-protein phosphatase C)CD8A (CD8 alpha/beta Coreceptor)HLA-DR (Human leukocyte antigen DR)ITGAL (Integrin alpha L (CD11a))ITGB2 (Complement receptor 4)MHC I (MHC class I})ITGB1 (Integrin beta-1)MR1 (Thioesterase PNKD)CD86 (T-lymphocyte activation antigen CD86)POLA1 (DNA polymerase alpha)DNA-directed primase/polymerase protein (PrimPol)DCK (Deoxycytidine kinase)RRM1N7-G (DNA guanine-N7 adduct)DNACSF3R (Granulocyte colony-stimulating factor receptor)
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