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Haploidentical donor bone marrow transplant
- Development stage
- Unknown
- Lead developer
- Blood and Marrow Transplant Clinical Trials Network
- Modality
- Small Molecules, Stem Cell Therapies → Cell Therapies, 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
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