Drug intelligence / Profile preview

Haploidentical Bone Marrow Transplantation

Development stage
Phase 2
Lead developer
Johns Hopkins Medicine
Modality
Stem Cell Therapies → Cell Therapies
Administration
Intravenous
01

Overview

Haploidentical Bone Marrow Transplantation (Haplo-BMT) is an allogeneic hematopoietic stem cell transplant procedure utilizing a donor who is a partial human leukocyte antigen (HLA) match, typically a biological parent, child, or sibling. This specific protocol, evaluated by the Medical College of Wisconsin in the BMT CTN 0603 trial, employs a nonmyeloablative conditioning regimen consisting of fludarabine, cyclophosphamide, and low-dose total body irradiation (TBI). To mitigate the high risk of graft-versus-host disease (GVHD) and graft rejection associated with HLA mismatch, the protocol utilizes high-dose post-transplantation cyclophosphamide (PTCy) on days 3 and 4. PTCy selectively depletes proliferating alloreactive T cells from the donor graft while sparing non-alloreactive T cells and hematopoietic stem cells, facilitating stable engraftment and a potent graft-versus-tumor effect in patients with leukemia and lymphoma who lack a fully matched donor.

Other names
Haploidentical BMTPartially HLA-Mismatched Bone Marrow TransplantationNonmyeloablative Haploidentical BMT

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