2-step haploidentical hematopoietic stem cell transplantation

This is a specialized haploidentical hematopoietic stem cell transplantation (HSCT) regimen being evaluated by Northwell Health for children and young adults with high-risk hematologic malignancies. The "two-step" approach is designed for patients who lack a fully matched human leukocyte antigen (HLA) donor, utilizing a half-matched related donor instead. The protocol involves an initial infusion of donor T-cells (Donor Lymphocyte Infusion) to provide a graft-versus-leukemia effect, followed by the administration of high-dose cyclophosphamide to selectively deplete alloreactive T-cells and induce immunological tolerance. This step is critical for preventing graft-versus-host disease (GVHD). Subsequently, CD34-selected hematopoietic stem cells from the donor are transplanted to achieve stable engraftment and immune reconstitution. This regimen aims to expand donor availability while minimizing the risks of severe GVHD and graft failure.

Other names

haploidentical hematopoietic stem cellsHLA-mismatched related donor HSCThaploidentical HSCT