allogeneic hematopoietic stem cells (Washington University School of Medicine)

Allogeneic hematopoietic stem cells are used as a curative cell therapy for patients with severe sickle cell disease (SCD). This specific therapeutic approach, developed and evaluated by the Washington University School of Medicine, utilizes a non-myeloablative conditioning regimen to facilitate the engraftment of stem cells from mismatched donors, including haploidentical relatives or mismatched unrelated donors. The conditioning regimen typically includes alemtuzumab, cyclophosphamide, fludarabine, and low-dose total body irradiation, which suppresses the recipient's immune system enough to allow donor cell survival without the extreme toxicity of traditional myeloablative protocols. Once engrafted, the donor hematopoietic stem cells produce healthy red blood cells containing normal hemoglobin, effectively replacing the recipient's sickle-prone erythropoiesis and mitigating the clinical complications of the disease.

Other names

allogeneic hematopoietic stem cell transplantationmismatched hematopoietic stem cell transplantationhaploidentical hematopoietic stem cell transplantation