allogeneic hematopoietic stem cell transplantation (St. Jude Children's Research Hospital)

Allogeneic hematopoietic stem cell transplantation (HCT) at St. Jude Children's Research Hospital for sickle cell disease (SCD) is a specialized therapeutic protocol utilizing a reduced-intensity conditioning (RIC) regimen. This approach is designed for children and young adults with severe SCD to achieve stable donor chimerism while minimizing the toxicities associated with traditional myeloablative conditioning. The protocol employs a combination of hydroxyurea, azathioprine, alemtuzumab, thiotepa, and low-dose total body irradiation (TBI) to prepare the recipient's bone marrow. The transplant uses G-CSF mobilized peripheral blood stem cells (PBSCs) from either a matched sibling donor (MSD) or a haploidentical (HAPLO) donor. Post-transplant management includes sirolimus for graft-versus-host disease (GVHD) prophylaxis and donor lymphocyte infusions (DLI) to ensure long-term engraftment and cure of the underlying hemoglobinopathy.

Other names

allogeneic hematopoietic stem cell transplant-St. Jude Children's Research Hospital-sickle cell diseaseRIC HCT for Severe Sickle Cell DiseaseReduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation