Haploidentical hematopoietic stem cell transplantation (St. Jude protocol)

This specialized hematopoietic stem cell transplantation (HSCT) protocol was developed by St. Jude Children's Research Hospital for the treatment of severe sickle cell disease (SCD) in pediatric patients with high-risk features, such as prior stroke or abnormal transcranial Doppler (TCD) results. The procedure involves the transplantation of stem cells from a partially HLA-matched (haploidentical) family donor. To minimize the risk of graft-versus-host disease (GVHD), the donor graft is processed using the CliniMACS device for CD34+ cell enrichment and T-cell (CD3+) depletion. The protocol utilizes a reduced-intensity conditioning (RIC) regimen consisting of busulfan, cyclophosphamide, thiotepa, and muromonab-CD3 (OKT3), supplemented by pre-transplant immunosuppression with hydroxyurea and azathioprine. Clinical development of this specific protocol was terminated after meeting stopping rules related to high rates of graft failure and rejection.

Other names

Haploidentical HSCTT-cell depleted haploidentical HSCTHaploidentical HSCT in Sickle Cell Disease With Stroke or Abnormal TCD