Stem Cell Transplant in Patients With Severe Sickle Cell Disease

  • End date
    Feb 15, 2026
  • participants needed
  • sponsor
    Kathleen Dorritie
Updated on 1 May 2022
cell transplantation
hemoglobin s
transplant conditioning
blood transfusion
iron overload
acute chest syndrome
chest syndrome
sickle cell trait


This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab).


Stem cell transplantation recipients will be given Alemtuzumab, which is a non-myeloablative pre-transplant conditioning regimen. This non-myeloablative therapy uses doses of chemotherapy and radiation to weaken (but not destroy) the patients bone marrow and immune system, while still allowing their body to accept the donor's stem cells. Alemtuzumab will be given 7 days prior to stem cell infusion at 0.03 mg/kg IV, 6 days prior to stem cell infusion at 0.1 mg/kg IV, and 5 thru 3 days prior to stem cell infusion at 0.3 mg/kg IV.

Condition Sickle Cell Disease
Treatment Total body irradiation, Sirolimus, alemtuzumab
Clinical Study IdentifierNCT03421756
SponsorKathleen Dorritie
Last Modified on1 May 2022


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