Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease
Status:
Completed
Trial end date:
2007-10-01
Target enrollment:
Participant gender:
Summary
This protocol studied the effect of administration of a myeloablative pretransplant
preparative regimen followed by an infusion of donor stem cells in children with severe
sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a
genetically matched sibling.
The primary aim of the study was to evaluate how well the donated cells migrated to the bone
marrow and begin producing healthy red blood cells, white blood cells and platelets
(engrafted), how well the recipients immune system recovered, and assess any regimen related
toxicities including a potentially life-threatening transplant related complication called
graft-versus-host-disease or GVHD.