Sickle Cell Disease Clinical Trial
Official title:
Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease
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.
The secondary objectives of this protocol evaluated the effect of this transplant procedure on the subsequent clinical course of patients with severe SCD. Specifically, to determine whether pre-transplant organ dysfunction (brain, heart, lung, kidney, liver, spleen, etc) resultant from sickle hemoglobinopathy can be reversed following this particular transplant procedure. ;
Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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