Hematopoietic Stem Cell Transplantation for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Doppler Ultrasound Using Reduced-Intensity Conditioning and T-Cell Depleted HSC From Partially Matched Family Donors
Secondary objectives for this protocol include the following:
- To estimate 1-year overall and event free survival after transplantation. An event is
defined as toxicity (graft failure, death, grade III/IV acute GHVD), or a
sickle-related event (stroke, acute chest syndrome, pain crisis).
- To obtain preliminary information regarding donor engraftment among different cell
subsets, including unsorted mononuclear cell, and lymphoid fractions during the first
year after transplant.
- To observe the rate of acute and chronic GVHD during the first year after transplant.
- To assess the proportion of research participants who experience poor graft integrity
and therefore require additional donor stem cells or lymphocytes.
- To document the effect of stem cell transplant on the central nervous system as defined
by radiological imaging and neuropsychological testing.
- To investigate immune reconstitution after transplantation
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
To assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy.
Wing Leung, M.D.
St. Jude Children's Research Hospital
United States: Food and Drug Administration
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