1)Mixed Chimerism to Treat Sickle Cell Disease: Extended Protocol 2)Mixed Chimerism to Treat Non-Malignant Disorders of the Hematopoietic Stem Cell 3)Transplantation of Allogeneic Bone Marrow Enriched for Graft Facilitating Cells in the Treatment of Aplastic Anemia
Hematopoietic stem cell transplantation (HSCT) is emerging as a therapeutic alternative for
patients with sickle cell disease. Conventional HSCT therapy has been limited to extremely
high-risk hemoglobinopathy patients. Those patients who may be difficult to identify before
end-organ damage develops.
Also, conventional HSCT is only available to the minority of candidates who have
Histocompatibility Leukocyte Antigen (HLA) identical siblings to donate bone marrow or
mobilized peripheral blood stem cells.
This study proposes two important improvements over conventional HSCT:
- Donor peripheral blood or bone marrow will be processed via a new technology, which
will deplete mature immune cells while enriching hematopoietic stem cells (HSC) and
graft facilitating cells (FC).
- A reduced intensity recipient conditioning regimen will be used to promote mixed
allogeneic chimerism, as opposed to full donor chimerism, following HSCT.
These two elements may significantly improve the benefit:risk ratio of HSCT for patients
with hemoglobinopathies. Stem cell transplantation may become a more feasible option for
patients that do not have HLA-identical siblings that can donate stem cells. Also,
transplantation may be offered to patients earlier in the disease progression, before
end-organ damage occurs.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Level of Donor Chimerism from Enriched Hematopoietic Stem Cell Engraftment
Tests are done at key time points to monitor for donor chimerism by evaluating presence of bone marrow-derived hematopoietic stem cells.
From one month to three years
Roger Herzig, M.D.
James Graham Brown Cancer Center
United States: Food and Drug Administration
|St. Christopher's Hospital for Children||Philadelphia, Pennsylvania 19134-1095|
|Duke University Medical Center||Durham, North Carolina 27710|
|University of Louisville||Louisville, Kentucky 40202|