Hematologic Diseases Clinical Trial
Official title:
In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders
The hypothesis is to determine if a preparative regimen of busulfan, cyclophosphamide, and antithymocyte globulin (ATG) plus allogeneic stem cell transplantation will be effective in the treatment of immune deficiencies and histiocytic disorders.
Subjects will begin chemotherapy as a preparative regimen, which is intended to completely
eliminate their defective immune system and bone marrow. The preparative regimen consists of
the chemotherapy drugs (busulfan, cyclophosphamide, and antithymocyte globulin (ATG)).
Transplantation: subjects will then have a source of blood stem cells (bone marrow) from
their donor administered into their catheter. Medication will be given to help prevent
Graft-Versus Host Disease (GVHD). The ATG will help to deplete the donor stem cells of the
type of cells that can cause GVHD and will also help to promote engraftment of the new stem
cells.
Recovery Phase: The second phase of treatment consists of a period after transplantation
during which we wait for the return of bone marrow function. This usually takes two to four
weeks. Subjects will be given a blood cell growth factor, G-CSF, to help speed recovery of
the white blood cells and potentially decrease the risk of infection and decrease the time
until the bone marrow recovers.
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