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Aplastic Anemia

More About the Institute's Approach to BMT for Aplastic Anemia

Aplastic anemia is treated with either bone marrow transplantation or immunosuppressive drugs. High dose chemotherapy with allogeneic bone marrow transplantation is a treatment for severe aplastic anemia that has failed to respond to immunosuppressive therapy. The procedure is normally associated with considerable risk, and a number of life-threatening complications may occur, including graft failure (rejection) and acute and chronic Graft-versus-Host Disease (GVHD). Patients receiving HLA non-identical transplants are at highest risk for rejection and GVHD.

Previously, patients who received bone marrow transplants from poorly matched donors have had a high incidence of Graft vs. Host Disease (GVHD). GVHD is caused by mature immune cells in the donor's bone marrow (the graft) recognizing the host (recipient) as foreign and attacking it, leading to life-threatening complications. Attempts to remove those mature cells before transplant resulted in a greater chance that the donor's marrow would not engraft, although the incidence of GVHD was lowered.

The procedure that will be evaluated in this study uses a unique preparation process for the donor's marrow that selectively removes only the mature immune cells, leaving the graft Facilitating Cells, and stem cells along with the other necessary cells, to be transplanted.

It is hoped that this new process will allow for successful engraftment of the donor's marrow, and reduce the occurrence of GVHD. In the long-term, it is also possible that this new procedure will reduce the need for immunosuppressive drug therapy.

The new process, developed at the Institute for Cellular Therapeutics, may enable patients who need a bone marrow transplant, but do not have a closely matched door, to receive a transplant from any family member who is willing to donate.

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Institute for Cellular Therapeutics
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