Goal 2: Reduce Human Disease

Transplantation across HLA barriers in aplastic anemia

Allogeneic stem cell transplantation is curative in aplastic anemia with much less intrinsic toxicity than transplantation in hematologic malignancies. The recent BMT-CTN trial demonstrated 97% survival at one year with little subsequent decline. However patients without matched related or unrelated donors have graft-rejection rates of up to 50%. Preliminary data from the Netherlands suggests that anti-thymocyte globulin dosing based on recipient size and lymphocyte count can overcome this immunologic barrier. This approach needs to be studied in a large multicenter trial given both the rarity of refractory severe aplastic anemia and the relative minority of patients without donors. This disproportionately affects minority patients.

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Idea No. 452