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Murine and math models for the level of stable mixed chimerism to cure ß-thalassemia by nonmyeloablative bone marrow transplantation
(New York Academy of Sciences, 2005) Roberts C.; Kean L.; Archer D.; Balkan C.; Hsu L.L.
Stable mixed chimeric stem cell transplantation in hemoglobinopathies exploits shorter erythroid survival in hemolytic anemias, providing normal donor red blood cells with a competitive survival advantage. This study examined the level of stable mixed chimerism necessary for complete hematological cure of the thalassemic phenotype, using a nonmyeloablative busulfan chemotherapeutic preparation. Thalassemic mice transplanted from congenic wild-type donors developed partial mixed chimerism. Hematologic cure required >80% donor red blood cells and only >13% donor white blood cells. Murine and human transplant results were compared with a math model for survival advantage of donor peripheral blood cells produced by steady-state chimeric marrow. © 2005 New York Academy of Sciences.