title: Murine and math models for the level of stable mixed chimerism to cure beta-thalassemia by nonmyeloablative bone marrow transplantation.
abstract: 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.
authors.affiliation: Palmetto Health Systems, Columbia, South Carolina 29203, USA.
authors.forename: Carla
authors.initials: C
authors.lastname: Roberts
authors.forename: Leslie
authors.initials: L
authors.lastname: Kean
authors.forename: David
authors.initials: D
authors.lastname: Archer
authors.forename: Can
authors.initials: C
authors.lastname: Balkan
authors.forename: Lewis L
authors.initials: LL
authors.lastname: Hsu
mesh_terms: D000818:Animals; D020296:Animals, Congenic; D016026:Bone Marrow Transplantation; D002066:Busulfan / Q000494:pharmacology; D004906:Erythrocyte Count; D006085:Graft Survival; D006410:Hematopoiesis; D006801:Humans; D007958:Leukocyte Count; D051379:Mice; D008810:Mice, Inbred C57BL; D008954:Models, Biological; D012074:Remission Induction; D016019:Survival Analysis; D018183:Transplantation Chimera / Q000097:blood*; D019172:Transplantation Conditioning / Q000379:methods*; D017086:beta-Thalassemia / Q000601:surgery*
chemical_list: D002066:Busulfan