Murine and Math Models for the Level of Stable Mixed Chimerism to Cure β‐Thalassemia by Nonmyeloablative Bone Marrow Transplantation
Articles > Article · 2005
Authors: CARLA ROBERTS, LESLIE KEAN, DAVID ARCHER, Can BALKAN, LEWIS L. HSU
Annals of the New York Academy of Sciences, International, WoS, 2005, vol 1054, pp 423-428
Summary
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.