Homozygous α-Thalassemia Treated with Intrauterine Transfusions and Unrelated Donor Hematopoietic Cell Transplantation

Recently, intrauterine transfusions and hematopoietic cell transplantation (HCT) have changed homozygous alpha-thalassemia from a frequently fatal disease to a potentially survivable condition. We present a patient with Hemoglobin Bart's disease who was cured after failing to engraft with 1 unrelated HCT, but engrafting after a second unrelated donor HCT. (J Pediatr 2009;154:766-8)