Homozygous alpha-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.
Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Joanna S
JS
Yi
Christopher L
CL
Moertel
K Scott
KS
Baker
D001805:Blood Transfusion, Intrauterine; D046528:Chimerism; D002658:Developmental Disabilities / Q000209:etiology; D017707:Erythrocyte Transfusion; D005260:Female; D005315:Fetal Diseases / Q000097:blood / Q000235:genetics / Q000628:therapy*; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006455:Hemoglobins, Abnormal; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D011247:Pregnancy; D011296:Prenatal Diagnosis; D014019:Tissue Donors; D017085:alpha-Thalassemia / Q000235:genetics / Q000628:therapy*
D006455:Hemoglobins, Abnormal; C030448:hemoglobin Bart's