title: Successful unrelated cord blood transplantation for homozygous α-thalassemia.
abstract: A now 10-year-old Laotian female was delivered at 30-week gestation by cesarean section because of severe hydrops. Fetal blood sampling revealed homozygous α-thalassemia. After immediate resuscitation, the infant was supported with frequent red cell transfusions. At 44 months of age, she received a 5 of 6 human leukocyte antigen-matched unrelated cord blood transplantation. She was treated with phlebotomy and chelation therapy with Deferasirox for correction of hemosiderosis and has been transfusion-independent since 41 days after transplant. She is currently 6 years after transplantation with stable, 100% donor engraftment, resolved iron overload, and normal growth and development.
authors: *National Institutes of Health/National Cancer Institute/Experimental Transplantation and Immunology Branch, Bethesda, MD †Department of Pediatric Hematology/Oncology, Children's National Medical Center, Washington, DC Departments of ‡Pediatrics, Division of Hematology/Oncology ∥Pathology/Microbiology, University of Nebraska Medical Center §Department of Pediatrics, Creighton University School of Medicine, Omaha, NE.
authors: Burak
authors: B
authors: Gumuscu
authors: Elizabeth I
authors: EI
authors: Thompson
authors: Alfred C
authors: AC
authors: Grovas
authors: Terrence L
authors: TL
authors: Zach
authors: Phyllis I
authors: PI
authors: Warkentin
authors: Peter F
authors: PF
authors: Coccia
mesh_terms: D001803:Blood Transfusion; D015913:Chelation Therapy; D002648:Child; D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D006720:Homozygote; D006801:Humans; D019190:Iron Overload / Q000209:etiology / Q000628:therapy; D018962:Phlebotomy; D016896:Treatment Outcome; D017085:alpha-Thalassemia / Q000150:complications / Q000235:genetics* / Q000628:therapy*