Gumuscu Burak, Thompson Elizabeth I, Grovas Alfred C, Zach Terrence L, Warkentin Phyllis I, Coccia Peter F
*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.
J Pediatr Hematol Oncol. 2013 Oct;35(7):570-2. doi: 10.1097/MPH.0b013e31827e7f6a.
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.