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Successful unrelated cord blood transplantation for homozygous α-thalassemia

Burak Gumuscu1, Elizabeth I Thompson, Alfred C Grovas

  • 1*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.

Journal of Pediatric Hematology/Oncology
|January 23, 2013
PubMed

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.

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