Successful unrelated cord blood transplantation for homozygous α-thalassemia.

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.
AuthorsBurak Gumuscu, Elizabeth I Thompson, Alfred C Grovas, Terrence L Zach, Phyllis I Warkentin, Peter F Coccia
JournalJournal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 35 Issue 7 Pg. 570-2 (Oct 2013) ISSN: 1536-3678 [Electronic] United States
PMID23337553 (Publication Type: Case Reports, Journal Article)
  • Blood Transfusion
  • Chelation Therapy
  • Child
  • Cord Blood Stem Cell Transplantation
  • Female
  • Homozygote
  • Humans
  • Iron Overload (etiology, therapy)
  • Phlebotomy
  • Treatment Outcome
  • alpha-Thalassemia (complications, genetics, therapy)

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