Successful unrelated cord blood transplantation for homozygous α-thalassemia

J Pediatr Hematol Oncol. 2013 Oct;35(7):570-2. doi: 10.1097/MPH.0b013e31827e7f6a.

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.

Publication types

  • Case Reports

MeSH terms

  • Blood Transfusion
  • Chelation Therapy
  • Child
  • Cord Blood Stem Cell Transplantation*
  • Female
  • Homozygote*
  • Humans
  • Iron Overload / etiology
  • Iron Overload / therapy
  • Phlebotomy
  • Treatment Outcome
  • alpha-Thalassemia / complications
  • alpha-Thalassemia / genetics*
  • alpha-Thalassemia / therapy*