Umbilical cord blood transplantation for thalassemia major

Hematopoietic cell transplantation is curative therapy for thalassemia major. Although the clinical application of hematopoietic cell transplantation has relied on marrow collected from related and unrelated donors as the primary source of donor hematopoietic cells, umbilical cord blood (UCB) is an alternative source of hematopoietic cells and represents a suitable allogeneic donor pool in the event that a marrow donor is not available. Progress in developing UCB transplantation for thalassemia is reviewed and the most likely areas of future clinical investigation are discussed.