Advances in allogeneic stem cell transplantation for hemoglobinopathies.

摘要

Hematopoietic stem cell transplantation (SCT) is currently the only potential curative therapy for thalassemia and sickle cell disease. A myeloablative conditioning regimen has been in use to eradicate the disease. Nowadays, improved preparative and conditioning methods are used including reduced intensity conditioning regimens. Such developments have allowed transplantation of more advanced hemoglobinopathy diseases. Stem cell transplant sources became more accessible including umbilical cord blood and alternate donor. However, donor human leukocyte antigen (HLA) disparity still carries a significant risk of morbidity and mortality.