Stem cell transplantation for thalassemia

Abstract

Thalassemia, one of the most common genetic disorders, is considered to be a global problem. Several millions of the patients suffer from severe thalassemic diseases. Stem cell transplantation is currently the only curative therapy. Bone marrow transplantation offers a high probability of cure when performed in young children. There is a higher risk as the patient becomes older, especially the high incidence of graft rejection. Modified conditioning regimens live been developed to overcome graft rejection in patients with class III or full blown manifestations. The alternative use of stem cell from cord blood makes possible earlier transplant with better chance of cure, although the engraftment is slower compared to bone marrow transplantation. More experiences with regard to stem cell transplantation in adult patients, the use of stem cell transplantation from related donors as well as matched unrelated donors are necessary.