Recent advances in the management of thalassemia

Although the current treatment of thalassemia with regular transfusions and assiduous chelation leads to a good quality of life and long survival, it is cumbersome and expensive. Various treatments have recently been explored. Bone marrow transplantation can cure thalassemia, but there was severe mortality in initial trials. It is safely successful only in patients in good clinical condition and with a compatible donor. Certain drugs, including azacytidine, butyrate, hydroxyurea, and erythropoietin may increase the production of fetal hemoglobin; their practical value is being explored. New potential oral iron chelators are under investigation. L1, the best evaluated, appears effective, but its potential toxicity remains undefined.