Treatment of beta-thalassemia

Abstract

The striking improvement in the life expectancy of patients with homozygous beta-thalassemia observed over the past three decades is mainly due to the institution of adequate transfusion regimens and effective iron chelation therapy with nightly subcutaneous desferrioxamine. The prognosis appears particularly favorable for children with thalassemia born since these methods have become widely available. The first successful "cure" of beta-thalassemia was achieved in 1981 after bone marrow transplantation. Recent advances in transfusion techniques, pharmacology, molecular genetics, transplant immunology, and clinical practice today offer considerable promise in further advancing our knowledge and treatment of this disease.