New trends in the treatment of beta-thalassemia

Thalassemia is the world's most common hereditary disease, and is a paradig m of monogenic genetic diseases. Because of increased population mobility, the disease is found today throughout the world, even in places far from th e tropical areas in which it arose. Therapy of thalassemia has in the past been confined to transfusion and chelation. Recently, novel modes of therap y have been developed for thalassemia, based on the pathophysiology and mol ecular pathology of the disease, both of which have been extensively studie d. This review will discuss the therapeutic modalities currently in use for the supportive treatment of thalassemia, both those that are standard ther apy and those that are in clinical trials. We will include transfusion, che lation (intravenous and oral), antioxidants and various inducers of fetal h emoglobin (hydroxyurea, erythropoietin, butyrates, hemin). Most of the newe r therapies are suitable primarily for thalassemia intermedia patients. In addition, the treatment modalities currently in use for the curative treatm ent of thalassemia major will be discussed, including bone marrow transplan tation in its various forms. Experimental therapeutic methods, such as intr auterine bone marrow transplantation and gene therapy. are included. Physic ians caring for thalassemia patients have an increasing variety of treatmen t options available. Future clinical studies will determine the place of ne wer agents and modalities in improving the qualify of life as well as the l ife expectancy of thalassemia patients. (C) 2000 Elsevier Science Ireland L td. All rights reserved.