How I treat transfusional iron overload.
Patients with β-thalassemia major (TM) and other refractory anemias requiring regular blood transfusions accumulate iron that damages the liver, endocrine system, and most importantly the heart. The prognosis in TM has improved remarkably over the past 10 years. This improvement has resulted from the development of magnetic resonance imaging (MRI) techniques, especially T2*, to accurately measure cardiac and liver iron, and from the availability of 3 iron-chelating drugs. In this article we describe the use of MRI to determine which adult and pediatric patients need to begin iron chelation therapy and to monitor their progress. We summarize the properties of each of the 3 drugs, deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), including their efficacy, patient acceptability, and side effects. We describe when to initiate or intensify therapy, switch to another drug, or use combined therapy. We also discuss the management of refractory anemias other than TM that may require multiple blood transfusions, including sickle cell anemia and myelodysplasia. The development of a potential fourth chelator FBS 0701 and the combined use of oral chelators may further improve the quality of life and survival in patients with TM and other transfusion-dependent patients.
Department of Haematology, University College London and Royal Free Hospital, London, United Kingdom. vhoffbrand@googlemail.com
A Victor
AV
Hoffbrand
Ali
A
Taher
Maria Domenica
MD
Cappellini
D001565:Benzoates / Q000627:therapeutic use; D000077588:Deferasirox; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use; D006402:Hematologic Diseases / Q000628:therapy; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D011728:Pyridones / Q000627:therapeutic use; D065227:Transfusion Reaction; D014230:Triazoles / Q000627:therapeutic use
D001565:Benzoates; D007502:Iron Chelating Agents; D011728:Pyridones; D014230:Triazoles; D000077543:Deferiprone; D003676:Deferoxamine; D000077588:Deferasirox