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Chelation therapy for thalassemia and other iron overload states

Authors
Stanley L Schrier, MD
Bruce R Bacon, MD
Section Editor
William C Mentzer, MD
Deputy Editor
Jennifer S Tirnauer, MD

INTRODUCTION

Phlebotomy cannot be used to remove excess iron in transfusion-dependent patients (eg, beta thalassemia major, severe beta thalassemia intermedia, sickle cell anemia, myelodysplasia, aplastic anemia) with iron overload or in the rare patient with hemochromatosis and an unstable hemodynamic status (eg, severe cardiac involvement) [1]. Thus, if treatment for iron overload is deemed necessary in such patients, one must use an iron chelating agent.

The use of chelating agents for the treatment of iron overload states in children and adults will be discussed here, with an emphasis on iron chelation therapy in thalassemia [2,3]. Overviews of the causes and diagnosis of iron overload are presented separately. (See "Iron overload syndromes other than hereditary hemochromatosis" and "Approach to the patient with suspected iron overload" and "Methods to determine hepatic iron content".)

Treatment of iron overload using phlebotomy, which is available for non-anemic patients (eg, hereditary hemochromatosis), is discussed separately. Such treatment is also available for formerly transfusion-dependent individuals (eg, thalassemia, sickle cell disease, myelodysplasia, aplastic anemia) who have been cured of their underlying disease following successful hematopoietic cell transplantation (HCT). (See "Management of patients with hereditary hemochromatosis", section on 'Phlebotomy' and "Long-term management of the thalassemic patient after hematopoietic cell transplantation", section on 'Phlebotomy'.)

Treatment of acute iron poisoning, a medical emergency, which includes supportive care, gastrointestinal decontamination, and intensive iron chelation with intravenous deferoxamine, is presented separately. (See "Acute iron poisoning".)

CLINICAL OVERVIEW

Decisions to begin treatment with iron chelation require clinical judgment since both the prognosis of the underlying disease and the potential benefit of chelation must be taken into account [4]. The level of evidence to guide the clinician in initiating chelation therapy is quite variable as shown by the following examples in three different disorders associated with iron overload:

                                              

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