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Iron Overload and Chelation Therapy in Non-Transfusion Dependent Thalassemia
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Int. J. Mol. Sci. 2018, 19(1), 182; doi:10.3390/ijms19010182

Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management

Department of Internal Medicine, American University of Beirut Medical Center, Beirut 11-0236, Lebanon
Department of Medicine, Indiana University School of Medicine, Indianapolis, IN 46202, USA
Department of Medicine, Ca’Granda Foundation IRCCS, University of Milan, 20122 Milan, Italy
Department of Clinical Science and Community, University of Milan, 20122 Milan, Italy
These authors contributed equally to this work.
Author to whom correspondence should be addressed.
Received: 17 December 2017 / Revised: 5 January 2018 / Accepted: 6 January 2018 / Published: 8 January 2018
(This article belongs to the Special Issue Thalassemia in 2017)
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Patients with non-transfusion-dependent thalassemia (NTDT) experience many clinical complications despite their independence from frequent transfusions. Morbidities in NTDT stem from the interaction of multiple pathophysiological factors: ineffective erythropoiesis, iron overload (IOL), and hypercoagulability. Ineffective erythropoiesis and hemolysis are associated with chronic hypoxia and a hypercoagulable state. The latter are linked to a high prevalence of thromboembolic and cerebrovascular events, as well as leg ulcers and pulmonary hypertension. IOL in NTDT patients is a cumulative process that can lead to several iron-related morbidities in the liver (liver fibrosis), kidneys, endocrine glands (endocrinopathies), and vascular system (vascular disease). This review sheds light on the pathophysiology underlying morbidities associated with NTDT and summarizes the mainstays of treatment and some of the possible future therapeutic interventions. View Full-Text
Keywords: non-transfusion dependent thalassemia; morbidity; management; ineffective erythropoiesis; iron overload; iron chelation therapy non-transfusion dependent thalassemia; morbidity; management; ineffective erythropoiesis; iron overload; iron chelation therapy

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Sleiman, J.; Tarhini, A.; Bou-Fakhredin, R.; Saliba, A.N.; Cappellini, M.D.; Taher, A.T. Non-Transfusion-Dependent Thalassemia: An Update on Complications and Management. Int. J. Mol. Sci. 2018, 19, 182.

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