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Drugs
Published in: Drugs 15/2014

01-10-2014 | Therapy in Practice

Management of Non-Transfusion-Dependent Thalassemia: A Practical Guide

Authors: Ali T. Taher, Maria Domenica Cappellini

Published in: Drugs | Issue 15/2014

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Abstract

Despite their transfusion-independence, non-transfusion-dependent thalassemia (NTDT) patients experience a variety of serious clinical complications that require prompt and comprehensive management. Transfusion therapy may still be an important part of management of this disease, in cases of acute stress, to support growth and development in childhood, or to prevent clinical morbidities stemming from ineffective erythropoiesis or hemolytic anemia. Although splenectomy is associated with improvements in hemoglobin levels, it leads to several short- and long-term adverse events, warranting caution in application of this intervention. Fetal hemoglobin induction therapy has been evaluated in non-randomized studies, with benefits extending beyond hematologic improvements to lowering morbidity risk. Effective and safe iron chelation therapy is now available for NTDT patients in whom iron overload develops, irrespective of transfusions, due to increased intestinal absorption, ultimately leading to clinically high iron burden levels and subsequent morbidity. Optimal management of NTDT patients requires a holistic approach targeting all hallmarks of the disease to ensure favorable patient outcomes.
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Metadata
Title
Management of Non-Transfusion-Dependent Thalassemia: A Practical Guide
Authors
Ali T. Taher
Maria Domenica Cappellini
Publication date
01-10-2014
Publisher
Springer International Publishing
Published in
Drugs / Issue 15/2014
Print ISSN: 0012-6667
Electronic ISSN: 1179-1950
DOI
https://doi.org/10.1007/s40265-014-0299-0

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