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

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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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Acknowledgments

The authors thank Kaivan Khavandi MD of EVIDA MEDICAL DMCC, Dubai, UAE, for medical editorial assistance. Financial support for medical editorial assistance was provided by the Hemoglobinopathy Research Initiative, Reading, UK, who received an unrestricted grant from Novartis Pharma AG, Basel, Switzerland. The grant provider did not have any role in the design or writing of, or the decision to submit, this manuscript for publication.

Conflicts of interest

AT Taher reports receiving research funding and honoraria from Novartis. MD Cappellini reports receiving honoraria from Novartis, Genzyme, and Celgene.

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Authors and Affiliations

  1. Department of Internal Medicine, American University of Beirut Medical Center, P.O. Box 11-0236, Beirut, 11072020, Lebanon

    Ali T. Taher

  2. Department of Clinical Sciences and Community, IRCCS Ca’ Granda Foundation Maggiore Policlinico Hospital, University of Milan, Milan, Italy

    Maria Domenica Cappellini

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  1. Ali T. Taher
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  2. Maria Domenica Cappellini
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Correspondence to Ali T. Taher.

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Taher, A.T., Cappellini, M.D. Management of Non-Transfusion-Dependent Thalassemia: A Practical Guide. Drugs 74, 1719–1729 (2014). https://doi.org/10.1007/s40265-014-0299-0

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  • DOI: https://doi.org/10.1007/s40265-014-0299-0

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