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Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series
Thalassemia syndromes are characterized by the inability to produce normal hemoglobin. Ineffective erythropoiesis and red cell transfusions are sources of excess iron that the human organism is unable to remove. Iron that is not saturated by transferrin is a toxic agent that, in transfusion-dependen...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7699680/ https://www.ncbi.nlm.nih.gov/pubmed/33233561 http://dx.doi.org/10.3390/ijms21228771 |
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author | Pinto, Valeria Maria Forni, Gian Luca |
author_facet | Pinto, Valeria Maria Forni, Gian Luca |
author_sort | Pinto, Valeria Maria |
collection | PubMed |
description | Thalassemia syndromes are characterized by the inability to produce normal hemoglobin. Ineffective erythropoiesis and red cell transfusions are sources of excess iron that the human organism is unable to remove. Iron that is not saturated by transferrin is a toxic agent that, in transfusion-dependent patients, leads to death from iron-induced cardiomyopathy in the second decade of life. The availability of effective iron chelators, advances in the understanding of the mechanism of iron toxicity and overloading, and the availability of noninvasive methods to monitor iron loading and unloading in the liver, heart, and pancreas have all significantly increased the survival of patients with thalassemia. Prolonged exposure to iron toxicity is involved in the development of endocrinopathy, osteoporosis, cirrhosis, renal failure, and malignant transformation. Now that survival has been dramatically improved, the challenge of iron chelation therapy is to prevent complications. The time has come to consider that the primary goal of chelation therapy is to avoid 24-h exposure to toxic iron and maintain body iron levels within the normal range, avoiding possible chelation-related damage. It is very important to minimize irreversible organ damage to prevent malignant transformation before complications set in and make patients ineligible for current and future curative therapies. In this clinical case-based review, we highlight particular aspects of the management of iron overload in patients with beta-thalassemia syndromes, focusing on our own experience in treating such patients. We review the pathophysiology of iron overload and the different ways to assess, quantify, and monitor it. We also discuss chelation strategies that can be used with currently available chelators, balancing the need to keep non-transferrin-bound iron levels to a minimum (zero) 24 h a day, 7 days a week and the risk of over-chelation. |
format | Online Article Text |
id | pubmed-7699680 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-76996802020-11-29 Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series Pinto, Valeria Maria Forni, Gian Luca Int J Mol Sci Review Thalassemia syndromes are characterized by the inability to produce normal hemoglobin. Ineffective erythropoiesis and red cell transfusions are sources of excess iron that the human organism is unable to remove. Iron that is not saturated by transferrin is a toxic agent that, in transfusion-dependent patients, leads to death from iron-induced cardiomyopathy in the second decade of life. The availability of effective iron chelators, advances in the understanding of the mechanism of iron toxicity and overloading, and the availability of noninvasive methods to monitor iron loading and unloading in the liver, heart, and pancreas have all significantly increased the survival of patients with thalassemia. Prolonged exposure to iron toxicity is involved in the development of endocrinopathy, osteoporosis, cirrhosis, renal failure, and malignant transformation. Now that survival has been dramatically improved, the challenge of iron chelation therapy is to prevent complications. The time has come to consider that the primary goal of chelation therapy is to avoid 24-h exposure to toxic iron and maintain body iron levels within the normal range, avoiding possible chelation-related damage. It is very important to minimize irreversible organ damage to prevent malignant transformation before complications set in and make patients ineligible for current and future curative therapies. In this clinical case-based review, we highlight particular aspects of the management of iron overload in patients with beta-thalassemia syndromes, focusing on our own experience in treating such patients. We review the pathophysiology of iron overload and the different ways to assess, quantify, and monitor it. We also discuss chelation strategies that can be used with currently available chelators, balancing the need to keep non-transferrin-bound iron levels to a minimum (zero) 24 h a day, 7 days a week and the risk of over-chelation. MDPI 2020-11-20 /pmc/articles/PMC7699680/ /pubmed/33233561 http://dx.doi.org/10.3390/ijms21228771 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Pinto, Valeria Maria Forni, Gian Luca Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title | Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title_full | Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title_fullStr | Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title_full_unstemmed | Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title_short | Management of Iron Overload in Beta-Thalassemia Patients: Clinical Practice Update Based on Case Series |
title_sort | management of iron overload in beta-thalassemia patients: clinical practice update based on case series |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7699680/ https://www.ncbi.nlm.nih.gov/pubmed/33233561 http://dx.doi.org/10.3390/ijms21228771 |
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