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铁过载:β-地中海贫血的阿喀琉斯之踵。

Iron overload: The achilles heel of β-thalassemia.

机构信息

Adults Thalassemia Unit, 2nd Department of Internal Medicine, Aristotle University, Hippokration General Hospital, Thessaloniki, Greece.

Adults Thalassemia Unit, 2nd Department of Internal Medicine, Aristotle University, Hippokration General Hospital, Thessaloniki, Greece.

出版信息

Transfus Clin Biol. 2024 Aug;31(3):167-173. doi: 10.1016/j.tracli.2024.06.001. Epub 2024 Jun 6.

Abstract

Systematic transfusions coupled with iron chelation therapy have substantially improved the life expectancy of thalassemia patients in developed nations. As the human organism does not have a protective mechanism to remove excess iron, iron overload is a significant concern in thalassemia, leading to organ damage, especially in the heart and liver. Thus, iron chelation therapy is crucial to prevent or reverse organ iron overload. There are three widely used iron chelators, either as monotherapy or in combination. The choice of iron chelator depends on several factors, including local guidelines, drug availability, and the individual clinical scenario. Despite treatment advancements, challenges persist, especially in resource-limited settings, highlighting the need for improved global healthcare access. This review discusses clinical management, current treatments, and future directions for thalassemia, focusing on iron overload and its complications. Furthermore, it underscores the progress in transforming thalassemia into a manageable chronic condition and the potential of novel therapies to further enhance patient outcomes.

摘要

在发达国家,系统性输血联合铁螯合疗法显著提高了地中海贫血患者的预期寿命。由于人体没有去除多余铁的保护机制,铁过载是地中海贫血的一个重要关注点,可导致器官损伤,特别是心脏和肝脏。因此,铁螯合疗法对于预防或逆转器官铁过载至关重要。目前有三种广泛使用的铁螯合剂,可单独使用或联合使用。铁螯合剂的选择取决于多个因素,包括当地指南、药物供应情况和个体临床情况。尽管治疗取得了进展,但仍存在挑战,特别是在资源有限的环境中,这突显了需要改善全球医疗保健的可及性。本文讨论了地中海贫血的临床管理、当前治疗方法和未来方向,重点关注铁过载及其并发症。此外,本文强调了将地中海贫血转变为可管理的慢性疾病方面的进展,以及新型疗法进一步改善患者预后的潜力。

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