β-地中海贫血:治愈仍是梦想吗?

IF 4.7 4区 医学 N/A MEDICINE, GENERAL & INTERNAL
Minerva medica Pub Date : 2023-12-01 Epub Date: 2023-08-03 DOI:10.23736/S0026-4806.23.08501-4
Mohammad H Hodroj, Nathalie Akiki, Rayan Bou-Fakhredin, Ali T Taher
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引用次数: 0

摘要

β 地中海贫血症是一种单基因疾病,其特点是血红蛋白生成减少,导致慢性贫血。影响β-地中海贫血患者临床表现的因素有多种,铁过载或红细胞生成功能低下等并发症也与该病有关。迄今为止,人们一直在采用几种保守疗法,即输血、铁螯合剂和美国食品及药物管理局批准的药物 Luspatercept,以及其他值得商榷的永久性疗法。目前正在进行其他临床试验,以便为这些患者开发更好、更安全的治疗技术。本综述将讨论β-地中海贫血症的不同治疗策略,包括新型疗法,以及正在开发的所有可能的治疗方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Beta-thalassemia: is cure still a dream?

β-thalassemia is a monogenic disorder characterized by decreased hemoglobin production, resulting in chronic anemia. There are several factors affecting the clinical presentation of patients with β-thalassemia, and several complications such as iron overload or ineffective erythropoiesis have been linked to this disease. Until nowadays, several conservative therapies namely blood transfusions, iron chelation, and the FDA-approved drug Luspatercept have been adopted alongside other debatable permanent cures. Other clinical trials are being conducted to develop better and safer management techniques for these patients. This review will discuss the different treatment strategies of β-thalassemia including novel therapies, besides all possible curative therapies that are being developed for this disease.

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来源期刊
Minerva medica
Minerva medica 医学-医学:内科
CiteScore
6.40
自引率
6.40%
发文量
358
审稿时长
>12 weeks
期刊介绍: Minerva Medica publishes scientific papers on internal medicine. Manuscripts may be submitted in the form of editorials, original articles, review articles, case reports, special articles, letters to the Editor and guidelines. The journal aims to provide its readers with papers of the highest quality and impact through a process of careful peer review and editorial work. Duties and responsibilities of all the subjects involved in the editorial process are summarized at Publication ethics.
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