输血依赖性β-地中海贫血（TDT）的治疗：来自中东的专家见解和实践综述。

IF 6.9 2区医学 Q1 HEMATOLOGY

Blood Reviews Pub Date : 2024-01-01 DOI:10.1016/j.blre.2023.101138

Amal El-Beshlawy , Hany Dewedar , Salwa Hindawi , Salam Alkindi , Azza A. Tantawy , Mohamed A. Yassin , Ali T. Taher

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引用次数: 0

摘要

β地中海贫血是世界范围内最常见的单基因疾病之一，在中东地区发病率特别高。因此，我们在疾病管理和制定解决方案以应对资源限制带来的挑战方面积累了长期经验。该地区还参与了大多数铁螯合剂的临床试验和开发计划，以及更新颖的无效红细胞生成靶向治疗。在这篇综述中，我们对输血依赖性β-地中海贫血患者的管理提供了一个实用的概述，主要是由这些经验驱动的，目的是将知识传授给面临类似挑战的其他地区的同事。

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Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East

β-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesis-targeted therapy. In this review, we provide a practical overview of management for patients with transfusion-dependent β-thalassemia, primarily driven by such experiences, with the aim of transferring knowledge to colleagues in other regions facing similar challenges.

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来源期刊

Blood Reviews 医学-血液学

CiteScore

13.80

自引率

1.40%

发文量

期刊介绍： Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.