Endocrine and bone complications in β-thalassemia intermedia: current understanding and treatment.

Endocrine and bone complications in β-thalassemia intermedia: current understanding and treatment.
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DOI:
10.1155/2015/813098
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发表时间:
2015
影响因子:
--
通讯作者:
Abbas HA
Abbas HA
中科院分区:
生物学3区
文献类型:
--
作者:
Inati A;Noureldine MA;Mansour A;Abbas HA

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中间型地中海贫血(TI),也称为非输血依赖性地中海贫血(NTDT),是一种地中海贫血,其中受影响的患者不需要终身定期输血才能生存,但在某些临床环境中可能需要偶尔甚至频繁输血并持续一定的时间。NTDT包括三种不同的临床形式:中间型β-地中海贫血(β-TI)、Hb E/β-地中海贫血和中间型α-地中海贫血(Hb H病)。在过去的十年中,我们对NTDT(特别是β-TI)的分子特征、病理生理学和并发症的了解大大增加,但仍然缺乏关于疾病及其各种并发症的最佳治疗的数据。本文就β-TI常见的并发症,主要是内分泌和骨骼并发症作一综述。
Thalassemia intermedia (TI), also known as nontransfusion dependent thalassemia (NTDT), is a type of thalassemia where affected patients do not require lifelong regular transfusions for survival but may require occasional or even frequent transfusions in certain clinical settings and for defined periods of time. NTDT encompasses three distinct clinical forms: β-thalassemia intermedia (β-TI), Hb E/β-thalassemia, and α-thalassemia intermedia (Hb H disease). Over the past decade, our understanding of the molecular features, pathophysiology, and complications of NTDT particularly β-TI has increased tremendously but data on optimal treatment of disease and its various complications are still lacking. In this paper, we shall review a group of commonly encountered complications in β-TI, mainly endocrine and bone complications.
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