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中间型β地中海贫血的内分泌和骨骼并发症:当前的认识与治疗

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

作者信息

Inati Adlette, Noureldine MohammadHassan A, Mansour Anthony, Abbas Hussein A

机构信息

Department of Pediatrics, Rafic Hariri University Hospital, Beirut, Lebanon ; School of Medicine, Lebanese American University, Byblos, Lebanon ; Lebanese American University and University Medical Center-Rizk Hospital, Beirut, Lebanon.

School of Medicine, Lebanese American University, Byblos, Lebanon.

出版信息

Biomed Res Int. 2015;2015:813098. doi: 10.1155/2015/813098. Epub 2015 Mar 5.

Abstract

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.

摘要

中间型地中海贫血(TI),也称为非输血依赖型地中海贫血(NTDT),是一种地中海贫血类型,患病患者生存不需要终身定期输血,但在某些临床情况下和特定时间段可能需要偶尔甚至频繁输血。NTDT包括三种不同的临床形式:β-中间型地中海贫血(β-TI)、Hb E/β-地中海贫血和α-中间型地中海贫血(Hb H病)。在过去十年中,我们对NTDT尤其是β-TI的分子特征、病理生理学和并发症的认识有了极大提高,但关于该疾病及其各种并发症的最佳治疗数据仍然缺乏。在本文中,我们将综述β-TI中一组常见的并发症,主要是内分泌和骨骼并发症。

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