δβ-地中海贫血和 α-三倍体:表型不一致时是否值得进行基因重测?

IF 1.2 4区 医学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY
Hemoglobin Pub Date : 2024-09-01 Epub Date: 2024-10-14 DOI:10.1080/03630269.2024.2414109
Cristina Giubbilei, Simona D'Angelo, Ilaria Fotzi, Massimo Mogni, Paola Guglielmelli, Alessandro Maria Vannucchi, Valentina Carrai
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引用次数: 0

摘要

地中海贫血是一组异质性的血红蛋白病;由于基因匹配不同,中间型地中海贫血的表型可能千差万别。在 NGS 时代之前,诊断往往与表型不匹配,隐藏了一些如今可以完全解释临床表现的基因发现。在本报告中,我们强调了在表型不匹配地中海贫血的情况下,重新评估基因检测以获得正确诊断的重要性。从疑似δ/β地中海贫血杂合子开始,重新评估发现了α基因三复制杂合子合并δ和β杂合子,这一新发现完全符合患者的临床表现。该病例提供了一个机会,强调对总球蛋白基因进行扩展研究对于正确诊断地中海贫血症至关重要,尤其是在临床发病表型较难分辨且在首次临床检查时存在疑问的情况下。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
δβ-Thalassemia and α-Triplication: Is Genetic Retesting Worthwhile in Case of Non-Coherent Phenotype?

Thalassemia is a heterogenous group of hemoglobinopathies; intermediate thalassemia's phenotype can be very variegated due to different genetic matching. Before NGS-era, diagnosis often mismatched with phenotypes, hiding some genetic findings that nowadays could completely explain clinical presentation. In this report, we emphasize the importance of reevaluating genetic testing to achieve a correct diagnosis in case of phenotype mismatch thalassemia. Starting from a suspect of δ/β thalassemia heterozygosity, reevaluating revealed heterozygosity for α-gene triplication combined to δ and β heterozygosity, a new finding that completely suited patient's clinical manifestation. This case provided the opportunity to underline that an extended study on total globin genes is essential for correct diagnosis of thalassemia, especially when clinical onset phenotypes are more divisive and questionable at a first clinical work-up.

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来源期刊
Hemoglobin
Hemoglobin 医学-生化与分子生物学
CiteScore
1.70
自引率
10.00%
发文量
59
审稿时长
3 months
期刊介绍: Hemoglobin is a journal in the English language for the communication of research and information concerning hemoglobin in humans and other species. Hemoglobin publishes articles, reviews, points of view The journal covers topics such as: structure, function, genetics and evolution of hemoglobins biochemical and biophysical properties of hemoglobin molecules characterization of hemoglobin disorders (variants and thalassemias), consequences and treatment of hemoglobin disorders epidemiology and prevention of hemoglobin disorders (neo-natal and adult screening) modulating factors methodology used for diagnosis of hemoglobin disorders
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