垂体形态改变是由TITF1/NKX2.1突变引起的良性遗传性舞蹈病的标志。

IF 1.6 4区 医学 Q3 CLINICAL NEUROLOGY Neurogenetics Pub Date : 2022-04-01 Epub Date: 2022-01-25 DOI:10.1007/s10048-021-00680-3
Steffi Thust, Liana Veneziano, Michael H Parkinson, Kailash P Bhatia, Elide Mantuano, Cristina Gonzalez-Robles, Indran Davagnanam, Paola Giunti
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引用次数: 3

摘要

良性遗传性舞蹈病(BHC)是一种罕见的遗传异质性运动障碍,在大多数情况下,常规神经影像学显示正常。迄今为止,仅在7例BHC患者中描述了垂体囊性异常和空蝶鞍的特征。我们报告了来自2个家族的4例BHC表型患者,其中3例接受了靶向垂体磁共振成像和基因检测。两个家族的四名患者均表现出典型的BHC表型。垂体核磁共振成像显示垂体鞍形态异常。对3例患者进行了基因检测,发现其中3例患者发生了导致BHC的突变,并在其中1例患者中发现了TITF1/NKX2-1基因的新型无义突变。在同一家族的两名受影响成员中存在异常垂体蝶鞍,支持了这一迹象是由于TITF1基因突变导致的BHC表型谱的明显特征的假设。有趣的是,这些异常似乎是在成人生活中发展起来的,并且是渐进的。至少26%的脑-肺-甲状腺综合征患者会出现这种情况。作为这些患者治疗的一部分,我们建议在成人生活中也进行专门的垂体成像脑MRI随访,因为异常可能在舞蹈病发作数年后发生。
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Altered pituitary morphology as a sign of benign hereditary chorea caused by TITF1/NKX2.1 mutations.

Benign hereditary chorea (BHC) is a rare genetically heterogeneous movement disorder, in which conventional neuroimaging has been reported as normal in most cases. Cystic pituitary abnormalities and features of empty sella have been described in only 7 patients with BHC to date. We present 4 patients from 2 families with a BHC phenotype, 3 of whom underwent targeted pituitary MR imaging and genetic testing. All four patients in the two families displayed a classic BHC phenotype. The targeted pituitary MR imaging demonstrated abnormal pituitary sella morphology. Genetic testing was performed in three patients, and showed mutations causing BHC in three of the patients, as well as identifying a novel nonsense mutation of the TITF1/NKX2-1 gene in one of the patients. The presence of the abnormal pituitary sella in two affected members of the same family supports the hypothesis that this sign is a distinct feature of the BHC phenotype spectrum due to mutations in the TITF1 gene. Interestingly, these abnormalities seem to develop in adult life and are progressive. They occur in at least 26% of patients affected with Brain-lung-thyroid syndrome. As a part of the management of these patients we recommend to perform follow-up MRI brain with dedicated pituitary imaging also in adult life as the abnormality can occur years after the onset of chorea.

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来源期刊
Neurogenetics
Neurogenetics 医学-临床神经学
CiteScore
3.90
自引率
0.00%
发文量
24
审稿时长
6 months
期刊介绍: Neurogenetics publishes findings that contribute to a better understanding of the genetic basis of normal and abnormal function of the nervous system. Neurogenetic disorders are the main focus of the journal. Neurogenetics therefore includes findings in humans and other organisms that help understand neurological disease mechanisms and publishes papers from many different fields such as biophysics, cell biology, human genetics, neuroanatomy, neurochemistry, neurology, neuropathology, neurosurgery and psychiatry. All papers submitted to Neurogenetics should be of sufficient immediate importance to justify urgent publication. They should present new scientific results. Data merely confirming previously published findings are not acceptable.
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