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患有沃夫勒姆综合征患者的神经感觉障碍:描述性和纵向分析

Neurosensory Affectation in Patients Affected by Wolfram Syndrome: Descriptive and Longitudinal Analysis.

作者信息

Esteban-Bueno Gema, Berenguel Hernández Aída María, Fernández Fernández Nicolás, Navarro Cabrero Miguel, Coca Juan R

机构信息

Clinical Management Unit Almería Periphery-Almería Health District, Andalusian Health Service, 04120 Costacabana, Almería, Spain.

Spanish Association for the Research and Support of Wolfram Syndrome, 04120 Costacabana, Almería, Spain.

出版信息

Healthcare (Basel). 2023 Jun 29;11(13):1888. doi: 10.3390/healthcare11131888.

Abstract

Wolfram syndrome (WS) is a rare neurodegenerative disease usually of autosomal recessive origin. There is limited research about sensorineural hearing loss, despite it being a fundamental entity. It is important to broaden the study of this disease and specify a set of tests required for an adequate assessment of patients for efficient monitoring and control. The fundamental objective of this research is to understand WS from a biomedical perspective in order to help in its diagnosis, follow-up, and control. Pure tones audiometry, tympanometry, speech perception, the speech intelligibility index without aid, and testing at high frequencies were among the audiological measurements utilised since they were deemed suitable for standardised follow-up. Mixed linear models were used to examine the effects of age, time, or mean interaction in pure-tone (IPT), the average of high frequencies (HFA), auditory brainstem response (ABR), and brainstem auditory evoked potentials (BAEP). The genetic analysis allowed mutations to be classified into three phenotype-genotype groups, where the phenotype indicated the severity of the hearing loss. Patients with homozygous gene changes had a more severe neurosensory phenotype. The early discovery of sensorineural hearing loss and WS is crucial since it allows intensive follow-up and treatment of the person affected from the start.

摘要

沃夫勒姆综合征(WS)是一种罕见的神经退行性疾病,通常为常染色体隐性遗传。尽管感音神经性听力损失是该疾病的一个基本特征,但相关研究有限。拓宽对这种疾病的研究并明确一套充分评估患者以进行有效监测和控制所需的检查非常重要。本研究的基本目标是从生物医学角度了解WS,以帮助其诊断、随访和控制。纯音听力测定、鼓室图、言语感知、无助听器时的言语可懂度指数以及高频测试是所采用的听力学测量方法,因为它们被认为适用于标准化随访。混合线性模型用于检验年龄、时间或纯音(IPT)、高频平均值(HFA)、听性脑干反应(ABR)和脑干听觉诱发电位(BAEP)中的平均相互作用的影响。基因分析可将突变分为三个表型 - 基因型组,其中表型表明听力损失的严重程度。纯合基因改变的患者具有更严重的神经感觉表型。感音神经性听力损失和WS的早期发现至关重要,因为这能让受影响的人从一开始就得到密集的随访和治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f012/10340328/4302cb8a21f6/healthcare-11-01888-g001.jpg

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