非综合征性bbs1相关视网膜变性患者的临床-视网膜电图不匹配

IF 0.6 Q3 MEDICINE, GENERAL & INTERNAL
Mariana Ferreira Francisco, Miguel Raimundo, Ana Luísa Carvalho, João Pedro Marques
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引用次数: 0

摘要

一位30多岁的男性患者,M390R Bardet-Biedl综合征-1 (BBS1)变异纯合子,由于视力下降和畏光,在成年早期开始随访。眼底检查显示双侧颞叶楔状视盘苍白和萎缩性黄斑病变,转化为眼底自体荧光中枢性低荧光。尽管临床和多模态成像特征提示锥显性变性,但视网膜电图(ERG)显示暗位和光位反应减少,暗位损伤更大。尽管进行了全面的系统评估和长期随访,但未发现BBS的其他特征。据我们所知,这是第一例记录在案的非综合征性bbs1相关疾病,在ERG上表现为杆状锥体营养不良表型,临床表现(症状和影像学特征)为锥体显性疾病。临床/影像学和ERG结果之间的差异反映了bbs1相关疾病的复杂性,并突出了基因检测和综合表型在遗传性视网膜疾病中的关键作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical-electroretinography mismatch in a patient with non-syndromic BBS1-associated retinal degeneration.

A male patient in his late thirties, homozygous for the M390R Bardet-Biedl Syndrome-1 (BBS1) variant, began follow-up in early adulthood, due to decreased visual acuity and photophobia. Fundus examination showed bilateral temporal wedge-shaped pallor of the optic disc and atrophic maculopathy, translating into central hypoautofluorescence on fundus autofluorescence. Even though clinical and multimodal imaging features suggested cone-dominant degeneration, electroretinography (ERG) showed reduced scotopic and photopic responses, with greater scotopic impairment. Despite a thorough systemic evaluation and long-term follow-up, no other features of BBS were found. To our knowledge, this is the first documented case of non-syndromic BBS1-associated disease with a rod-cone dystrophy phenotype on ERG and a clinical presentation (symptoms and imaging features) of cone-dominant disease. The discrepancy between clinical/imaging and ERG findings reflects the complexity of BBS1-related disease and highlights the crucial role of genetic testing and comprehensive phenotyping in inherited retinal disorders.

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来源期刊
BMJ Case Reports
BMJ Case Reports Medicine-Medicine (all)
CiteScore
1.40
自引率
0.00%
发文量
1588
期刊介绍: BMJ Case Reports is an important educational resource offering a high volume of cases in all disciplines so that healthcare professionals, researchers and others can easily find clinically important information on common and rare conditions. All articles are peer reviewed and copy edited before publication. BMJ Case Reports is not an edition or supplement of the BMJ.
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