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一种罕见的玻璃体视网膜退行性疾病:一名儿科患者的戈德曼-法夫尔综合征合并脉络膜新生血管形成

A Rare Vitreoretinal Degenerative Disorder: Goldmann-Favre Syndrome Complicated with Choroidal Neovascularization in a Pediatric Patient.

作者信息

Szala Klaudia, Wójcik-Niklewska Bogumiła

机构信息

Students' Scientific Society, Department of Ophthalmology, Faculty of Medical Sciences in Katowice, Medical University of Silesia in Katowice, 40-055 Katowice, Poland.

Department of Pediatric Ophtalmology, Faculty of Medical Sciences in Katowice, Medical University of Silesia in Katowice, 40-055 Katowice, Poland.

出版信息

Diagnostics (Basel). 2025 Mar 5;15(5):622. doi: 10.3390/diagnostics15050622.

Abstract

Goldmann-Favre syndrome (GFS) is a rare vitreoretinal degenerative disorder caused by mutations in the NR2E3 gene located on the short arm of chromosome 15. This condition, inherited in an autosomal recessive manner, was first described by Favre in two siblings, with Ricci later confirming its hereditary pattern. In GFS, rod photoreceptors are essentially replaced by S-cone photoreceptors. Enhanced S-Cone Syndrome (ESCS) and Goldmann-Favre syndrome are two distinct entities within the spectrum of retinal degenerative diseases, both caused by mutations in the NR2E3 gene. Despite sharing a common genetic basis, these conditions exhibit significantly different clinical phenotypes. ESCS is characterized by an excessive number of S-cones (blue-sensitive cones) with degeneration of rods and L-/M-cones, leading to increased sensitivity to blue light and early-onset night blindness. In contrast, GFS is considered a more severe form of ESCS, involving additional features such as retinal schisis, vitreous degeneration, and more pronounced visual impairment. GFS typically manifests in the first decade of life as night blindness (nyctalopia) and progressive visual acuity impairment. The clinical features include degenerative vitreous changes such as liquefaction, strands, and bands, along with macular and peripheral retinoschisis, posterior subcapsular cataract, atypical pigmentary dystrophy, and markedly abnormal or nondetectable electroretinograms (ERGs). Although peripheral retinoschisis is more common in GFS, central retinoschisis may also occur. Despite the consistent genetic basis, the phenotype of GFS can vary significantly among individuals. The differential diagnosis should consider diseases within the retinal degenerative spectrum, including retinitis pigmentosa, congenital retinoschisis, and secondary pigmentary retinopathy.

摘要

戈德曼-法夫尔综合征(GFS)是一种罕见的玻璃体视网膜退行性疾病,由位于15号染色体短臂上的NR2E3基因突变引起。这种疾病以常染色体隐性方式遗传,最初由法夫尔在两名兄弟姐妹中描述,后来里奇证实了其遗传模式。在GFS中,视杆光感受器基本上被S-视锥光感受器所取代。增强型S-视锥综合征(ESCS)和戈德曼-法夫尔综合征是视网膜退行性疾病谱中的两种不同病症,均由NR2E3基因突变引起。尽管有共同的遗传基础,但这些病症表现出明显不同的临床表型。ESCS的特征是S-视锥细胞(蓝光敏感视锥细胞)数量过多,同时视杆细胞和L/M-视锥细胞退化,导致对蓝光的敏感性增加和早发性夜盲。相比之下,GFS被认为是ESCS的一种更严重形式,还涉及其他特征,如视网膜劈裂、玻璃体变性和更明显的视力损害。GFS通常在生命的第一个十年表现为夜盲和进行性视力损害。临床特征包括玻璃体的退行性变化,如液化、条索和带状物,以及黄斑和周边视网膜劈裂、后囊下白内障、非典型色素性营养不良,以及明显异常或无法检测到的视网膜电图(ERG)。尽管周边视网膜劈裂在GFS中更常见,但中央视网膜劈裂也可能发生。尽管有一致的遗传基础,但GFS的表型在个体之间可能有很大差异。鉴别诊断应考虑视网膜退行性疾病谱中的疾病,包括色素性视网膜炎、先天性视网膜劈裂和继发性色素性视网膜病变。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1499/11899382/754479d4ec05/diagnostics-15-00622-g001.jpg

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