Departement of Retina, Rothschild Foundation hospital, Paris, France.
Departement of Retina, Centre d'investigation clinique, Paris, France.
Ophthalmic Genet. 2022 Dec;43(6):817-823. doi: 10.1080/13816810.2022.2121841. Epub 2022 Sep 13.
Albinism is a group of genetic disorders characterized by general skin and retinal hypopigmentation. It is in most cases an autosomal recessive condition. Foveal hypoplasia (FH) is one of the main criteria for the diagnosis of albinism. The aim of this study was to analyze the macular profile of the parents of patients with albinism.
This study included a case series of 27 patients with albinism seen in Rothschild Foundation between April 2017 and February 2020. Spectral-domain optical coherence tomography (SD-OCT) and OCT angiography (OCT-A) were performed in every patient when possible and in every available parents. FH was graded according to Thomas' classification based on OCT. Next generation sequencing-based gene panel testing was performed in parents and children when a FH was detected on OCT in a parent.
Twenty-seven patients with albinism were examined. Nine parents had FH based on the OCT B-scan (33%). In parents without FH based on the SD-OCT B-scan (67%), OCT-A showed a reduced avascular zone in the deep vascular plexus in 4 parents. Six parents carried variants that could explain their phenotype, including TYR R402Q hypomorphic alleles.
This study showed the presence of FH in parents of patients with albinism, and aimed to genetically explain this phenotype.
白化病是一组以皮肤和视网膜普遍色素减退为特征的遗传疾病。它在大多数情况下是常染色体隐性疾病。黄斑发育不良(FH)是白化病诊断的主要标准之一。本研究旨在分析白化病患者父母的黄斑情况。
本研究包括 2017 年 4 月至 2020 年 2 月在 Rothschild 基金会就诊的 27 例白化病患者的病例系列。对每例患者尽可能进行频域光学相干断层扫描(SD-OCT)和 OCT 血管造影(OCT-A)检查,并对每例有条件的父母进行检查。根据基于 OCT 的 Thomas 分类法对 FH 进行分级。当在父母的 OCT 上检测到 FH 时,对父母和孩子进行基于下一代测序的基因面板检测。
检查了 27 例白化病患者。9 位父母根据 OCT B 扫描存在 FH(33%)。在根据 SD-OCT B 扫描不存在 FH 的父母(67%)中,4 位父母的 OCT-A 显示深层血管丛的无血管区减少。6 位父母携带的变异可解释其表型,包括 TYR R402Q 低功能等位基因。
本研究显示白化病患者的父母存在 FH,并旨在从遗传学上解释这种表型。