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在临床环境中使用全基因组测序和与非免疫性胎儿水肿相关的 281 个基因的计算机综合面板进行诊断的效果。

Diagnostic yield using whole-genome sequencing and in-silico panel of 281 genes associated with non-immune hydrops fetalis in clinical setting.

机构信息

Department of Molecular Medicine and Surgery, Karolinska Institutet, Stockholm, Sweden.

Department of Clinical Genetics, Karolinska University Hospital, Stockholm, Sweden.

出版信息

Ultrasound Obstet Gynecol. 2022 Oct;60(4):487-493. doi: 10.1002/uog.24911. Epub 2022 Aug 30.

Abstract

OBJECTIVE

To investigate the diagnostic yield of clinical whole-genome sequencing (WGS) in prenatally diagnosed non-immune hydrops fetalis (NIHF).

METHODS

This was a retrospective study of 23 fetuses with prenatally diagnosed NIHF, negative for trisomies and copy-number variants, referred for analysis by WGS with an in-silico panel of 281 genes associated with hydrops fetalis. Due to identification of a high proportion of causative variants in the HRAS gene in the main cohort, Sanger sequencing of HRAS was performed in a replication cohort, consisting of 24 additional fetuses with NIHF that were negative for trisomies and copy-number variants and had not undergone WGS.

RESULTS

Of the 23 fetuses in the main cohort, a molecular diagnosis was achieved in 12 (52.2%). Pathogenic or likely pathogenic variants were identified in seven genes: HRAS (n = 5), RIT1 (n = 2), FOXP3 (n = 1), GLB1 (n = 1), MAP2K1 (n = 1), PTPN11 (n = 1) and RASA1 (n = 1). The inheritance pattern of the 12 causative variants was autosomal dominant in 10 cases (HRAS, MAP2K1, PTPN11, RASA1, RIT1), autosomal recessive in one (GLB1) and X-linked recessive in one (FOXP3). Of the 24 fetuses in the replication cohort, a pathogenic variant in HRAS was identified in one, resulting in an overall frequency of causative HRAS variants of 12.8% (6/47) in our two cohorts.

CONCLUSIONS

We demonstrate a diagnostic yield of 52% with clinical WGS in NIHF using an in-silico panel of 281 genes. However, the high diagnostic yield may be attributed to the small sample size and possible over-representation of severe phenotypes in the included fetuses. Bearing in mind that chromosomal abnormalities were excluded in our cohorts, a detection rate of up to 75% is possible in prenatally diagnosed NIHF when WGS analysis includes calling of chromosomal aberrations. © 2022 The Authors. Ultrasound in Obstetrics & Gynecology published by John Wiley & Sons Ltd on behalf of International Society of Ultrasound in Obstetrics and Gynecology.

摘要

目的

探讨临床全基因组测序(WGS)在产前诊断非免疫性胎儿水肿(NIHF)中的诊断效能。

方法

这是一项回顾性研究,纳入 23 例产前诊断为 NIHF、染色体非整倍体和拷贝数变异均为阴性的胎儿,采用 WGS 进行分析,使用与胎儿水肿相关的 281 个基因的计算机panel。由于在主要队列中 HRAS 基因中发现了很高比例的致病变异,因此在一个由 24 例 NIHF 胎儿组成的复制队列中进行了 HRAS 的 Sanger 测序,这些胎儿的染色体非整倍体和拷贝数变异均为阴性,且未行 WGS。

结果

在主要队列的 23 例胎儿中,12 例(52.2%)获得了分子诊断。在 7 个基因中发现了致病性或可能致病性变异:HRAS(n=5)、RIT1(n=2)、FOXP3(n=1)、GLB1(n=1)、MAP2K1(n=1)、PTPN11(n=1)和 RASA1(n=1)。12 个致病变异的遗传模式为 10 例(HRAS、MAP2K1、PTPN11、RASA1、RIT1)为常染色体显性遗传,1 例(GLB1)为常染色体隐性遗传,1 例(FOXP3)为 X 连锁隐性遗传。在复制队列的 24 例胎儿中,有 1 例 HRAS 存在致病性变异,导致我们两个队列中 HRAS 致病变异的总发生率为 12.8%(6/47)。

结论

我们使用 281 个基因的计算机panel 进行临床 WGS,在 NIHF 中显示出 52%的诊断效能。然而,高诊断率可能归因于样本量小,且纳入的胎儿可能存在严重表型的过度代表。鉴于我们的队列中排除了染色体异常,当 WGS 分析包括染色体异常的检测时,产前诊断的 NIHF 的检出率可达 75%。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a9a3/9804469/1091d6e2f3e8/UOG-60-487-g001.jpg

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