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隐性PIEZO1病导致的非免疫性胎儿水肿的围产期表现:一项具有挑战性的胎儿诊断。

Perinatal presentations of non-immune hydrops fetalis due to recessive PIEZO1 disease: A challenging fetal diagnosis.

作者信息

Ghesh Leïla, Désir Julie, Haye Damien, Le Tanno Pauline, Devillard Françoise, Cogné Benjamin, Marangoni Martina, Tecco Laura, Heron Delphine, Le Vaillant Claudine, Joubert Madeleine, Beneteau Claire

机构信息

CHU de Nantes, UF 9321 de Fœtopathologie et Génétique, Nantes, France.

Service de Génétique Médicale, Hôpital Erasme - Cliniques Universitaires de Bruxelles, Brussels, Belgium.

出版信息

Clin Genet. 2023 May;103(5):560-565. doi: 10.1111/cge.14274. Epub 2022 Dec 12.

Abstract

Hydrops fetalis is a rare disorder associated with significant perinatal complications and a high perinatal mortality of at least 50%. Nonimmune hydrops fetalis (NIHF) is more frequent and results from a wide variety of etiologies. One cause of NIHF is lymphatic malformation 6 (LMPHM6) due to biallelic loss-of-function (LoF) variants in PIEZO1. Most individuals are diagnosed postnatally and only few clinical data are available on fetal presentations. We report six novel biallelic predicted LoF variants in PIEZO1 identified by exome sequencing in six fetuses and one deceased neonate from four unrelated families affected with LMPHM6. During the pregnancy, most cases are revealed by isolated NIHF at second trimester of gestation. At post-mortem examination ascites, pleural effusions and telengectasies can guide the etiological diagnosis. We aim to further describe the perinatal presentation of this condition which could be underdiagnosed.

摘要

胎儿水肿是一种罕见的疾病,伴有严重的围产期并发症,围产期死亡率至少为50%,较高。非免疫性胎儿水肿(NIHF)更为常见,由多种病因引起。NIHF的一个病因是由于PIEZO1基因双等位基因功能丧失(LoF)变异导致的淋巴管畸形6(LMPHM6)。大多数个体在出生后被诊断,关于胎儿表现的临床数据很少。我们报告了通过外显子组测序在来自四个无关家庭的六名胎儿和一名死亡新生儿中鉴定出的PIEZO1基因中的六个新的双等位基因预测LoF变异,这些家庭患有LMPHM6。在怀孕期间,大多数病例在妊娠中期通过孤立的NIHF被发现。尸检时,腹水、胸腔积液和毛细血管扩张可指导病因诊断。我们旨在进一步描述这种可能未被充分诊断的疾病的围产期表现。

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