Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona and Fundacio de Recerca Clínic Barcelona-Institut d'Investigacions Biomediques August Pi i Sunyer (FRCB-IDIBAPS), Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), Instituto de Salud Carlos III, Madrid, Spain.
Prenat Diagn. 2024 Jan;44(1):77-80. doi: 10.1002/pd.6500. Epub 2023 Dec 18.
At 16 + 6-weeks a fetal scan performed in the second pregnancy of a 42 y.o. woman identified a right multicystic dysplastic kidney, left renal agenesis, absent urinary bladder, myocardial hypertrophy, increased nuchal fold, a single umbilical artery, and oligohydramnios. Trio exome sequencing analysis detected a novel pathogenic NONO variant. Postmortem examination after the termination of pregnancy confirmed the ultrasound findings and also revealed pulmonary hypoplasia, retrognathia and low-set ears. The variant was a novel de novo hemizygous pathogenic loss-of-function variant in NONO [NM_007363.5], associated with a rare X-linked recessive neurodevelopmental disorder, named intellectual developmental disorder, X-linked syndromic 34 (OMIM#300967). The postnatal characteristic features of this disorder include intellectual disability, developmental delay, macrocephaly, structural abnormalities involving the corpus callosum and/or cerebellum, left ventricular noncompaction and other congenital heart defects. In the prenatal setting, the phenotype has been poorly described, with all described cases presenting with heart defects. This case highlights the need of further clinical delineation to include renal abnormalities in the prenatal phenotype spectrum.
在第二次妊娠 16+6 周时,对一位 42 岁女性进行了胎儿扫描,发现右侧多囊性发育不良肾、左侧肾发育不全、无膀胱、心肌肥厚、颈后皮肤皱褶增厚、单脐动脉和羊水过少。三核苷酸外显子组测序分析发现了一个新的致病性 NONO 变异。妊娠终止后的尸检检查证实了超声检查的结果,还发现了肺发育不良、小下颌和低位耳。该变异是 NONO[NM_007363.5]中的新型新生纯合致病性功能丧失变异,与一种罕见的 X 连锁隐性神经发育障碍有关,命名为智力发育障碍、X 连锁综合征 34(OMIM#300967)。该疾病的产后特征包括智力残疾、发育迟缓、大头畸形、胼胝体和/或小脑结构异常、左心室致密化不全和其他先天性心脏缺陷。在产前环境中,该表型描述较差,所有描述的病例均存在心脏缺陷。本病例强调需要进一步的临床描述,将肾脏异常纳入产前表型谱中。