Stokes Stephanie, Snipes Madeline, Moore Lee D, Schlabritz-Lutsevich Natalia, Amy Vidalin, Maher James
Department of Obstetrics and Gynecology, Augusta University, Augusta, Georgia, USA.
Augusta University School of Medicine, Augusta, Georgia, USA.
Mol Genet Genomic Med. 2025 Sep;13(9):e70128. doi: 10.1002/mgg3.70128.
Spinal muscular atrophy (SMA), caused by pathogenic variants in the survival motor neuron (SMN) gene, is the most common genetic cause of mortality in children under the age of two. Prior reports of obstetric sonograms performed in pregnancies with severe forms of fetal SMA have discrepant findings that may stem from a failure to account for the SMN2 copy number.
We present a neonate diagnosed with SMA type 0 postnatally (0SMN1/1SMN2 genotype). Antenatally, the fetus was noted to have HLHS (hypoplastic left heart syndrome), 2:1 AV block (atrioventricular), thickened nuchal translucency, polyhydramnios, and perceived maternal decreased fetal movement, and the mother declined genetic testing. A literature search was conducted to analyze potential prenatal findings in severe SMA type 0.
The most common associations from 32 cases of SMA type 0 include cardiac defects, increased NT (nuchal translucency), decreased fetal movement, and contractures noted postnatally. Other associations that were present in the literature and in our case include nonvertex presentation, polyhydramnios, and fractures after birth.
Prenatal onset SMA type 0 with one copy of SMN2 appears to have a distinct phenotype. Cardiac anomalies, increased nuchal translucency, and decreased maternal perception of fetal movement in the third trimester are the most frequent findings, and if found, should prompt SMA testing.
脊髓性肌萎缩症(SMA)由存活运动神经元(SMN)基因的致病变异引起,是两岁以下儿童死亡的最常见遗传原因。先前关于患有严重形式胎儿SMA的妊娠进行产科超声检查的报告结果不一致,这可能源于未考虑SMN2拷贝数。
我们报告了一名出生后被诊断为0型SMA的新生儿(0SMN1/1SMN2基因型)。产前,胎儿被发现患有左心发育不全综合征(HLHS)、2:1房室传导阻滞、颈部半透明带增厚、羊水过多,且母亲感觉胎儿活动减少,母亲拒绝进行基因检测。我们进行了文献检索,以分析严重0型SMA的潜在产前检查结果。
32例0型SMA最常见的相关表现包括心脏缺陷、颈部半透明带增加、胎儿活动减少以及出生后出现挛缩。文献及我们的病例中存在的其他相关表现包括非头先露、羊水过多和出生后骨折。
具有一份SMN2拷贝的产前发病0型SMA似乎具有独特的表型。心脏异常、颈部半透明带增加以及孕晚期母亲感觉胎儿活动减少是最常见的表现,如果发现这些情况,应促使进行SMA检测。