Panaitescu Anca Maria, Huluță Iulia, Gorecki Gabriel-Petre, Cima Luminita Nicoleta, Voiculescu Vlad M, Nedelea Florina Mihaela, Gică Nicolae
Department of Obstetrics and Gynecology, Faculty of Medicine, Carol Davila University of Medicine and Pharmacy, 020021 Bucharest, Romania.
Department of Obstetrics and Gynecology, Filantropia Clinical Hospital Bucharest, 011171 Bucharest, Romania.
Children (Basel). 2024 Mar 5;11(3):310. doi: 10.3390/children11030310.
MIRAGE syndrome is a recently described congenital condition characterized genetically by heterozygous gain-of-function missense mutations in the growth repressor sterile alpha domain containing 9 (SAMD9) located on the arm of chromosome 7 (7q21.2). The syndrome is rare and is usually diagnosed in newborns and children with myelodysplasia, infection, restriction of growth, adrenal hypoplasia, genital phenotypes, and enteropathy, hence the acronym MIRAGE. The aims of this paper are (1) to present fetal ultrasound features in a case where MIRAGE syndrome was diagnosed prenatally and (2) to review the existing literature records on prenatal manifestations of MIRAGE syndrome. In our case, the fetus had severe early fetal growth restriction (FGR) with normal Doppler studies, atypical genitalia, oligohydramnios, and hyperechogenic bowel at the routine mid-gestation anomaly scan. Amniocentesis excluded infections and numeric or structural chromosomal abnormalities while whole exome sequencing (WES) of the fetal genetic material identified the specific mutation. Targeted testing in parents was negative, suggesting the "de novo" mutation in the fetus. We could not identify other specific case reports in the literature on the prenatal diagnosis of MIRAGE syndrome. In cases reported in the literature where the diagnosis of MIRAGE syndrome was achieved postnatally, there are mentions related to the marked FGR on prenatal ultrasound. Severe early-onset FGR with no other apparent cause seems to be a central prenatal feature in these babies, and WES should be offered, especially if there are other structural abnormalities. Prenatal diagnosis of MIRAGE syndrome is possible, allowing for reproductive choices, improved counseling of parents, and better preparation of neonatal care.
MIRAGE综合征是一种最近才被描述的先天性疾病,其遗传学特征是位于7号染色体长臂(7q21.2)上的生长抑制因子含无菌α结构域9(SAMD9)发生杂合性功能获得性错义突变。该综合征很罕见,通常在患有骨髓发育异常、感染、生长受限、肾上腺发育不全、生殖器表型和肠病的新生儿和儿童中被诊断出来,因此得名MIRAGE。本文的目的是:(1)介绍一例产前诊断为MIRAGE综合征的胎儿超声特征;(2)回顾有关MIRAGE综合征产前表现的现有文献记录。在我们的病例中,胎儿在常规孕中期异常扫描时出现严重的早期胎儿生长受限(FGR),多普勒检查正常,生殖器不典型,羊水过少,肠回声增强。羊水穿刺排除了感染以及数目或结构染色体异常,而对胎儿遗传物质进行的全外显子组测序(WES)确定了特定突变。对父母进行的靶向检测为阴性,提示胎儿存在“新发”突变。我们在文献中未能找到其他关于MIRAGE综合征产前诊断的具体病例报告。在文献中报道的产后诊断为MIRAGE综合征的病例中,提到了产前超声显示的明显FGR。这些婴儿产前的主要特征似乎是无其他明显原因的严重早发性FGR,应进行WES检测,尤其是在存在其他结构异常的情况下。MIRAGE综合征的产前诊断是可能的,这有助于做出生育选择,改善对父母的咨询,并更好地准备新生儿护理。