Gavril Eva-Cristiana, Luca Alina Costina, Curpan Alexandrina-Stefania, Popescu Roxana, Resmerita Irina, Panzaru Monica Cristina, Butnariu Lacramioara Ionela, Gorduza Eusebiu Vlad, Gramescu Mihaela, Rusu Cristina
Department of Medical Genetics, Faculty of Medicine, "Grigore T. Popa" University of Medicine and Pharmacy, University Street, No 16, 700115 Iasi, Romania.
Department of Pediatric Cardiology, Faculty of Medicine, "Grigore T. Popa" University of Medicine and Pharmacy, University Street, No 16, 700115 Iasi, Romania.
Children (Basel). 2021 Aug 30;8(9):751. doi: 10.3390/children8090751.
Wolf-Hirschhorn syndrome (WHS), a rare disorder determined by distal 4p deletion, is characterized by a pre and postnatal growth retardation, hypotonia, intellectual disability, epilepsy, craniofacial dysmorphism, and congenital fusion anomalies. The clinical aspects are dependent on the deletion' size. Our aim was to identify rare specific characteristics in a cohort of seven cases with 4p deletion and to assess the utility of Multiplex ligation-dependent probe amplification (MLPA) (cheap and sensitive test)-combined kits-as a diagnostic test and selection tool for cases that require other investigations (chromosomal microarray analysis-CMA, karyotype). For all cases we conducted a clinical examination with the main features identified: facial dysmorphism, intellectual disability, postnatal development delay, cardiac defects and hypotonia. In some cases, we observed seizures, structural brain abnormalities, immunodeficiencies, and renal anomalies. Prenatal growth retardation was detected in a relatively small number of cases, but postnatal growth failure was a constant feature. In all cases, the clinical diagnosis was confirmed by genetic analyses: karyotype and/or MLPA. In conclusion, renal and brain defects, as well as immunodeficiency are rare manifestations and should be looked for. Although CMA is the standard test, in our experience, MLPA is also a reliable screening method as the identified cases were either confirmed by MLPA or selected for further investigations.
沃尔夫-赫希霍恩综合征(WHS)是一种由4号染色体短臂末端缺失导致的罕见疾病,其特征为出生前后生长发育迟缓、肌张力低下、智力障碍、癫痫、颅面畸形以及先天性融合异常。临床症状取决于缺失的大小。我们的目的是在一组7例4号染色体短臂缺失病例中识别罕见的特定特征,并评估多重连接依赖探针扩增(MLPA)(一种廉价且灵敏的检测方法)联合试剂盒作为诊断检测以及针对需要其他检查(染色体微阵列分析-CMA、核型分析)的病例的筛选工具的效用。对于所有病例,我们进行了临床检查,确定了主要特征:面部畸形、智力障碍、出生后发育迟缓、心脏缺陷和肌张力低下。在某些病例中,我们观察到癫痫发作、脑结构异常、免疫缺陷和肾脏异常。产前生长发育迟缓在相对少数病例中被检测到,但出生后生长发育不良是一个持续存在的特征。在所有病例中,临床诊断均通过基因分析得以证实:核型分析和/或MLPA。总之,肾脏和脑部缺陷以及免疫缺陷是罕见的表现,应予以关注。尽管CMA是标准检测方法,但根据我们的经验,MLPA也是一种可靠的筛查方法,因为所识别的病例要么通过MLPA得以确诊,要么被选作进一步检查。