Plasschaert Ellen, Descheemaeker Mie-Jef, Van Eylen Lien, Noens Ilse, Steyaert Jean, Legius Eric
Department of Human Genetics, Laboratory for Neurofibromatosis Research, KU Leuven, Leuven, Belgium; Center for Human Genetics, UZ Leuven, Leuven, Belgium.
Am J Med Genet B Neuropsychiatr Genet. 2015 Jan;168B(1):72-80. doi: 10.1002/ajmg.b.32280. Epub 2014 Nov 12.
Neurofibromatosis type 1 (NF1) is an autosomal dominant genetic condition. While considerable work has focused on cognitive functioning, several research groups also observed difficulties in social functioning as a prominent feature of NF1. These problems and the possible link between NF1 and Autism Spectrum Disorder (ASD) have become increasingly important in recent NF1 literature. The aim of the current study was to assess ASD characteristics in a hospital-based NF1 pediatric population (n = 82) using the standardized Children Social Behavior Questionnaire (CSBQ) and Social Responsiveness Scale (SRS) to account for the prevalence, severity, and nature of social problems. In a parallel study, comprehensive ASD assessment was performed in a subgroup of NF1 children with a strong suspicion of ASD (n = 31). Results indicate that NF1 children have more social problems than typical controls, more frequently reported above 8 years. The SRS shows that 63% is at risk of ASD symptoms. According to item analyses, most problems were observed on items measuring orientation in, understanding of and being tuned onto a social situation (CSBQ) and social cognition and communication (SRS). In the parallel study, 27 NF1 children were diagnosed with ASD. These children have a distinct phenotype compared to a heterogeneous ASD group, with pronounced social-communicative impairments and fewer restrictive/repetitive behaviors. This study provides a better understanding of social problems in NF1 and the phenotypical overlap with ASD symptomatology. Despite their willingness to engage with others, NF1 children with or without ASD encounter various difficulties in their social-communicative life.
1型神经纤维瘤病(NF1)是一种常染色体显性遗传病。虽然大量研究聚焦于认知功能,但多个研究小组也观察到社交功能障碍是NF1的一个突出特征。在最近的NF1文献中,这些问题以及NF1与自闭症谱系障碍(ASD)之间可能存在的联系变得越来越重要。本研究的目的是使用标准化的儿童社会行为问卷(CSBQ)和社会反应量表(SRS),对以医院为基础的NF1儿科人群(n = 82)进行ASD特征评估,以了解社交问题的患病率、严重程度和性质。在一项平行研究中,对31名高度怀疑患有ASD的NF1儿童亚组进行了全面的ASD评估。结果表明,NF1儿童比正常对照组有更多的社交问题,且在8岁以上儿童中更为常见。SRS显示,63%的儿童有ASD症状风险。根据项目分析,大多数问题出现在测量对社交情境的定向、理解和融入(CSBQ)以及社会认知和沟通(SRS)的项目上。在平行研究中,27名NF1儿童被诊断为患有ASD。与异质性ASD组相比,这些儿童具有独特的表型,存在明显的社会沟通障碍,且限制/重复行为较少。本研究有助于更好地理解NF1中的社交问题以及与ASD症状的表型重叠。尽管NF1儿童无论是否患有ASD都愿意与他人交往,但他们在社会交往生活中仍会遇到各种困难。