Neuroimaging. Hospital Universitario Quirónsalud, Madrid, Spain.
Department of Pediatric Neurology. Hospital Universitario Quirónsalud, Madrid, Spain.
Eur J Paediatr Neurol. 2021 Nov;35:8-15. doi: 10.1016/j.ejpn.2021.09.008. Epub 2021 Sep 16.
KBG syndrome is characterized by dental, craniofacial and skeletal anomalies, short stature and global developmental delay or intellectual disability. It is caused by microdeletions or truncating mutations of ANKRD11. We report four unrelated probands with this syndrome due to de novo ANKRD11 aberrations that may contribute to a better understanding of the genetics and pathophysiology of this autosomal dominant syndrome. Clinical, cognitive and MRI assessments were performed. Three of the patients showed normal intellectual functioning, whereas the fourth had a borderline level of intellectual functioning. However, all of them showed deficits in various cognitive and socioemotional processes such as attention, executive functions, empathy or pragmatic language. Moreover, all probands displayed marked asymmetry of the uncinate fascicles and an abnormal gyrification pattern in the left frontal lobe. Thus, structural neuroimaging anomalies seem to have been overlooked in this syndrome. Disturbed frontal gyrification and/or lower structural integrity of the uncinate fascisulus might be unrecognized neuroimaging features of KBG syndrome caused by ANKRD11 aberrations. Present results also point out that this syndrome is not necessarily associated with global developmental delay and intellectual disability, but it can be related to other neurodevelopmental disorders or subclinical levels of attention-deficit hyperactivity disorder, autism, communication disorders or specific learning disabilities.
KBG 综合征的特征为牙齿、颅面和骨骼异常、身材矮小以及全面发育迟缓或智力残疾。其病因是ANKRD11 的微缺失或截断突变。我们报道了 4 例由 ANKRD11 异常导致的该综合征的无关联先证者,这可能有助于更好地理解该常染色体显性综合征的遗传学和病理生理学。进行了临床、认知和 MRI 评估。其中 3 例患者的智力功能正常,而第 4 例患者的智力功能处于边缘水平。然而,他们所有人都在注意力、执行功能、同理心或实用语言等各种认知和社会情感过程中表现出缺陷。此外,所有先证者均显示钩束的明显不对称和左侧额叶的异常脑回模式。因此,结构神经影像学异常似乎在该综合征中被忽视了。额叶脑回发育不良和/或钩束结构完整性降低可能是ANKRD11 异常导致的 KBG 综合征的未被识别的神经影像学特征。目前的结果还表明,该综合征不一定与全面发育迟缓或智力残疾相关,但它可能与其他神经发育障碍或亚临床水平的注意缺陷多动障碍、自闭症、沟通障碍或特定学习障碍相关。