Department of Pediatric Neurology, Children's Hospital, Helsinki University Central Hospital, University of Helsinki, Helsinki, Finland.
Am J Med Genet A. 2012 Dec;158A(12):3119-25. doi: 10.1002/ajmg.a.35642. Epub 2012 Nov 19.
Hypochondroplasia (HCH), an autosomal dominant skeletal dysplasia caused by mutations in the FGFR3 gene, has not been commonly associated with neurological problems. Temporal lobe dysgenesis associated with epilepsy was recently described in single patients. In this retrospective study, we assessed neurological and neuroimaging aspects of 13 FGFR3 (N540K) mutation verified HCH patients in Finland. Eight patients had neurocognitive difficulties, ranging from specific learning disorder (2/13) to mild intellectual disability (5/13) or global developmental delay (1/13). Six of 13 patients had a history of seizures or epilepsy. Eight patients had undergone MRI. They all had structural abnormalities consistent with temporal lobe dysgenesis. Six patients had peritrigonal white matter reduction, and 4 had abnormally shaped lateral ventricles. We recommend a close follow-up of development in patients with HCH and a low threshold for neuroimaging.
软骨发育不全症(HCH)是一种常染色体显性遗传性骨骼发育不良,由 FGFR3 基因突变引起,通常与神经系统问题无关。最近在单个患者中描述了与癫痫相关的颞叶发育不良。在这项回顾性研究中,我们评估了芬兰 13 名 FGFR3(N540K)突变证实的 HCH 患者的神经和神经影像学方面。8 名患者存在神经认知困难,从特定学习障碍(2/13)到轻度智力障碍(5/13)或全面发育迟缓(1/13)不等。13 名患者中有 6 名有癫痫发作或癫痫病史。8 名患者接受了 MRI 检查。他们都有符合颞叶发育不良的结构异常。6 名患者有胼胝体周围白质减少,4 名患者有侧脑室形状异常。我们建议对 HCH 患者进行密切的发育随访,并对神经影像学检查保持低阈值。