Hsu Mandy, Majeed Abdul-Jawad J, Newmaster Kyra T, Harkins Elias, Mainali Gayatra, Naik Sunil, Paudel Sita
Pediatric Neurology, Penn State College of Medicine, Hershey, USA.
Neurology, Children's Hospital Los Angeles, Los Angeles, USA.
Cureus. 2025 Apr 10;17(4):e82039. doi: 10.7759/cureus.82039. eCollection 2025 Apr.
Chari malformations are a group of developmental malformations involving the cerebellum, classified into four subtypes. Type 1 CM (CM1) can cause a heterogenous set of symptoms, ranging from neck pain, occipital headache, and nystagmus to cranial nerve palsies, limb numbness, and cerebellar dysfunction. Diagnosis can be particularly challenging in part due to the lack of understanding surrounding its pathogenesis, though it has been suggested that there may be some genetic component contributing. This report describes a case of familial CM1 in which a mother and her two daughters all demonstrated symptomatic and radiographic evidence of CM1. Notably, the two children presented with symptoms requiring surgical decompression prior to the age of three, which is uncommon. The mother only presented with symptoms after giving birth to her two children. Though the children both had initial symptoms, including difficulty swallowing as well as an established growth hormone deficiency, the mother did not present with such symptoms and did not have any known hypothalamic dysfunction. The pathogenesis of CM1 remains unknown, though it has been suggested that there is some genetic component involved. This report of familial CM1 adds to the body of literature. Familial MRI head screening and whole exome sequencing in individuals with family members showing signs of CM1 could help reveal a genetic pathogenesis for CM1.
Chiari畸形是一组涉及小脑的发育性畸形,分为四个亚型。1型Chiari畸形(CM1)可导致一系列不同的症状,从颈部疼痛、枕部头痛、眼球震颤到颅神经麻痹、肢体麻木和小脑功能障碍。诊断可能特别具有挑战性,部分原因是对其发病机制缺乏了解,尽管有人认为可能存在一些遗传因素。本报告描述了一例家族性CM1病例,其中一位母亲和她的两个女儿均表现出CM1的症状和影像学证据。值得注意的是,这两个孩子在三岁之前就出现了需要手术减压的症状,这并不常见。这位母亲在生下两个孩子后才出现症状。尽管两个孩子最初都有症状,包括吞咽困难以及已确诊的生长激素缺乏症,但母亲并未出现此类症状,也没有任何已知的下丘脑功能障碍。CM1的发病机制仍然未知,尽管有人认为涉及一些遗传因素。这份家族性CM1报告丰富了文献资料。对有家庭成员显示CM1迹象的个体进行家族性头部MRI筛查和全外显子组测序,可能有助于揭示CM1的遗传发病机制。