Neuromedicine, Institute of Postgraduate Medical Education and Research Bangur Institute of Neurology, Kolkata, West Bengal, India.
Neuromedicine, Institute of Postgraduate Medical Education and Research Bangur Institute of Neurology, Kolkata, West Bengal, India
BMJ Case Rep. 2021 Jan 25;14(1):e239630. doi: 10.1136/bcr-2020-239630.
A 5-year-old male child of consanguineous parentage, without any adverse perinatal history, presented with progressive cognitive regression predominantly in the language and attention domains, for 2 years. He had simultaneous pyramidal and extrapyramidal involvement, frequent generalised tonic-clonic seizures and recurrent respiratory tract infections. Examination was significant for vertical supranuclear gaze palsy, coarse facial features and splenomegaly. Given the clinical features, in the background of consanguinity and mother's history of spontaneous pregnancy losses, inborn errors of metabolism were suspected. Following relevant investigations including tailored genetic study, Niemann-Pick disease type C (NPC) was diagnosed. Interestingly, MRI brain showed bilateral T2/fluid-attenuated inversion recovery claustrum hyperintensities, which are more commonly associated with autoimmune encephalitis and febrile infection-related epilepsy syndrome and not reported previously in NPC. Additionally, language regression as a presenting manifestation in NPC as opposed to classical dysarthria makes this case truly unique.
一名 5 岁的男性儿童,有血缘关系的父母,无任何不良围产期病史,主要表现为语言和注意力领域的进行性认知倒退,病程为 2 年。他同时存在锥体束和锥体外系受累、频繁的全面强直阵挛发作和反复呼吸道感染。检查发现垂直核上性眼球运动麻痹、面容粗糙和脾肿大。鉴于临床表现,在血缘关系和母亲自发性妊娠丢失的背景下,考虑代谢性疾病。在进行了包括靶向基因研究在内的相关检查后,诊断为尼曼-匹克病 C 型(NPC)。有趣的是,MRI 脑显示双侧 T2/液体衰减反转恢复(FLAIR)豆状核高信号,这更常见于自身免疫性脑炎和发热感染相关癫痫综合征,而不是 NPC 中之前报道的。此外,与 NPC 中的经典构音障碍相反,语言倒退作为首发表现使这个病例非常独特。