Lina M Zapata-Restrepo, Bruce L Miller, Juan Rivas, Katherine Possin, Victor Valcour, Stefanie D Piña-Escudero, Agustin Ibañez, Kenneth S Kosik
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引用次数: 0
摘要
早发性阿尔茨海默病(EOAD)是一种罕见的痴呆症,通常比晚发性病例进展更快,更常与常染色体显性突变相关。一名47岁男性表现为进行性认知和行为下降,有EOAD家族史,后来发现有一种新的致病性PSEN1变异(c.519 G > T, p.Leu173Phe)。包括神经成像和实验室检查在内的初步评估结果并不显著。随后的神经心理测试显示记忆障碍、执行功能障碍和神经精神症状。这些特征,以及鉴定的突变,与涉及PSEN1的第三跨膜结构域的EOAD的表型表现一致。用胆碱酯酶抑制剂和抗精神病药物治疗效果有限。值得注意的是,延长的随访时间,从早期症状阶段开始超过10年,是本病例研究的一个独特而有价值的特征,为遗传证实的EOAD的自然过程提供了罕见的纵向见解。
Case of early onset Alzheimer's disease associated with a novel PSEN1 variant identified in Colombia.
Early-onset Alzheimer's disease (EOAD) is a rare form of dementia that often progresses more quickly than late-onset cases, and is more commonly associated with autosomal dominant mutations. A 47-year-old male presented with progressive cognitive and behavioral decline, a family history of EOAD, and was later found to have a novel pathogenic PSEN1 variant (c.519 G > T, p.Leu173Phe). Initial evaluations, including neuroimaging and laboratory tests, were unremarkable. Neuropsychological testing later revealed memory impairment, executive dysfunction, and neuropsychiatric symptoms. These features, alongside the identified mutation, are consistent with phenotypic presentations of EOAD involving the third transmembrane domain of PSEN1. Pharmacological treatment with cholinesterase inhibitors and antipsychotics yielded limited benefit. Notably, the extended follow-up time, of more than 10 years from the early symptomatic stage, is a unique and valuable feature of this case study, providing rare longitudinal insight into the natural course of genetically confirmed EOAD.