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[尼曼-匹克病的成人精神科问题]

[Adult psychiatric aspects of Niemann-Pick disease].

作者信息

Faludi Gábor, Gonda Xénia, Döme Péter

机构信息

Klinikai és Kutatási Mentálhigiénés Osztály, Semmelweis Egyetem, Kútvölgyi Klinikai Tömb, Budapest, Hungary.

出版信息

Neuropsychopharmacol Hung. 2013 Jun;15(2):95-103.

Abstract

Niemann-Pick disease (NPD) is a group of distinct rare disorders (i.e. NPD-A; NPD-B; NPD-C) - with autosomal recessive inheritance pattern - within the class of the inborn disorders of the sphingolipid metabolism (called sphingolipidoses). Since patients with NPD-A do not survive into adulthood and most patients with NPD-B are free from neuropsychiatric symptoms we discuss only briefly type-A and -B NPD and mainly constrict our review discussing the neuropsychiatric symptoms along with the pathomechanism and the treatment of NPD-C. NPD-C is clinically heterogeneous, with notable variations in age at onset, course and symptoms. Along with systemic signs, neurologic and psychiatric symptoms are quite frequent in NPD-C and in its adult form sometimes psychiatric symptoms are the first ones appearing. Unfortunately, the majority of clinicans (including adult psychiatrists and neurologists) are not aware of the symptom group characteristic to NPD-C so patients with this disorder are frequently misdiagnosed in the clinical practice. Since neuropsychiatric manifestations of NPD-C may be treated with a substrate reduction agent (miglustat) with greater awareness of the identification of neuropsychiatric symptoms in due course is the prerequisite of proper and early diagnosis and treatment.

摘要

尼曼-匹克病(NPD)是一组不同的罕见疾病(即NPD-A、NPD-B、NPD-C),具有常染色体隐性遗传模式,属于鞘脂代谢先天性疾病(称为鞘脂沉积症)。由于NPD-A患者无法存活至成年,且大多数NPD-B患者没有神经精神症状,因此我们仅简要讨论A型和B型NPD,主要集中回顾NPD-C的神经精神症状、发病机制及治疗。NPD-C在临床上具有异质性,发病年龄、病程和症状存在显著差异。除全身症状外,NPD-C患者常出现神经和精神症状,在其成人形式中,有时精神症状是最早出现的症状。不幸的是,大多数临床医生(包括成人精神科医生和神经科医生)并不了解NPD-C的症状群,因此该疾病患者在临床实践中常被误诊。由于NPD-C的神经精神表现可用一种底物减少剂(米格列醇)进行治疗,因此提高对神经精神症状的识别意识是及时、正确诊断和治疗的前提。

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