Danube University Krems, Vienna, Austria. Europe.
Adv Exp Med Biol. 2012;942:187-213. doi: 10.1007/978-94-007-2869-1_8.
Though inherited mitochondrial disorders (MIDs) are most well known for their syndromic forms, for which widely known acronyms (MELAS, MERRF, NARP, LHON etc.) have been coined, the vast majority of inherited MIDs presents in a non-syndromic form. Since MIDs are most frequently multisystem disorders already at onset or during the disease course, a MID should be suspected if there is a combination of neurological and non-neurological abnormalities. Neurological abnormalities occurring as a part of a MID include stroke-like episodes, epilepsy, migraine-like headache, movement disorders, cerebellar ataxia, visual impairment, encephalopathy, cognitive impairment, dementia, psychosis, hypopituitarism, aneurysms, or peripheral nervous system disease, such as myopathy, neuropathy, or neuronopathy. Non-neurological manifestations concern the ears, the endocrine organs, the heart, the gastrointestinal tract, the kidneys, the bone marrow, and the skin. Whenever there is an unexplained combination of neurological and non-neurological disease in a patient or kindred, a MID should be suspected and appropriate diagnostic measures initiated. Genetic testing should be guided by the phenotype, the biopsy findings, and the biochemical results.
虽然遗传性线粒体疾病 (MID) 以其综合征形式最为人所知,为此已经创造了广为人知的缩写词(MELAS、MERRF、NARP、LHON 等),但绝大多数遗传性 MID 以非综合征形式出现。由于 MID 通常在发病时或疾病过程中已经是多系统疾病,因此如果存在神经和非神经异常的组合,应怀疑存在 MID。作为 MID 一部分的神经系统异常包括中风样发作、癫痫、偏头痛样头痛、运动障碍、小脑共济失调、视力障碍、脑病、认知障碍、痴呆、精神病、垂体功能减退、动脉瘤或周围神经系统疾病,如肌病、神经病或神经元病。非神经系统表现涉及耳朵、内分泌器官、心脏、胃肠道、肾脏、骨髓和皮肤。每当患者或亲属出现不明原因的神经和非神经疾病组合时,应怀疑存在 MID 并启动适当的诊断措施。基因检测应根据表型、活检结果和生化结果进行指导。