Phua Chun Seng, Kumar Kishore Raj, Levy Stanley
Department of Neurology, Alfred Hospital, Melbourne, VIC 3004, Australia; Monash University, Department of Neuroscience, VIC 3004, Australia.
Kinghorn Centre for Clinical Genomics, Garvan Institute of Medical Research, Darlinghurst, NSW, Australia; Molecular Medicine Laboratory, Concord Repatriation General Hospital, Sydney, NSW, Australia; Neurology Department, Concord Repatriation General Hospital, Sydney, NSW, Australia; Sydney Medical School, Faculty of Medicine and Health, University of Sydney, Sydney, NSW, Australia.
J Neurol Sci. 2020 Dec 15;419:117167. doi: 10.1016/j.jns.2020.117167. Epub 2020 Oct 3.
Neurometabolic causes of dystonia are heterogenous and can be challenging to diagnose, yet many of these disorders are potentially treatable. The first step in the workup is to clinically phenotype the underlying condition, followed by ordering selected diagnostic tests based on the clinician's judgement and clinical suspicion. In this review, we highlight the diagnostic clues to various disorders, including lysosomal storage diseases, mitochondrial cytopathies, metal storage disorders, organic acidurias, disorders in carbohydrate metabolism, neurotransmitter diseases and vitamin and cofactor deficiencies. We discuss key diagnostic clues to the presence of these conditions, as well as currently available treatments. We highlight that recognition and characterization of these secondary causes of dystonia facilitate their management, including possible treatment of the underlying neurometabolic disorder.
肌张力障碍的神经代谢病因具有异质性,诊断起来可能具有挑战性,但其中许多疾病都有潜在的可治疗方法。检查的第一步是对潜在疾病进行临床表型分析,然后根据临床医生的判断和临床怀疑进行选定的诊断测试。在本综述中,我们强调了各种疾病的诊断线索,包括溶酶体贮积病、线粒体细胞病、金属贮积病、有机酸尿症、碳水化合物代谢紊乱、神经递质疾病以及维生素和辅助因子缺乏症。我们讨论了这些疾病存在的关键诊断线索以及目前可用的治疗方法。我们强调,识别和表征这些肌张力障碍的继发原因有助于对其进行管理,包括对潜在神经代谢障碍的可能治疗。