Norman Fixel Institute for Neurological Diseases at UFHealth, Movement Disorders Division, Department of Neurology, University of Florida, 3009 SW Williston Rd, Gainesville, FL, 32608, USA.
Neurotherapeutics. 2020 Oct;17(4):1582-1602. doi: 10.1007/s13311-020-00890-x.
Multiple system atrophy (MSA) is a progressive neurodegenerative disease variably associated with motor, nonmotor, and autonomic symptoms, resulting from putaminal and cerebellar degeneration and associated with glial cytoplasmic inclusions enriched with α-synuclein in oligodendrocytes and neurons. Although symptomatic treatment of MSA can provide significant improvements in quality of life, the benefit is often partial, limited by adverse effects, and fails to treat the underlying cause. Consistent with the multisystem nature of the disease and evidence that motor symptoms, autonomic failure, and depression drive patient assessments of quality of life, treatment is best achieved through a coordinated multidisciplinary approach driven by the patient's priorities and goals of care. Research into disease-modifying therapies is ongoing with a particular focus on synuclein-targeted therapies among others. This review focuses on both current management and emerging therapies for this devastating disease.
多系统萎缩（MSA）是一种进行性神经退行性疾病，与运动、非运动和自主症状有关，源于壳核和小脑变性，并伴有少突胶质细胞和神经元中富含α-突触核蛋白的神经胶质细胞质包涵体。虽然 MSA 的对症治疗可以显著提高生活质量，但益处往往是部分的，受到不良反应的限制，并且无法治疗根本原因。与疾病的多系统性质以及运动症状、自主衰竭和抑郁会影响患者对生活质量的评估的证据一致，通过以患者的优先事项和护理目标为驱动的协调多学科方法来进行治疗是最佳的。针对疾病修饰疗法的研究正在进行中，特别是针对突触核蛋白靶向疗法的研究。本综述重点介绍了这种毁灭性疾病的当前治疗方法和新兴疗法。
