Perez-Lloret Santiago, Flabeau Olivier, Fernagut Pierre-Olivier, Pavy-Le Traon Anne, Rey María Verónica, Foubert-Samier Alexandra, Tison Francois, Rascol Olivier, Meissner Wassilios G
Laboratory of Epidemiology and Experimental Pharmacology Institute for Biomedical Research (BIOMED) School of Medical Sciences Pontifical Catholic University of Argentina (UCA) Buenos Aires Argentina.
The National Scientific and Technical Research Council (CONICET) Buenos Aires Argentina.
Mov Disord Clin Pract. 2015 Feb 2;2(1):6-16. doi: 10.1002/mdc3.12145. eCollection 2015 Mar.
MSA is a progressive neurodegenerative disorder characterized by autonomic failure and a variable combination of poor levodopa-responsive parkinsonism and cerebellar ataxia (CA). Current therapeutic management is based on symptomatic treatment. Almost one third of MSA patients may benefit from l-dopa for the symptomatic treatment of parkinsonism, whereas physiotherapy remains the best therapeutic option for CA. Only midodrine and droxidopa were found to be efficient for neurogenic hypotension in double-blind, controlled studies, whereas other symptoms of autonomic failure may be managed with off-label treatments. To date, no curative treatment is available for MSA. Recent results of neuroprotective and -restorative trials have provided some hope for future advances. Considerations for future clinical trials are also discussed in this review.
多系统萎缩（MSA）是一种进行性神经退行性疾病，其特征为自主神经功能衰竭，以及左旋多巴反应性差的帕金森综合征和小脑共济失调（CA）的多种不同组合。目前的治疗管理基于对症治疗。几乎三分之一的MSA患者可能从左旋多巴治疗帕金森综合征的症状中获益，而物理治疗仍是CA的最佳治疗选择。在双盲对照研究中，仅发现米多君和屈昔多巴对神经源性低血压有效，而自主神经功能衰竭的其他症状可采用标签外治疗。迄今为止，尚无针对MSA的治愈性治疗方法。神经保护和恢复性试验的最新结果为未来的进展带来了一些希望。本综述还讨论了未来临床试验的相关考虑因素。
