Child Neurology Resident, Children's Mercy Hospital, Kansas City, MO, USA.
Division of Neurology, Movement Disorders Clinic, Tourette Syndrome Center of Excellence, Children's Mercy Hospital, Professional Office Building, Office 1M02.35, 2240 Kenwood Avenue, Kansas City, MO, 64108, USA.
CNS Drugs. 2018 Jan;32(1):33-45. doi: 10.1007/s40263-017-0486-0.
Tourette syndrome (TS) is a neurodevelopmental disorder of unknown etiology characterized by spontaneous, involuntary movements and vocalizations called tics. Once thought to be rare, TS affects 0.3-1% of the population. Tics can cause physical discomfort, emotional distress, social difficulties, and can interfere with education and desired activities. The pharmacologic treatment of TS is particularly challenging, as currently the genetics, neurophysiology, and neuropathology of this disorder are still largely unknown. However, clinical experience gained from treating TS has helped us better understand its pathogenesis and, as a result, derive treatment options. The strongest data exist for the antipsychotic agents, both typical and atypical, although their use is often limited in children and adolescents due to their side-effect profiles. There are agents in a variety of other pharmacologic categories that have evidence for the treatment of TS and whose side-effect profiles are more tolerable than the antipsychotics; these include clonidine, guanfacine, baclofen, topiramate, botulinum toxin A, tetrabenazine, and deutetrabenazine. A number of new agents are being developed and tested as potential treatments for TS. These include valbenazine, delta-9-tetrahydrocannabidiol, and ecopipam. Additionally, there are agents with insufficient data for efficacy, as well as agents that have been shown to be ineffective. Those without sufficient data for efficacy include clonazepam, ningdong granule, 5-ling granule, omega-3 fatty acids, and n-acetylcysteine. The agents that have been shown to be ineffective include pramipexole and metoclopramide. We will review all of the established pharmacologic treatments, and discuss those presently in development.
妥瑞氏症（TS）是一种病因不明的神经发育障碍，其特征是自发性、不由自主的运动和发声，称为抽搐。妥瑞氏症曾被认为很少见，但实际上它影响了 0.3-1%的人口。抽搐会导致身体不适、情绪困扰、社交困难，并且会干扰教育和期望的活动。妥瑞氏症的药物治疗特别具有挑战性，因为目前这种疾病的遗传学、神经生理学和神经病理学仍在很大程度上未知。然而，从治疗妥瑞氏症中获得的临床经验帮助我们更好地理解了其发病机制，并因此得出了治疗选择。最强的数据存在于抗精神病药物中，包括典型和非典型药物，尽管由于其副作用特征，它们在儿童和青少年中的使用往往受到限制。还有许多其他药理类别的药物被证明可用于治疗妥瑞氏症，其副作用特征比抗精神病药物更耐受；这些包括可乐定、胍法辛、巴氯芬、托吡酯、肉毒杆菌毒素 A、盐酸哌甲酯和脱氢盐酸哌甲酯。许多新的药物正在被开发和测试作为妥瑞氏症的潜在治疗方法。这些包括瓦伦扎嗪、Delta-9-四氢大麻酚和 ecopipam。此外，还有一些药物的疗效数据不足，还有一些药物已被证明无效。那些疗效数据不足的药物包括氯硝西泮、宁东颗粒、5-灵颗粒、欧米伽-3 脂肪酸和 N-乙酰半胱氨酸。那些被证明无效的药物包括普拉克索和甲氧氯普胺。我们将回顾所有已确立的药物治疗方法，并讨论目前正在开发的治疗方法。
