Department of Neurology, Vanderbilt University Medical Center, Nashville, TN, USA.
J Parkinsons Dis. 2012;2(3):215-23. doi: 10.3233/JPD-2012-012095.
Deep brain stimulation provides significant symptomatic benefit for people with advanced Parkinson's disease whose symptoms are no longer adequately controlled with medication. Preliminary evidence suggests that subthalamic nucleus stimulation may also be efficacious in early Parkinson's disease, and results of animal studies suggest that it may spare dopaminergic neurons in the substantia nigra.
We report the methodology and design of a novel Phase I clinical trial testing the safety and tolerability of deep brain stimulation in early Parkinson's disease and discuss previous failed attempts at neuroprotection.
We recently conducted a prospective, randomized, parallel-group, single-blind pilot clinical trial of deep brain stimulation in early Parkinson's disease. Subjects were randomized to receive either optimal drug therapy or deep brain stimulation plus optimal drug therapy. Follow-up visits occurred every six months for a period of two years and included week-long therapy washouts.
Thirty subjects with Hoehn & Yahr Stage II idiopathic Parkinson's disease were enrolled over a period of 32 months. Twenty-nine subjects completed all follow-up visits; one patient in the optimal drug therapy group withdrew from the study after baseline. Baseline characteristics for all thirty patients were not significantly different.
This study demonstrates that it is possible to recruit and retain subjects in a clinical trial testing deep brain stimulation in early Parkinson's disease. The results of this trial will be used to support the design of a Phase III, multicenter trial investigating the efficacy of deep brain stimulation in early Parkinson's disease.
深部脑刺激为药物治疗效果不佳的晚期帕金森病患者提供了显著的症状改善。初步证据表明，丘脑底核刺激对早期帕金森病也可能有效，动物研究结果表明它可能保护黑质中的多巴胺能神经元。
我们报告了一项新的 I 期临床试验的方法和设计，该试验旨在测试早期帕金森病深部脑刺激的安全性和耐受性，并讨论之前神经保护失败的尝试。
我们最近进行了一项前瞻性、随机、平行组、单盲的早期帕金森病深部脑刺激的试点临床试验。受试者被随机分配接受最佳药物治疗或深部脑刺激加最佳药物治疗。随访每六个月进行一次，为期两年，并包括为期一周的治疗洗脱期。
在 32 个月的时间里，共有 30 名 Hoehn & Yahr 分期 II 期特发性帕金森病患者入组。29 名受试者完成了所有随访；药物治疗组的一名患者在基线后退出了研究。所有 30 名患者的基线特征无显著差异。
本研究表明，在早期帕金森病深部脑刺激的临床试验中招募和保留受试者是可行的。该试验的结果将用于支持一项 III 期、多中心研究的设计，该研究旨在调查深部脑刺激对早期帕金森病的疗效。
