Deep Brain Stimulation (DBS) of the Globus pallidus (GP) in Huntington's disease (HD): A prospective, randomized, controlled, multi-centre study

有大量证据表明脑深部电刺激（DBS）可成功对症治疗其他运动障碍，但在亨廷顿病（HD）中只有少数DBS病例报告。因此，DBS在HD对症治疗的前瞻性初步试验中进行了评价。根据我们的初步试验结果，有证据表明，苍白球DBS可显著改善HD患者的舞蹈病症状。它对总UHDRS也有积极的影响。因此，本前瞻性多中心试验旨在证明在44例HD患者中采用假刺激对照设计的苍白球DBS的短期和长期有效性和安全性。该研究的重点是对生活质量（QOL）和运动改善的潜在影响。主要终点将是UHDRS从基线到术后12周的变化（对照组无刺激vs.真实组有刺激）。此外，还将在术后12周进行一系列运动评分、定量运动测量、认知和精神评估以及QOL问卷调查。将在随后的3个月揭盲期（次要终点术后6个月）评价对这些评分的长期影响。将对UHDRS和副作用（AE）进行长达12个月的开放随访。计划中的临床试验还为额外资助的神经生理学研究开辟了前景，以探索HD患者的脑深部电刺激机制。

There is high level of evidence for deep brain stimulation (DBS) for successful symptomatic treatment of other movement disorders but only a few case reports of DBS in Huntington´s Disease (HD). Thus, DBS was evaluated in a prospective pilot trial for the symptomatic treatment of HD. Based on our pilot trial results there is evidence, that DBS of the pallidum significantly improves especially choreatic symptoms in HD. It also has positive influence on total UHDRS. Therefore this prospective multi-centre trial aims at proving the short and long-term efficacy and safety of pallidal DBS in 44 HD patients in a sham-stimulation controlled design. The study focuses on potential influence on Quality of life (QOL) and motoric improvement. Primary endpoint will be the UHDRS change from baseline to 12 weeks postoperatively (control group off stimulation vs. verum group on stimulation). Furthermore there will be a battery of motor scores, quantitative motor measurements, cognitive and psychiatric evaluations and questionnaires for QOL at 12 weeks postoperatively. Long-term effects on these scores will be evaluated in the following unblinded 3 months phase (secondary endpoint 6 months postoperatively). An open follow-up up to 12 months will be assessed for UHDRS and side effects (AE). The planned clinical trial furthermore opens perspective for additional funded neurophysiological studies to explore mechanisms of deep brain stimulation in patients with HD.