一项针对帕金森病痴呆患者深部脑刺激治疗运动症状的随机对照试验方案(DBS-MODE)。

Protocol of a randomized controlled trial investigating Deep Brain Stimulation for MOtor symptoms in patients with Parkinson's disease DEmentia (DBS-MODE).

机构信息

Amsterdam UMC location University of Amsterdam, Neurology, Meibergdreef 9, Amsterdam, Netherlands.

Amsterdam Neuroscience, Neurodegeneration, Amsterdam, Netherlands.

出版信息

BMC Neurol. 2023 Apr 21;23(1):160. doi: 10.1186/s12883-023-03142-5.

DOI:10.1186/s12883-023-03142-5

PMID:37085773

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10120102/

Abstract

BACKGROUND

Deep brain stimulation (DBS) of the subthalamic nucleus (STN) is an established treatment for disabling motor symptoms of Parkinson's disease (PD) that persist despite optimal pharmacological treatment. Currently, DBS is not performed if there is concomitant significant cognitive impairment based on concerns of cognitive deterioration, higher complication rate and less functional improvement. However, this has not been investigated so far.

METHODS

A single center, prospective, randomized, open-label, blinded end-point (PROBE design) pilot clinical trial is being performed. Patients are eligible for the trial if they have PD dementia (PDD), are able to provide informed consent, and experience disabling motor response fluctuations, bradykinesia, dyskinesia, or painful dystonia, despite optimal pharmacological treatment. In total 44 patients will be randomized to either STN-DBS accompanied by best medical treatment (DBS group) or to best medical treatment alone (BMT group). The primary outcome measure is the change from baseline to 30 weeks on the Movement Disorder Society-Unified Parkinson's Disease Rating Scale part III score in a standardized off-drug phase. The main secondary outcome measures consist of scales assessing cognitive aspects of daily living, neuropsychiatric symptoms and impulsive compulsive disorders. Additional secondary outcome measures include motor signs during on-drug phase, dyskinesia, motor fluctuations, cognitive performance, (severe) adverse events, treatment satisfaction, and caregiver burden. Patients will be followed during 52 weeks after randomization.

DISCUSSION

The Deep Brain Stimulation for MOtor symptoms in patients with Parkinson's disease DEmentia (DBS-MODE) trial directly compares the effectiveness and safety of DBS with BMT in patients with PDD.

TRIAL REGISTRATION

The DBS-MODE trial has been registered in the International Clinical Trial Registry Platform (NL9361) on the 24 of March 2021 ( https://trialsearch.who.int/Trial2.aspx?TrialID=NL9361 ).

摘要

背景

深部脑刺激（DBS）于丘脑底核（STN）是一种已确立的治疗方法，适用于尽管经过最佳药物治疗但仍持续存在的帕金森病（PD）致残运动症状。目前，如果存在同时伴有显著认知障碍，则不进行 DBS，因为担心认知恶化、更高的并发症发生率和更少的功能改善。然而，到目前为止，这一点尚未得到调查。

方法

正在进行一项单中心、前瞻性、随机、开放标签、盲终点（PROBE 设计）的试点临床试验。如果患者患有帕金森病痴呆（PDD），能够提供知情同意，并且尽管经过最佳药物治疗仍经历致残性运动波动、运动迟缓、运动障碍或疼痛性肌张力障碍，则有资格参加试验。共有 44 名患者将被随机分为 STN-DBS 联合最佳药物治疗（DBS 组）或仅最佳药物治疗（BMT 组）。主要结局测量指标是在标准化停药期内从基线到 30 周的运动障碍协会统一帕金森病评定量表第三部分评分的变化。主要次要结局指标包括评估日常生活认知方面、神经精神症状和冲动强迫障碍的量表。其他次要结局指标包括药物治疗期的运动体征、运动障碍、运动波动、认知表现、（严重）不良事件、治疗满意度和照顾者负担。患者将在随机分组后 52 周内进行随访。