Department of Neurology, IRCCS Humanitas Research Hospital, Rozzano, Milano, Italy.
Boston Scientific Neuromodulation, Valencia, CA, United States of America.
PLoS One. 2024 Sep 27;19(9):e0303381. doi: 10.1371/journal.pone.0303381. eCollection 2024.
Deep Brain Stimulation (DBS) is an established therapeutic approach for the treatment of dystonia. However, to date, no large-scale or comprehensive DBS dystonia patient registry has been yet undertaken. Here, we describe the protocol for a world-wide registry of clinical outcomes in dystonia patients implanted with DBS.
This protocol describes a multicenter, international clinical outcomes registry consisting of up to 200 prospectively enrolled participants at up to 40 different sites to be implanted with a constant-current, multiple independent current controlled (MICC) DBS device (Vercise DBS Systems, Boston Scientific) for treatment of dystonia. Key inclusion criteria for registry candidates include the following: understanding of study requirements and treatment procedures, a signed written informed consent form prior to participation, and meeting all criteria established in the locally applicable Instructions for Use (IFU) for the implanted DBS system. Key clinical endpoints include (but are not limited to) the evaluation of disease state (Burke-Fahn-Marsden Dystonia Rating Scale [BFMDRS], Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), quality of life (Short Form Health Survey-36, Short Form Health Survey-10), and treatment satisfaction (Clinical Global Impression of Change [CGI-Clinician; CGI-Subject; CGI-Caregiver]) at 6-months, 12-months, 2-years, and 3-years post-lead placement. Adverse events are documented and reported using structured questionnaires.
Treatment of patients with dystonia using DBS has progressed considering recent technological advances. This international dystonia outcomes registry aims to collect and evaluate real-world clinical data derived from patients who have been implanted with a constant-current, MICC-equipped DBS system (with available directional capabilities), per standard of care.
深部脑刺激（DBS）是治疗肌张力障碍的一种已确立的治疗方法。然而，迄今为止，尚未开展过针对 DBS 治疗肌张力障碍患者的大型或全面的登记研究。在此，我们介绍一项针对接受 DBS 治疗的肌张力障碍患者的全球临床结局登记研究的方案。
本方案描述了一项多中心、国际性的临床结局登记研究，该研究纳入最多 40 个不同地点的最多 200 名符合条件的前瞻性入组患者，这些患者将植入恒流、多独立电流控制（MICC）的 DBS 装置（Vercise DBS 系统，波士顿科学公司）以治疗肌张力障碍。登记候选者的主要纳入标准包括：了解研究要求和治疗程序、在参与前签署书面知情同意书、以及符合植入的 DBS 系统当地适用的使用说明（IFU）中规定的所有标准。主要临床终点包括（但不限于）疾病状态评估（Burke-Fahn-Marsden 肌张力障碍评定量表[BFMDRS]、多伦多西部痉挛性斜颈评定量表[TWSTRS]）、生活质量（健康调查简表-36，健康调查简表-10）以及治疗满意度（临床医生总体印象变化量表[CGI-Clinician；CGI-Subject；CGI-Caregiver]），评估时间分别为术后 6 个月、12 个月、2 年和 3 年。使用结构化问卷记录和报告不良事件。
考虑到最近的技术进步，使用 DBS 治疗肌张力障碍的患者已经取得了进展。这个国际性的肌张力障碍结局登记研究旨在收集和评估按照标准护理为接受恒流、MICC 配置（具有可用定向功能）的 DBS 系统植入的患者获得的真实世界临床数据。
