The efficacy and limits of magnetic resonance-guided focused ultrasound pallidotomy for Parkinson's disease: a Phase I clinical trial
The efficacy and limits of magnetic resonance-guided focused ultrasound pallidotomy for Parkinson's disease: a Phase I clinical trial
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DOI：
10.3171/2018.2.jns172514
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发表时间：
2019-06-01
影响因子：
4.1
通讯作者：
Chang, Jin Woo
中科院分区：
文献类型：
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作者：
Jung, Na Young;Park, Chang Kyu;Chang, Jin Woo
目的近年来，磁共振引导聚焦超声（MRgFUS）作为一种创新的治疗方法，已成为治疗包括特发性震颤、帕金森病（PD）和一些精神疾病在内的多种神经系统疾病的新方法。因此，已经使用各种目标尝试了这种模式的临床应用。本研究的目的是确定的可行性，初步的有效性，和潜在的副作用，单侧MRgFUS苍白球毁损术治疗帕金森病dyskinesia.METHODS的前瞻性，非随机，单臂临床试验进行了2013年12月和2016年5月之间在一个单一的三级医疗中心。入组了10例药物难治性、运动障碍为主的PD患者。在提供书面知情同意书后，参与者使用Exablate 4000器械（InSightec）接受了单侧MRgFUS苍白球切开术。根据1年随访方案，对患者的运动改善、神经心理学影响和不良事件进行了系列评价。主要指标包括统一帕金森病评定量表（UPDRS）和统一运动障碍评定量表（UDysRS）评分从基线到1周、1个月、3个月、6个月和1年的变化。次要指标包括神经心理成套测验和生活质量问卷（SF-36）。技术故障和安全性问题也仔细评估，通过监测所有事件在研究periods.Results单侧MRgFUS苍白球毁损术成功地进行了10例（80%），患者随访超过6个月。临床结局显示，在6个月随访时，“停药”后的ADIRS第III部分评分显著改善32.2%（p = 0.018），UDysRS显著改善52.7%（p = 0.017），1年随访时分别显著改善39.1%（p = 0.046）和42.7%（p = 0.046）。这些结果伴随着生活质量的改善。在8例中，1例患者遭受了不寻常的副作用的超声处理，但是，没有患者经历了持续的aftereffects.CONCLUSIONS在本研究中，这标志着第一个阶段I试点研究的单方面MRgFUS苍白球毁损术帕金森氏症运动障碍，作者证明了苍白球毁损使用MRgFUS和某些局限性，这是不完全的热毁损，由于技术问题，必然相关的疗效。需要进一步调查和长期随访，以验证MRgFUS在临床实践中的使用。
OBJECTIVE Recently, MR-guided focused ultrasound (MRgFUS) has emerged as an innovative treatment for numerous neurological disorders, including essential tremor, Parkinson's disease (PD), and some psychiatric disorders. Thus, clinical applications with this modality have been tried using various targets. The purpose of this study was to determine the feasibility, initial effectiveness, and potential side effects of unilateral MRgFUS pallidotomy for the treatment of parkinsonian dyskinesia.METHODS A prospective, nonrandomized, single-arm clinical trial was conducted between December 2013 and May 2016 at a single tertiary medical center. Ten patients with medication-refractory, dyskinesia-dominant PD were enrolled. Participants underwent unilateral MRgFUS pallidotomy using the Exablate 4000 device (InSightec) after providing written informed consent. Patients were serially evaluated for motor improvement, neuropsychological effects, and adverse events according to the 1-year follow-up protocol. Primary measures included the changes in the Unified Parkinson's Disease Rating Scale (UPDRS) and Unified Dyskinesia Rating Scale (UDysRS) scores from baseline to 1 week, 1 month, 3 months, 6 months, and 1 year. Secondary measures consisted of neuropsychological batteries and quality of life questionnaire (SF-36). Technical failure and safety issues were also carefully assessed by monitoring all events during the study period.RESULTS Unilateral MRgFUS pallidotomy was successfully performed in 8 of 10 patients (80%), and patients were followed up for more than 6 months. Clinical outcomes showed significant improvements of 32.2% in the "medication-off" UPDRS part III score (p = 0.018) and 52.7% in UDysRS (p = 0.017) at the 6-month follow-up, as well as 39.1% (p = 0.046) and 42.7% (p = 0.046) at the 1-year follow-up, respectively. These results were accompanied by improvement in quality of life. Among 8 cases, 1 patient suffered an unusual side effect of sonication; however, no patient experienced persistent aftereffects.CONCLUSIONS In the present study, which marks the first Phase I pilot study of unilateral MRgFUS pallidotomy for parkinsonian dyskinesia, the authors demonstrated the efficacy of pallidal lesioning using MRgFUS and certain limitations that are unavoidably associated with incomplete thermal lesioning due to technical issues. Further investigation and long-term follow-up are necessary to validate the use of MRgFUS in clinical practice.