Randomized Trial of Focused Ultrasound Subthalamotomy for Parkinson's Disease
Randomized Trial of Focused Ultrasound Subthalamotomy for Parkinson's Disease
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DOI：
10.1056/nejmoa2016311
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发表时间：
2020-12-24
影响因子：
158.5
通讯作者：
Obeso, Jose A.
中科院分区：
文献类型：
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作者：
Martinez-Fernandez, Raul;Manez-Miro, Jorge U.;Obeso, Jose A.
丘脑底核是脑深部电刺激治疗帕金森病主要运动特征的首选神经外科靶点。聚焦超声是一种成像引导的方法，用于创建治疗性病变的脑深部结构，包括丘脑底nucleus.METHODSWe随机分配，在2：1的比例，患者明显不对称帕金森氏病的运动体征不完全控制的药物或谁是不符合条件的脑深部刺激手术进行聚焦超声丘脑底核切开术对他们的主要运动体征或假手术的一面。主要疗效结局是运动障碍协会统一帕金森病评定量表（MDS-MRS）运动评分从基线至4个月变化的组间差异（即，第III部分）在停药状态下受影响更严重的身体侧（范围0至44，评分越高表明帕金森病越严重）。在4个月时评估主要安全性结局（手术相关并发症）。在40例入组患者中，27例被分配接受聚焦超声丘脑底核切开术（积极治疗），13例接受假手术（对照）。积极治疗组中，受累侧的平均MDS-MRS III评分从基线时的19.9降至4个月时的9.9（最小二乘均值差，9.8分; 95%置信区间[CI]，8.6 - 11.1），对照组为18.7 - 17.1（最小二乘均值差，1.7分; 95%CI，0.0 - 3.5）;组间差异为8.1分（95%CI，6.0 - 10.3; P
BACKGROUNDThe subthalamic nucleus is the preferred neurosurgical target for deep-brain stimulation to treat cardinal motor features of Parkinson's disease. Focused ultrasound is an imaging-guided method for creating therapeutic lesions in deep-brain structures, including the subthalamic nucleus.METHODSWe randomly assigned, in a 2:1 ratio, patients with markedly asymmetric Parkinson's disease who had motor signs not fully controlled by medication or who were ineligible for deep-brain stimulation surgery to undergo focused ultrasound subthalamotomy on the side opposite their main motor signs or a sham procedure. The primary efficacy outcome was the between-group difference in the change from baseline to 4 months in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) motor score (i.e., part III) for the more affected body side (range, 0 to 44, with higher scores indicating worse parkinsonism) in the off-medication state. The primary safety outcome (procedure-related complications) was assessed at 4 months.RESULTSAmong 40 enrolled patients, 27 were assigned to focused ultrasound subthalamotomy (active treatment) and 13 to the sham procedure (control). The mean MDS-UPDRS III score for the more affected side decreased from 19.9 at baseline to 9.9 at 4 months in the active-treatment group (least-squares mean difference, 9.8 points; 95% confidence interval [CI], 8.6 to 11.1) and from 18.7 to 17.1 in the control group (least-squares mean difference, 1.7 points; 95% CI, 0.0 to 3.5); the between-group difference was 8.1 points (95% CI, 6.0 to 10.3; P