Department of Neurosurgery, Tokyo Women's Medical University, Tokyo, Japan.
Shinyurigaoka General Hospital, Kanagawa, Japan.
Neurosurgery. 2021 Mar 15;88(4):751-757. doi: 10.1093/neuros/nyaa536.
Several feasibility studies and a randomized, controlled, multicenter trial have demonstrated the safety and efficacy of unilateral transcranial magnetic resonance-guided focused ultrasound (FUS) lesioning of the ventral intermediate thalamic nucleus in treating essential tremor.
To evaluate the safety and efficacy of FUS thalamotomy in a Japanese patient cohort through a prospective, multicenter, single-arm confirmatory trial.
A total of 35 patients with disabling refractory essential tremor underwent unilateral FUS thalamotomy and were followed up for 12 post-treatment months. Safety was measured as the incidence and severity of treatment-related adverse events. Efficacy was measured as the tremor severity and quality of life improvements using the Clinical Rating Scale for Tremor and Questionnaire for Essential Tremor.
The mean skull density ratio (SDR) was 0.47. There was a significant decrease in the mean postural tremor score of the treated hand from baseline to 12 mo by 56.4% (95% CI: 46.7%-66.1%; P < .001), which was maintained at last follow-up. Quality of life improved by 46.3% (mean overall Questionnaire for Essential Tremor score of 17.4 [95% CI: 12.1-22.7]) and there were no severe adverse events. The most frequent adverse event was gait disturbance and all events resolved.
Unilateral FUS thalamotomy allowed significant and sustained tremor relief and improved the quality of life with an outstanding safety profile. The observed safety and efficacy of FUS thalamotomy were comparable to those reported in a previous multicenter study with a low SDR, and inclusion of the low SDR group did not affect effectiveness.
几项可行性研究和一项随机、对照、多中心试验已经证明了单侧经颅磁共振引导聚焦超声（FUS）毁损腹侧中间丘脑核治疗原发性震颤的安全性和有效性。
通过一项前瞻性、多中心、单臂确证性试验，评估 FUS 丘脑切开术在日本患者队列中的安全性和疗效。
共 35 例致残性难治性原发性震颤患者接受单侧 FUS 丘脑切开术，并在术后 12 个月进行随访。安全性通过治疗相关不良事件的发生率和严重程度来衡量。疗效通过震颤临床评分量表和原发性震颤问卷评估震颤严重程度和生活质量改善情况。
平均颅骨密度比（SDR）为 0.47。治疗手的姿势性震颤评分从基线到 12 个月时显著下降了 56.4%（95%可信区间：46.7%-66.1%；P<0.001），且在最后一次随访时仍保持。生活质量提高了 46.3%（原发性震颤问卷总评分平均为 17.4[95%可信区间：12.1-22.7]），且无严重不良事件。最常见的不良事件是步态障碍，所有事件均已解决。
单侧 FUS 丘脑切开术可显著且持续缓解震颤，并提高生活质量，具有出色的安全性。观察到的 FUS 丘脑切开术的安全性和疗效与低 SDR 之前的多中心研究报告的结果相当，且纳入低 SDR 组并未影响疗效。
