Bilateral Focused Ultrasound Thalamotomy for Essential Tremor (BEST-FUS Phase 2 Trial)
Bilateral Focused Ultrasound Thalamotomy for Essential Tremor (BEST-FUS Phase 2 Trial)
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DOI：
10.1002/mds.28716
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发表时间：
2021-07-20
影响因子：
8.6
通讯作者：
Lozano, Andres M.
中科院分区：
文献类型：
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作者：
Iorio-Morin, Christian;Yamamoto, Kazuaki;Lozano, Andres M.
背景：对于难治性原发性震颤患者，单侧磁共振引导聚焦超声丘脑切开术可改善对侧震颤。然而，这种方法不能解决同侧症状。目的本研究的目的是确定双侧丘脑切开术是否可以在受益大于不良反应的情况下进行。方法我们对特发性震颤患者进行了一项前瞻性、单臂、单盲的第二阶段试验，即第二侧磁共振引导聚焦超声丘脑切开术。患者随访3个月。主要结果是相对于基线的生活质量的变化，以及对问题的回答“鉴于你现在所知道的，你会再次治疗第二侧吗？”".次要结局包括震颤、步态、言语和不良反应。结果分析了10例患者。该研究满足两个主要结局，干预导致3个月时生活质量的临床显著改善（原发性震颤生活质量评分差异平均值，19.7; 95%CI，8.0-31.4; P = 0.004），所有患者报告他们将选择再次接受第二方治疗。所有患者的震颤均显著改善。7例发生轻度不良反应，包括2例一过性步态障碍和跌倒，1例构音障碍和吞咽困难，1例轻度吞咽困难持续3个月。结论分期双侧磁共振引导下聚焦超声丘脑核切开术与单侧丘脑核切开术安全性相似，可改善特发性震颤患者的震颤和生活质量。需要在III期试验中进行更长期的随访和持续的累积，以验证这些结果。(c)2021国际帕金森和运动障碍协会
Background In patients with medically refractory essential tremor, unilateral magnetic resonance-guided focused ultrasound thalamotomy can improve contralateral tremor. However, this procedure does not address ipsilateral symptoms. Objective The objective of the current study was to determine whether bilateral thalamotomies can be performed with an acceptable safety profile where benefits outweigh adverse effects. Methods We conducted a prospective, single-arm, single-blinded phase 2 trial of second-side magnetic resonance-guided focused ultrasound thalamotomy in patients with essential tremor. Patients were followed for 3 months. The primary outcome was the change in quality of life relative to baseline, as well as the answer to the question "Given what you know now, would you treat the second side again?". Secondary outcomes included tremor, gait, speech, and adverse effects. Results Ten patients were analyzed. The study met both primary outcomes, with the intervention resulting in clinically significant improvement in quality of life at 3 months (mean Quality of Life in Essential Tremor score difference, 19.7; 95%CI, 8.0-31.4; P = 0.004) and all patients reporting that they would elect to receive the second-side treatment again. Tremor significantly improved in all patients. Seven experienced mild adverse effects, including 2 with transient gait impairment and a fall, 1 with dysarthria and dysphagia, and 1 with mild dysphagia persisting at 3 months. Conclusions Staged bilateral magnetic resonance-guided focused ultrasound thalamotomy can be performed with a reasonable safety profile similar to that seen with unilateral thalamotomy and improves the tremor and quality of life of patients with essential tremor. Longer-term follow-up and continued accrual in the phase 3 trial will be required to validate these findings. (c) 2021 International Parkinson and Movement Disorder Society