A Pilot Study of Focused Ultrasound Thalamotomy for Essential Tremor
A Pilot Study of Focused Ultrasound Thalamotomy for Essential Tremor
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DOI：
10.1056/nejmoa1300962
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发表时间：
2013-08-15
影响因子：
158.5
通讯作者：
Wintermark, Max
中科院分区：
文献类型：
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作者：
Elias, W. Jeffrey;Huss, Diane;Wintermark, Max
背景最近的进展使得高强度聚焦超声能够在磁共振成像（MRI）引导下通过完整的人类颅骨进行递送。本初步研究探讨了使用经颅MRI引导的聚焦超声丘脑切开术治疗原发性tremorth.MethodsFrom 2011年2月至2011年12月，在一项开放标签，非对照研究，我们使用经颅MRI引导的聚焦超声，以针对15例严重，药物难治性原发性震颤患者的丘脑单侧腹侧中间核。我们记录了所有安全性数据，并使用震颤临床评定量表测量震颤抑制的有效性，以计算总分（范围从0到160），手部分项评分（主要结局，范围从0到32）和残疾分项评分（范围从0到32），分数越高表示震颤越严重。我们使用原发性震颤生活质量问卷评估了患者对治疗效果的看法（范围从0到100%，分数越高表明感知的残疾越大）。结果所有患者都发生了丘脑目标的热消融。手术的不良反应包括短暂的感觉、小脑、运动和语言异常，4例患者出现持续性感觉异常。手震颤评分从基线时的20.4改善至12个月时的5.2（P=0.001）。总震颤评分从54.9改善至24.3（P=0.001）。残疾评分从18.2改善至2.8（P=0.001）。生活质量评分从37%提高到11%（P=0.001）。ConclusionsIn this pilot study，MRI引导下聚焦超声丘脑切开术治疗的15例患者的特发性震颤得到改善。需要进行大型的随机对照试验来评估该手术的有效性和安全性。（由聚焦超声外科基金会资助; ClinicalTrials.gov编号，NCT 01304758。）在一项非对照、开放标签的初步研究中，15例接受MRI引导下聚焦超声丘脑切开术治疗的患者的特发性震颤得到改善。不良反应包括4名患者出现持续性感觉异常。这项研究规模太小，无法评估该手术的安全性。特发性震颤是最常见的运动障碍，患病率高达4%，其特征在于激动剂和拮抗剂肌群的节律性振荡，通常在8和12 Hz之间。(1)这种疾病的原因仍然未知，尽管通常存在家族性成分，与编码富含亮氨酸重复序列和含免疫球蛋白结构域的蛋白1（LINGO 1）的基因中的特定多态性有关。虽然特发性震颤在医学上没有危险，但它在家庭和工作场所是渐进的和致残的。(2)震颤的程度并不总是与残疾的严重程度相关，（3）和患者...
BackgroundRecent advances have enabled delivery of high-intensity focused ultrasound through the intact human cranium with magnetic resonance imaging (MRI) guidance. This preliminary study investigates the use of transcranial MRI-guided focused ultrasound thalamotomy for the treatment of essential tremor.MethodsFrom February 2011 through December 2011, in an open-label, uncontrolled study, we used transcranial MRI-guided focused ultrasound to target the unilateral ventral intermediate nucleus of the thalamus in 15 patients with severe, medication-refractory essential tremor. We recorded all safety data and measured the effectiveness of tremor suppression using the Clinical Rating Scale for Tremor to calculate the total score (ranging from 0 to 160), hand subscore (primary outcome, ranging from 0 to 32), and disability subscore (ranging from 0 to 32), with higher scores indicating worse tremor. We assessed the patients' perceptions of treatment efficacy with the Quality of Life in Essential Tremor Questionnaire (ranging from 0 to 100%, with higher scores indicating greater perceived disability).ResultsThermal ablation of the thalamic target occurred in all patients. Adverse effects of the procedure included transient sensory, cerebellar, motor, and speech abnormalities, with persistent paresthesias in four patients. Scores for hand tremor improved from 20.4 at baseline to 5.2 at 12 months (P=0.001). Total tremor scores improved from 54.9 to 24.3 (P=0.001). Disability scores improved from 18.2 to 2.8 (P=0.001). Quality-of-life scores improved from 37% to 11% (P=0.001).ConclusionsIn this pilot study, essential tremor improved in 15 patients treated with MRI-guided focused ultrasound thalamotomy. Large, randomized, controlled trials will be required to assess the procedure's efficacy and safety. (Funded by the Focused Ultrasound Surgery Foundation; ClinicalTrials.gov number, NCT01304758.)In an uncontrolled, open-label pilot study, essential tremor improved in 15 patients treated with MRI-guided focused ultrasound thalamotomy. Adverse effects included persistent paresthesias in four patients. The study was too small to assess the safety of this procedure. Essential tremor, the most common movement disorder, with a prevalence as high as 4%, is characterized by a rhythmic oscillation of agonist and antagonist muscle groups, typically between 8 and 12 Hz.(1) The cause of this disorder remains unknown, although there is often a familial component with a link to a particular polymorphism in the gene encoding leucine-rich repeat and immunoglobulin domain-containing protein 1 (LINGO1). Although essential tremor is not medically dangerous, it is progressive and disabling in the home and workplace.(2) The degree of tremor does not always correlate with the severity of disability,(3) and patients with ...