A Pilot Study of Focused Ultrasound Thalamotomy for Essential Tremor

作者：Elias, W. Jeffrey^*; Huss, Diane; Voss, Tiffini; Loomba, Johanna; Khaled, Mohamad; Zadicario, Eyal; Frysinger, Robert C.; Sperling, Scott A.; Wylie, Scott; Monteith, Stephen J.; Druzgal, Jason; Shah, Binit B.; Harrison, Madaline; Wintermark, Max

来源：New England Journal of Medicine, 2013, 369(7): 640-648.

DOI：10.1056/NEJMoa1300962

• GAMMA-KNIFE THALAMOTOMY
• QUALITY STANDARDS SUBCOMMITTEE
• DEEP BRAIN-STIMULATION
• THALAMIC-STIMULATION
• AMERICAN-ACADEMY
• PHASED-ARRAY
• OF-LIFE
• SURGERY
• SKULL
• DISABILITY

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 ...

• 出版日期2013-8-15