Nagaratnam Sai A, Wilson Duncan, Chiang Han-Lin, Chang Florence C F, Qiu Jessica, Silsby Matthew, Fois Alessandro F, Martin Andrew, Mahant Neil, Fung Victor S C, Morales-Briceno Hugo
Movement Disorders Unit, Department of Neurology, Westmead Hospital, Westmead, New South Wales, Australia.
Division of General Neurology, Neurological Institute, Taipei Veterans General Hospital, Taipei, Taiwan.
Mov Disord Clin Pract. 2025 Apr 25. doi: 10.1002/mdc3.70079.
Proximal upper limb tremor is a common, functionally disabling condition. Medical therapy is often ineffective. IncobotulinumtoxinA can significantly improve tremor with minimal adverse effects.
We aimed to compare clinical, functional, and quality of life measures in patients with proximal upper limb tremor following incobotulinumtoxinA treatment.
We undertook a double-blind, randomized two-sequence, two-period, two-treatment, placebo-controlled cross-over trial. Twenty adult patients with proximal upper limb tremor were recruited from a tertiary movement disorders clinic. Patients were randomized 1:1 to alternate electromyographically guided intramuscular injections of either incobotulinumtoxinA or saline to the dominant upper limb using computer-generated randomization. Patients were injected at baseline, then 4 months after cross-over. Primary outcome was change in Goal Attainment Rating Scale (GARS) at two-months post intervention. Secondary outcomes were dominant upper limb The Essential Tremor Rating Assessment Scale (TETRAS), Fahn-Tolosa-Marin tremor rating scale (FTM-TRS) and three patient-related outcome measures. Outcomes were assessed at baseline and monthly intervals for 3-months post each intervention.
There was no significant improvement in the GARS at 2-months post injection (OR 1.83, 95% CI 0.14-24.32). Secondary outcomes showed a mean reduction in TETRAS score of -1.05 (95% CI -1.99 to -0.13) and FTM-TRS of -6.84 (95% CI -12.37 to -1.31) in those receiving incobotulinumtoxinA. There was no significant change in patient-rated outcome scores.
Single dose incobotulinumtoxinA injections are effective in reducing objective measures of proximal upper limb tremor and function. This did not translate into improved patient-rated outcomes in this single therapeutic dose study.
近端上肢震颤是一种常见的、导致功能障碍的疾病。药物治疗往往无效。因卡泊肉毒素A能显著改善震颤，且副作用极小。
我们旨在比较因卡泊肉毒素A治疗后近端上肢震颤患者的临床、功能和生活质量指标。
我们进行了一项双盲、随机、双序列、双周期、双治疗、安慰剂对照的交叉试验。从一家三级运动障碍诊所招募了20名患有近端上肢震颤的成年患者。使用计算机生成的随机化方法，将患者按1:1随机分组，交替接受肌电图引导下向优势上肢注射因卡泊肉毒素A或生理盐水。患者在基线时注射，然后在交叉后4个月注射。主要结局是干预后两个月目标达成评定量表（GARS）的变化。次要结局是优势上肢的原发性震颤评定量表（TETRAS）、法恩-托洛萨-马林震颤评定量表（FTM-TRS）以及三项患者相关结局指标。在每次干预后的基线及之后3个月内每月评估一次结局。
注射后2个月，GARS无显著改善（比值比1.83，95%置信区间0.14 - 24.32）。次要结局显示，接受因卡泊肉毒素A治疗的患者TETRAS评分平均降低-1.05（95%置信区间-1.99至-0.13），FTM-TRS评分降低-6.84（95%置信区间-12.37至-1.31）。患者自评结局评分无显著变化。
单剂量注射因卡泊肉毒素A可有效降低近端上肢震颤和功能的客观指标。在这项单治疗剂量研究中，这并未转化为患者自评结局的改善。
