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肉毒梭菌毒素-A 治疗局灶性任务特异性肌张力障碍职业音乐家的双盲、安慰剂对照、交叉研究。

Incobotulinum Toxin-A in Professional Musicians with Focal Task-Specific Dystonia: A Double Blind, Placebo Controlled, Cross-Over Study.

机构信息

New York University Grossman School of Medicine, US.

Icahn School of Medicine at Mount Sinai, US.

出版信息

Tremor Other Hyperkinet Mov (N Y). 2024 Jun 27;14:32. doi: 10.5334/tohm.903. eCollection 2024.

Abstract

BACKGROUND

Musician's focal task-specific dystonia is a complex disorder of fine motor control, with incomplete understanding of its etiology. There have been relatively few trials of botulinum toxin in upper limb task-specific dystonia, and prior studies have yielded variable results, leading to skepticism regarding the utility of this approach in elite performers.

METHODS

We conducted a double-blind, placebo-controlled, randomized, cross-over study of incobotulinum toxin-A in 21 professional musicians with focal upper extremity task-specific dystonia affecting performance on their instrument, using a novel paradigm of initial injections followed by booster injections at two- and four-week intervals. The primary outcome measure was the change in blinded dystonia rating of the active arm by two expert raters using a Clinical Global Impression numeric scale at week 8 compared to enrollment.

FINDINGS

19 men and 2 women with musicians' dystonia were enrolled over a six-year period. Nineteen patients completed the study. Analysis of the primary outcome measure in comparison to baseline revealed a change in dystonia severity of P = 0.04 and an improvement in overall musical performance of P = 0.027. No clinically significant weakness was observed, and neutralizing antibodies to toxin were not found.

INTERPRETATION

Despite its small sample size, our study demonstrated a statistically significant benefit of incobotulinum toxin-A injections as a treatment for musicians' task-specific dystonia. Tailoring the use of toxin with booster injections allowed refinement of dosing strategy and outcomes, with benefits that were meaningful to patients clearly visible on videotaped evaluations. In addition to its application to musicians' dystonia, this approach may have relevance to optimize application of botulinum toxin in other forms of focal dystonia such as blepharospasm, cervical dystonia, writer's cramp, and spasmodic dysphonia.

摘要

背景

音乐家特发性局限性运动障碍是一种精细运动控制的复杂障碍,其病因尚未完全了解。针对上肢任务特异性运动障碍,肉毒毒素的试验相对较少,且先前的研究结果存在差异,这导致人们对这种方法在精英演奏者中的应用效果产生了怀疑。

方法

我们对 21 名患有影响乐器演奏的上肢局限性运动障碍的专业音乐家进行了一项英夫利西单抗 A 的双盲、安慰剂对照、随机、交叉研究,采用新的初始注射方案,随后在 2 周和 4 周间隔进行增强注射。主要结局指标是在第 8 周与入组时相比,由两名专家评估者使用临床总体印象数字量表对主动臂的盲法运动障碍评分的变化。

结果

在 6 年期间,共有 19 名男性和 2 名女性患有音乐家的运动障碍患者入组。19 名患者完成了研究。与基线相比,对主要结局指标的分析显示,运动障碍严重程度的变化为 P = 0.04,整体音乐表现的改善为 P = 0.027。未观察到明显的临床肌无力,也未发现针对毒素的中和抗体。

解释

尽管样本量较小,但我们的研究表明英夫利西单抗 A 注射在治疗音乐家特发性局限性运动障碍方面具有统计学意义的益处。使用增强注射来调整毒素的使用,可以细化剂量策略和结果,通过视频评估可以明显看到对患者有意义的益处。除了在音乐家的运动障碍中的应用外,这种方法可能与优化肉毒毒素在其他形式的局限性运动障碍中的应用有关,如眼睑痉挛、颈性运动障碍、书写痉挛和痉挛性发音障碍。

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