Steven J Frucht, Mary Catherine George, Alexander Pantelyat, Eckart Altenmueller, Alexandra Nmashie, Jocelyn M Jiao, Michael Chen, David Feng, Susan Shin, Michelle C Kaku, David Simpson
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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.</p><p><strong>Methods: </strong>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.</p><p><strong>Findings: </strong>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.</p><p><strong>Interpretation: </strong>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.</p>","PeriodicalId":23317,"journal":{"name":"Tremor and Other Hyperkinetic Movements","volume":null,"pages":null},"PeriodicalIF":2.5000,"publicationDate":"2024-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11212776/pdf/","citationCount":"0","resultStr":"{\"title\":\"Incobotulinum Toxin-A in Professional Musicians with Focal Task-Specific Dystonia: A Double Blind, Placebo Controlled, Cross-Over Study.\",\"authors\":\"Steven J Frucht, Mary Catherine George, Alexander Pantelyat, Eckart Altenmueller, Alexandra Nmashie, Jocelyn M Jiao, Michael Chen, David Feng, Susan Shin, Michelle C Kaku, David Simpson\",\"doi\":\"10.5334/tohm.903\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Musician's focal task-specific dystonia is a complex disorder of fine motor control, with incomplete understanding of its etiology. 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引用次数: 0
摘要
背景:音乐家局灶性任务特异性肌张力障碍是一种复杂的精细运动控制障碍,其病因尚未完全明了。肉毒素治疗上肢任务特异性肌张力障碍的试验相对较少,之前的研究结果也不尽相同,导致人们对这种方法在精英表演者中的实用性持怀疑态度:我们对 21 名患有影响乐器演奏的局灶性上肢任务特异性肌张力障碍的专业音乐家进行了一项双盲、安慰剂对照、随机、交叉研究。主要研究结果是由两名专家使用临床整体印象数字量表对第8周时的活动臂肌张力障碍评分进行盲法评分,与入组时进行比较:研究结果:19 名男性和 2 名女性音乐家肌张力障碍患者参加了这项研究,为期六年。19名患者完成了研究。与基线相比,主要结果分析显示肌张力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。临床上未观察到明显的乏力,也未发现毒素中和抗体:尽管样本量较小,但我们的研究表明,注射伊可丁毒素 A 治疗音乐家的任务特异性肌张力障碍在统计学上有显著疗效。通过加强注射量身定制的毒素使用方法,使剂量策略和疗效得到了完善,患者从中获得的益处在录像评估中清晰可见。除了应用于音乐家的肌张力障碍外,这种方法还可用于优化肉毒杆菌毒素在其他形式的局灶性肌张力障碍(如眼睑痉挛、颈肌张力障碍、作家痉挛和痉挛性发音障碍)中的应用。
Incobotulinum Toxin-A in Professional Musicians with Focal Task-Specific Dystonia: A Double Blind, Placebo Controlled, Cross-Over Study.
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.