一项开放标签的试点研究，旨在考察氘代丁苯那嗪在孤立性肌张力障碍中的安全性、耐受性和疗效。
An Open-Label Pilot Study to Examine the Safety, Tolerability and Efficacy of Deutetrabenazine in Isolated Dystonia.
作者信息
Deik Andres, Aamodt Whitley, Cadet Christina, Lasker Aaron, Oliver Alexandria, Spindler Meredith, Tropea Thomas F, Vaswani Pavan, Siderowf Andrew
机构信息
Parkinson Disease and Movement Disorders Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
出版信息
Mov Disord Clin Pract. 2025 Apr;12(4):504-509. doi: 10.1002/mdc3.14327. Epub 2025 Jan 4.
BACKGROUND
Dystonia may respond to VMAT2 inhibition.
OBJECTIVES
Providing pilot data on the safety, tolerability, and efficacy of deutetrabenazine in non dopa-responsive dystonia.
METHODS
Deutetrabenazine was titrated by adults with isolated dystonia. Primary study endpoints included the proportion who maintained the individual, maximum tolerated dose for 6 weeks, and how many titrated to 48 mg/day. Secondary endpoints included rates of QTc prolongation/arrhythmias, suicidality, excessive daytime sleepiness, cognitive decline, and drug-induced parkinsonism. Exploratory endpoints for clinical efficacy were assessed.
RESULTS
Among 15 participants, four (26.7%) withdrew early and six (40%) titrated to 48 mg/day. Common adverse events included fatigue and diarrhea. Secondary safety endpoints did not change significantly, but MDS-UPDRS III scores worsened by ≥3 points in seven participants (46.7%). PGI-C and the blinded CGI-C and GDS improved in three women with blepharospasm.
CONCLUSIONS
Most participants tolerated deutetrabenazine for 6 weeks, and those with blepharospasm may have benefitted from its use.
背景
肌张力障碍可能对VMAT2抑制有反应。
目的
提供关于氘代丁苯那嗪在非多巴胺反应性肌张力障碍中的安全性、耐受性和疗效的初步数据。
方法
成人孤立性肌张力障碍患者滴定使用氘代丁苯那嗪。主要研究终点包括维持个体最大耐受剂量6周的比例，以及滴定至48毫克/天的人数。次要终点包括QTc延长/心律失常、自杀倾向、日间过度嗜睡、认知衰退和药物性帕金森综合征的发生率。评估临床疗效的探索性终点。
结果
15名参与者中，4人（26.7%）提前退出，6人（40%）滴定至48毫克/天。常见不良事件包括疲劳和腹泻。次要安全终点无显著变化，但7名参与者（46.7%）的MDS-UPDRS III评分恶化≥3分。3名眼睑痉挛女性的PGI-C以及盲法CGI-C和GDS有所改善。
结论
大多数参与者耐受氘代丁苯那嗪6周，眼睑痉挛患者可能从使用该药中获益。
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