UTILITY AND PHYSIOLOGY OF BOTULINUM TOXIN FOR INVOLUNTARY MOVEMENT DISORDERS

肉毒杆菌毒素治疗不自主运动障碍的效用和生理学

• 批准号: 3760267
• 负责人: M HALLETT
• 金额: --
• 依托单位: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3760267
• 关键词: abnormal involuntary movement; botulinum toxins; clinical trials; human subject; human therapy evaluation; longitudinal human study; muscle cramp; neuromuscular disorder chemotherapy; neuromuscular junction; physiology; torticollis; tremor

We have been studying the efficacy of local injections of botulinum toxin for the treatment of different types of focal dystonias. Botulinum toxin injected in small doses directly into muscle, binds to and inactivates the neuromuscular junction. Dystonic contraction is decreased and function improves Treatment is well-tolerated with minimal side effects. We have also used botulinum toxin to study the physiology of focal dystonias. A double-blind trial of botulinum toxin for hand dystonia (e.g. writer's cramp and musician's cramp) showed a significant improvement in function following injection of active botulinum toxin compared to placebo. Open-label studies have shown that 84% of patients have at least short- term benefit. Longer follow-up shows that approximately 50% of patients have persistent benefit. Women with nonlocalized symptoms or dystonic cramp who require less frequent injections were especially likely to continue treatment. Botulinum toxin injection is well-tolerated. Botulinum toxin injections have been similarly effective in our patients with other types of dystonia. Twelve patients with tremor have been treated to date. All of the patients with torticollis and head tremor who received at least 3 injections had at least mild improvement. 50% of the patients with arm tremor had substantial improvement. One patient noted increased tremor after injection. We are conducting a phase I-II trial of botulinum toxin type F to see if this will benefit patients who have lost response to type A. Type F appears to have similar efficacy and side effects to type A, although the duration of action is slightly less.

我们一直在研究局部注射肉毒杆菌毒素的疗效 用于治疗不同类型的局灶性肌张力障碍。肉毒杆菌毒素 直接将小剂量注射到肌肉中，结合并灭活 神经肌肉接头。肌张力障碍收缩减少， 功能改善，治疗耐受性好，副作用最小。 我们还用肉毒杆菌毒素研究了病灶的生理学。 声门困难。肉毒杆菌毒素治疗手部肌张力障碍的双盲试验 (如作家抽筋和音乐家抽筋)表现出显著的 注射活性肉毒毒素后功能的改善 与安慰剂相比。开放标签研究表明，84%的患者 至少有短期的好处。更长时间的跟踪显示， 大约50%的患者持续受益。女性与 不需要经常出现的非局限性症状或肌张力障碍抽筋 注射尤其有可能继续治疗。肉毒杆菌毒素 注射耐受性良好。肉毒杆菌毒素注射已经被 对其他类型的肌张力障碍患者也同样有效。十二 到目前为止，震颤的患者已经得到了治疗。所有的病人 有斜颈和头部震颤的人接受了至少3次注射 至少是轻微的改善。50%的手臂震颤患者有 有实质性的改善。一名患者注意到术后震颤增加 注射。我们正在进行肉毒杆菌毒素类型的I-II期试验 F，看看这是否会使对A型病毒失去反应的患者受益。 F型似乎与A型有相似的疗效和副作用， 尽管行动的持续时间略短。