Jinnah H A, Comella Cynthia L, Perlmutter Joel, Lungu Codrin, Hallett Mark
Departments of Neurology, Human Genetics and Pediatrics, Emory University, Atlanta, GA, United States.
Department of Neurology, Rush University Medical Center, Chicago, IL, United States.
Toxicon. 2018 Jun 1;147:89-95. doi: 10.1016/j.toxicon.2017.09.004. Epub 2017 Sep 6.
Numerous studies have established botulinum toxin (BoNT) to be safe and effective for the treatment of cervical dystonia (CD). Despite its well-documented efficacy, there has been growing awareness that a significant proportion of CD patients discontinue therapy. The reasons for discontinuation are only partly understood.
This summary describes longitudinal studies that provided information regarding the proportions of patients discontinuing BoNT therapy, and the reasons for discontinuing therapy. The data come predominantly from un-blinded long-term follow-up studies, registry studies, and patient-based surveys.
All types of longitudinal studies provide strong evidence that BoNT is both safe and effective in the treatment of CD for many years. Overall, approximately one third of CD patients discontinue BoNT. The most common reason for discontinuing therapy is lack of benefit, often described as primary or secondary non-response. The apparent lack of response is only rarely related to true immune-mediated resistance to BoNT. Other reasons for discontinuing include side effects, inconvenience, cost, or other reasons.
Although BoNT is safe and effective in the treatment of the majority of patients with CD, approximately one third discontinue. The increasing awareness of a significant proportion of patients who discontinue should encourage further efforts to optimize administration of BoNT, to improve BoNT preparations to extend duration or reduce side effects, to develop add-on therapies that may mitigate swings in symptom severity, or develop entirely novel treatment approaches.
大量研究已证实肉毒杆菌毒素（BoNT）用于治疗颈部肌张力障碍（CD）安全有效。尽管其疗效有充分记录，但人们越来越意识到相当一部分CD患者会停止治疗。停药原因仅部分得到理解。
本综述描述了纵向研究，这些研究提供了有关停止BoNT治疗的患者比例以及停药原因的信息。数据主要来自非盲法长期随访研究、登记研究和基于患者的调查。
所有类型的纵向研究都提供了有力证据，表明BoNT在治疗CD方面多年来既安全又有效。总体而言，约三分之一的CD患者停止使用BoNT。停药的最常见原因是缺乏疗效，通常被描述为原发性或继发性无反应。明显的无反应很少与对BoNT真正的免疫介导抵抗有关。其他停药原因包括副作用、不便、费用或其他原因。
尽管BoNT在治疗大多数CD患者方面安全有效，但约三分之一的患者会停药。对相当一部分停药患者的认识不断提高，应促使进一步努力优化BoNT的给药方式，改进BoNT制剂以延长作用时间或减少副作用，开发可能减轻症状严重程度波动的附加疗法，或开发全新的治疗方法。
