Longitudinal studies of botulinum toxin in cervical dystonia: Why do patients discontinue therapy?
Longitudinal studies of botulinum toxin in cervical dystonia: Why do patients discontinue therapy?
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DOI：
10.1016/j.toxicon.2017.09.004
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发表时间：
2018-06-01
期刊：
影响因子：
2.8
通讯作者：
Hallett, Mark
中科院分区：
文献类型：
--
作者：
Jinnah, H. A.;Comella, Cynthia L.;Hallett, Mark
背景：大量研究已证实肉毒毒素（BoNT）对于治疗颈肌张力障碍（CD）是安全有效的。尽管其疗效已有充分记录，但人们越来越认识到很大一部分克罗恩病患者会停止治疗。停止治疗的原因仅得到部分了解。方法：本摘要描述了纵向研究，这些研究提供了有关停止 BoNT 治疗的患者比例以及停止治疗的原因的信息。数据主要来自非盲法长期随访研究、登记研究和以患者为基础的调查。结果：所有类型的纵向研究都提供了强有力的证据，证明多年来 BoNT 治疗 CD 既安全又有效。总体而言，大约三分之一的 CD 患者停止 BoNT。停止治疗的最常见原因是缺乏益处，通常被描述为原发性或继发性无反应。明显缺乏反应很少与真正的免疫介导的 BoNT 耐药性相关。停止的其他原因包括副作用、不便、成本或其他原因。讨论：尽管 BoNT 对大多数 CD 患者的治疗是安全有效的，但大约三分之一的患者停止治疗。很大一部分停药患者的认识不断提高，应鼓励进一步努力优化 BoNT 的给药，改进 BoNT 制剂以延长持续时间或减少副作用，开发可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。 (C) 2017 Elsevier Ltd. 保留所有权利。
Background: 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.Methods: 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.Results: 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.Discussion: 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. (C) 2017 Elsevier Ltd. All rights reserved.