Dashtipour Khashayar, Wietek Stefan, Rubin Bruce, Maisonobe Pascal, Bahroo Laxman, Trosch Richard
Loma Linda University School of Medicine, Loma Linda, CA 92350 USA.
Ipsen, Cambridge, MA 02142 USA.
J Clin Mov Disord. 2020 Aug 31;7:8. doi: 10.1186/s40734-020-00090-x. eCollection 2020.
Cervical dystonia (CD), the most common focal dystonia, is a chronic neurological movement disorder characterized by sustained involuntary contractions of the neck muscles, leading to abnormal postures. AbobotulinumtoxinA (aboBoNT-A) was approved in the US initially as a 500 U per 1-mL dilution and subsequently, as a 500 U/2-mL dilution (or 250 U/mL), thereby providing clinicians with more flexible dosing options to better meet individual patient needs. The objective of this open-label extension study was to evaluate the longer term safety and efficacy of repeat treatments with aboBoNT-A using 2-mL dilutions in adults with cervical dystonia.
Patients ( = 112) from a 12-week, double-blind lead-in study (NCT01753310) received up to three additional treatments of aboBoNT-A, with re-treatment every 12-16 weeks based on clinical judgment. Safety was assessed through treatment-emergent adverse events (TEAEs). The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total and subscale scores were measured at day 1 of each treatment cycle (C), 4 weeks after each treatment, and 12 weeks after the third treatment. Descriptive statistics were used for all analyses.
In cycles 1, 2, 3, and 4, respectively, 35.7, 25.9, 30.2, and 22.8% of patients reported TEAEs. Dysphagia, muscular weakness, and neck pain were each reported by 10.7% of patients, over the full study duration. Mean TWSTRS total score decreased from 37.7 (SD 13.6 [C1, day 1]) to 30.1 (SD 12.8 [C3, week 12]). In each cycle, TWSTRS total and subscale scores decreased from day 1 to week 4 and increased between weeks 4 and 12, though the week 12 scores remained lower than day 1 scores.
Extended treatment of cervical dystonia with aboBoNT-A (up to 3 additional treatment cycles) using a 2-mL dilution is effective, with a positive risk-benefit profile.
ClinicalTrials.gov Identifier: NCT01753336. Registered 17 Dec 2012.
颈部肌张力障碍（CD）是最常见的局限性肌张力障碍，是一种慢性神经运动障碍，其特征为颈部肌肉持续不自主收缩，导致异常姿势。阿柏西普肉毒素A（aboBoNT - A）最初在美国获批时为每1毫升稀释液含500单位，随后又获批为500单位/2毫升稀释液（即250单位/毫升），从而为临床医生提供了更灵活的给药选择，以更好地满足个体患者需求。这项开放性扩展研究的目的是评估在成年颈部肌张力障碍患者中使用2毫升稀释液重复注射aboBoNT - A的长期安全性和有效性。
来自一项为期12周的双盲导入研究（NCT01753310）的患者（n = 112）接受了多达三次额外的aboBoNT - A治疗，根据临床判断每12 - 16周进行再次治疗。通过治疗中出现的不良事件（TEAE）评估安全性。在每个治疗周期（C）的第1天、每次治疗后4周以及第三次治疗后12周测量多伦多西部痉挛性斜颈评定量表（TWSTRS）总分及各子量表得分。所有分析均采用描述性统计。
在第1、2、3和4个周期中，分别有35.7%、25.9%、30.2%和22.8%的患者报告了TEAE。在整个研究期间，吞咽困难、肌肉无力和颈部疼痛各有10.7%的患者报告。TWSTRS总分均值从37.7（标准差13.6 [C1，第1天]）降至30.1（标准差12.8 [C3，第12周]）。在每个周期中，TWSTRS总分及各子量表得分从第1天到第4周下降，在第4周和第12周之间上升，不过第12周的得分仍低于第1天的得分。
使用2毫升稀释液对颈部肌张力障碍进行aboBoNT - A延长治疗（最多额外进行3个治疗周期）是有效的，且风险效益比良好。
ClinicalTrials.gov标识符：NCT01753336。于2012年12月17日注册。
