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阿柏西普肉毒毒素A神经毒素治疗颈部肌张力障碍的多中心观察性研究:ANCHOR-CD注册研究

Multicenter observational study of abobotulinumtoxinA neurotoxin in cervical dystonia: The ANCHOR-CD registry.

作者信息

Trosch Richard M, Espay Alberto J, Truong Daniel, Gil Ramon, Singer Carlos, LeWitt Peter A, Lew Mark F, Tagliati Michele, Adler Charles H, Chen Jack J, Marchese Dominic, Comella Cynthia L

机构信息

Oakland University William Beaumont School of Medicine, 32255 Northwestern Highway, Suite 40, Farmington Hills, MI 48334, United States.

Department of Neurology, UC Neuroscience Institute, University of Cincinnati, 260 Stetson Street, Cincinnati, OH 45267, United States.

出版信息

J Neurol Sci. 2017 May 15;376:84-90. doi: 10.1016/j.jns.2017.02.042. Epub 2017 Feb 21.

Abstract

BACKGROUND

The ANCHOR-CD prospective observational registry study evaluated the effectiveness of abobotulinumtoxinA in adult idiopathic cervical dystonia (CD) in clinical practice.

METHODS

Adults with CD were eligible. Treating physicians determined abobotulinumtoxinA dose and treatment interval. The primary endpoint was patient response rate (Toronto Western Spasmodic Torticollis Rating Scale [TWSTRS] score reduction≥25% and Patient Global Impression of Change [PGIC] score of +2 or +3 at Week 4 of Cycle 1).

RESULTS

350 patients enrolled (75% women; mean age 59±13.6years; 27.4% botulinum neurotoxin-naive) and 347 received at least 1 treatment. The median abobotulinumtoxinA dose for Cycle 1 was 500 Units. At Week 4, the responder rate was 30.6% (n=304) and the TWSTRS total score decreased 27.4% from baseline. PGIC of at least "Much improved" was documented in 43.6% of patients and maintained in Cycles 2 through 4 (43.3%, 48.9%, and 52.8%, respectively). A total of 39 adverse events (31 study drug-related) were reported in 17 patients (5%); the most common were dysphagia (n=6), muscle weakness (n=4), and neck pain (n=3).

CONCLUSION

This study confirmed the beneficial effect of abobotulinumtoxinA on CD in routine clinical practice as measured by improvements in TWSTRS and PGIC. No new safety concerns were identified.

摘要

背景

ANCHOR-CD前瞻性观察性注册研究评估了阿柏毒素A在临床实践中治疗成人特发性颈部肌张力障碍(CD)的有效性。

方法

符合条件的为患有CD的成年人。治疗医生确定阿柏毒素A的剂量和治疗间隔。主要终点是患者反应率(在第1周期第4周时,多伦多西部痉挛性斜颈评定量表 [TWSTRS] 评分降低≥25% 且患者整体印象变化 [PGIC] 评分为+2或+3)。

结果

350名患者入组(75% 为女性;平均年龄59±13.6岁;27.4% 未使用过肉毒杆菌神经毒素),347名患者接受了至少1次治疗。第1周期阿柏毒素A的中位剂量为500单位。在第4周时,反应率为30.6%(n = 304),TWSTRS总分较基线下降了27.4%。43.6% 的患者记录到PGIC至少为“明显改善”,并在第2至4周期维持(分别为43.3%、48.9% 和52.8%)。17名患者(5%)报告了共39起不良事件(31起与研究药物相关);最常见的是吞咽困难(n = 6)、肌肉无力(n = 4)和颈部疼痛(n = 3)。

结论

本研究证实了阿柏毒素A在常规临床实践中对CD的有益作用,这通过TWSTRS和PGIC的改善得以衡量。未发现新的安全问题。

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