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伴前屈和后屈姿势的痉挛性斜颈患者接受肉毒毒素 A 治疗的反应。

Treatment response to onabotulinumtoxinA in cervical dystonia patients with anterocollis and retrocollis.

机构信息

Division of Movement Disorders, Department of Neurology/Movement Disorders, Loma Linda University School of Medicine, Faculty Medical Offices, 11370 Anderson, Suite B-100, Loma Linda, CA, 92354, USA.

Allergan, an AbbVie Company, 2525 Dupont Drive, Irvine, CA, 92612, USA.

出版信息

Toxicon. 2024 Sep;248:108035. doi: 10.1016/j.toxicon.2024.108035. Epub 2024 Jul 24.

Abstract

Anterocollis (AC) and retrocollis (RC) are less common cervical dystonia (CD) subtypes that are often under-represented in CD clinical trials. Herein we describe real-world demographics, disease characteristics, and treatment response to onabotulinumtoxinA (onabotA) in AC or RC patients from an observational, multicenter, prospective registry, CD PROBE. After three onabotA treatments, outcomes (CDIP-58, PGIC, CGIC, CD severity, TWSTRS) in patients with predominant AC or RC were compared to torticollis (TC) and all CD subtypes combined. The mean dosages at each treatment ranged from 153.5 to 195.4 U (AC) to 184.0-213.4 U (RC). After treatment, AC and RC patients reported improvements in the CDIP-58. "Much" or "very much improved" on PGIC and CGIC was reported by AC patients (n = 11/23, 48%) and clinicians (n = 14/23, 61%); and by RC patients (n = 14/24, 58%) and clinicians (n = 19/24, 83%). The mean total TWSTRS decreased from 45.7 (n = 59) to 36.1 (n = 23, 21.0% improvement) for AC patients and from 40.1 (n = 55) to 31.6 (n = 23, 21.2% improvement) for RC patients; the proportion of AC and RC patients with severe CD decreased. Outcomes for AC and RC were generally consistent with those for TC and all subtypes combined. Dysphagia was reported in 4/59 (6.8%) of AC patients (one serious), 7/55 (12.7%) of RC patients (none serious), 29/494 (5.9%) of TC patients (none serious), and 64/1012 (6.3%) of all CD patients (two serious). No new safety signals were identified. In conclusion, treatment with onabotA may relieve CD symptoms in some patients with AC and RC, consistent with results for other CD subtypes and the known safety profile of onabotA for the treatment of CD.

摘要

前屈性斜颈(AC)和后屈性斜颈(RC)是较少见的颈部肌张力障碍(CD)亚型,在 CD 临床试验中常代表性不足。在此,我们描述了来自观察性、多中心、前瞻性登记研究 CD PROBE 的真实世界人口统计学、疾病特征以及对 AC 或 RC 患者使用肉毒毒素 A(onabotA)的治疗反应。在接受三次 onabotA 治疗后,与扭转痉挛(TC)和所有 CD 亚型相比,以 AC 或 RC 为主的患者的结局(CDIP-58、PGIC、CGIC、CD 严重程度、TWSTRS)。每次治疗的平均剂量范围为 153.5 至 195.4 U(AC)至 184.0 至 213.4 U(RC)。治疗后,AC 和 RC 患者报告 CDIP-58 有所改善。AC 患者(n=23,48%)和临床医生(n=23,61%)报告“明显”或“非常明显”改善的 PGIC 和 CGIC;RC 患者(n=24,58%)和临床医生(n=24,83%)报告改善。AC 患者的平均总 TWSTRS 从 45.7(n=59)降至 36.1(n=23,21.0%改善),RC 患者从 40.1(n=55)降至 31.6(n=23,21.2%改善);CD 严重程度的 AC 和 RC 患者比例降低。AC 和 RC 的结局通常与 TC 和所有亚型一致。59 例 AC 患者中有 4 例(6.8%)报告吞咽困难(1 例严重),55 例 RC 患者中有 7 例(12.7%)报告吞咽困难(无严重病例),494 例 TC 患者中有 29 例(5.9%)报告吞咽困难(无严重病例),1012 例所有 CD 患者中有 64 例(6.3%)报告吞咽困难(2 例严重)。没有发现新的安全信号。总之,肉毒毒素 A 的治疗可能会缓解一些 AC 和 RC 患者的 CD 症状,与其他 CD 亚型的结果以及肉毒毒素 A 治疗 CD 的已知安全性一致。

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