Efficacy and safety of a standardised 500 unit dose of Dysport® (Clostridium botulinum toxin type A haemaglutinin complex) in a heterogeneous cervical dystonia population: results of a prospective, multicentre, randomised, double-blind, placebo-controlled
Efficacy and safety of a standardised 500 unit dose of Dysport® (Clostridium botulinum toxin type A haemaglutinin complex) in a heterogeneous cervical dystonia population: results of a prospective, multicentre, randomised, double-blind, placebo-controlled
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DOI：
10.1007/s004150170028
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发表时间：
2001-12
影响因子：
6
通讯作者：
J. Wissel;P. Kaňovský;E. Růžička;Martin Bareš;H. Hortová;H. Streitová;R. Jech;Jan Roth;
中科院分区：
文献类型：
--
作者：
J. Wissel;P. Kaňovský;E. Růžička;Martin Bareš;H. Hortová;H. Streitová;R. Jech;Jan Roth;
在一组选定的原发性旋转性颈肌张力障碍（CD）患者中进行的剂量范围研究结果表明，500单位的Dysport（A型肉毒梭菌毒素血凝素复合物）是最佳起始剂量。本研究旨在证实该剂量在更能代表典型肌张力障碍临床的CD患者人群中的疗效和安全性特征。共有68例中重度CD患者（Tsui评分≥ 9）被随机分配接受安慰剂或Dysport 500单位。根据头部偏斜的临床模式，使用标准化注射方案进行治疗。共有21名患者（11名Dysport，10名安慰剂）以前没有接受过A型肉毒杆菌毒素（BtxA）注射，47名患者（24名Dysport，23名安慰剂）接受BtxA超过12周。在基线和第4、8和16周进行评估。在第4周被定义为无应答者的患者在第6周接受500单位Dysport的开放期重新治疗，并在第10周进行随访。在第4周（p=0.001）和第8周（p=0.002），Tsui评分存在显著的组间差异。同样，在第4周和第8周，患者和研究者对反应的评估存在显著的组间差异（p < 0.001），有利于Dysport。此外，在第4周时，与安慰剂组（33%）相比，更多的Dysport（49%）患者无疼痛（p=0.02）。总体而言，30/35例（86%）Dysport患者和14/33例（42%）安慰剂患者在第4周被归类为应答者。15/35例Dysport患者和9/33例安慰剂患者报告了不良事件。开放阶段治疗改善了Tsui（p < 0.001）和疼痛评分（p=0.011），并且23/24名患者被归类为应答者。尽管个体剂量滴定和肌肉选择是可取的，这项研究表明，在没有肌电图指导的情况下，将500单位的Dysport注射到临床确定的颈部肌肉中，对治疗患有以下疾病的患者是安全有效的：颈肌张力障碍的主要临床类型。
Results from a dose-ranging study in a selected group of de novo patients with rotational cervical dystonia (CD) suggest that 500 units of Dysport (Clostridium botulinumtoxin type A haemaglutinin complex) is the optimal starting dose. The present study aimed to confirm the efficacy and safety profile of this dose in a population of CD patients more representative of those seen in a typical dystonia clinic.A total of 68 patients with moderate to severe CD (Tsui score ≥ 9) were randomly assigned to receive placebo or Dysport 500 units. Treatment was administered according to the clinical pattern of head deviation, using a standardised injection protocol. A total of 21 patients (11 Dysport, 10 placebo) had not previously received botulinum toxin type A (BtxA) injections, and 47 patients (24 Dysport, 23 placebo) had received BtxA more than 12 weeks previously. Assessments were performed at baseline and weeks 4, 8 and 16. Patients defined as non-responders at week 4 were re-treated in an open phase with 500 units of Dysport at week 6, and were followed up at week 10.Significant between-group differences in Tsui scores were present at weeks 4 (p=0.001) and 8 (p=0.002). Similarly, there were significant between-group differences (p < 0.001) in patient and investigator assessments of response in favour of Dysport at weeks 4 and 8. Also, more Dysport (49 %) than placebo (33 %) patients were pain-free at week 4 (p=0.02). Overall, 30/35 (86 %) Dysport patients and 14/33 (42 %) placebo patients were classified as responders at week 4. Adverse events were reported by 15/35 Dysport patients and 9/33 placebo patients. Open phase treatment produced improvements in Tsui (p < 0.001) and pain scores (p=0.011), and 23/24 patients were classified as responders.Although individual dose titration and muscle selection is desirable, this study demonstrated that a dose of 500 units of Dysport injected into clinically identified neck muscles without electromyographic guidance is safe and effective in the treatment of patients with the major clinical types of cervical dystonia.