Allergan, an AbbVie company, Irvine, CA, USA.
Gillette Children's Specialty Healthcare, St Paul, MN, USA.
NeuroRehabilitation. 2021;49(3):469-479. doi: 10.3233/NRE-210071.
This is the first large study of onabotulinumtoxinA as treatment for pediatric upper limb spasticity.
Evaluate efficacy and safety of a single treatment with onabotulinumtoxinA plus occupational therapy (OT).
In this registrational phase III, multinational study (NCT01603602), participants were randomized 1:1:1 to onabotulinumtoxinA 3 U/kg/OT, 6 U/kg/OT, or placebo/OT. Primary endpoint was average change from baseline at weeks 4 and 6 in Modified Ashworth Scale-Bohannon (MAS) score. Secondary endpoints included Modified Tardieu Scale (MTS), Clinical Global Impression of Change (CGI) and functional Goal Attainment Scale (GAS).
235 participants were randomized. At weeks 4 and 6, onabotulinumtoxinA groups had greater mean reductions in MAS (both -1.9; p < 0.001) versus placebo (-1.2). OnabotulinumtoxinA doses improved dynamic tone per MTS. Mean CGI at weeks 4 and 6 was unchanged in the overall population, but improved in a post hoc analysis of patients with a single affected upper limb (UL) muscle group (elbow or wrist). GAS score for passive goals was significantly higher for 6 U/kg versus placebo at week 12. Most AEs were mild/moderate in severity; overall incidence was similar between groups.
OnabotulinumtoxinA (3 and 6 U/kg) was safe and effective in reducing upper limb spasticity in pediatric participants.
这是首个关于肉毒毒素 A 治疗儿科上肢痉挛的大型研究。
评估单次肉毒毒素 A 联合作业疗法（OT）治疗的疗效和安全性。
在这项注册的 III 期、多中心研究（NCT01603602）中，参与者按 1:1:1 的比例随机分配至肉毒毒素 A 3U/kg/OT、6U/kg/OT 或安慰剂/OT 组。主要终点是治疗 4 周和 6 周时改良 Ashworth 量表-博汉农评分（MAS）的平均变化。次要终点包括改良 Tardieu 量表（MTS）、临床总体印象变化量表（CGI）和功能目标实现量表（GAS）。
235 名参与者被随机分配。在治疗 4 周和 6 周时，肉毒毒素 A 组的 MAS 评分（均为-1.9；p<0.001）比安慰剂组（-1.2）的降幅更大。肉毒毒素 A 剂量改善了 MTS 下的动态肌张力。在总体人群中，治疗 4 周和 6 周时 CGI 评分没有变化，但在分析单上肢（UL）肌肉群（肘部或腕部）受累的患者时，CGI 评分得到改善。在第 12 周，6U/kg 组的被动目标 GAS 评分明显高于安慰剂组。大多数不良事件的严重程度为轻度/中度；各组的总体发生率相似。
肉毒毒素 A（3 和 6U/kg）在儿科参与者中安全且有效，可降低上肢痉挛程度。
