Beaumont Health, Oakland University School of Medicine, Grosse Pointe, Michigan.
Mazovian Neuropsychiatry Center in Zagorze, Wiazowna, Poland.
Pediatr Neurol. 2018 May;82:44-49. doi: 10.1016/j.pediatrneurol.2017.12.013. Epub 2018 Feb 7.
The effects of botulinum toxin are transient, and repeat injections are required in children with lower-limb spasticity. However, the efficacy of botulinum toxin in patients who have received previous injections has remained largely unexplored.
We present subgroup analyses of a phase III study conducted in ambulatory children (aged two to 17) with spastic equinus foot. Patients were randomized to single doses of abobotulinumtoxinA 10 U/kg/leg, 15 U/kg/leg, or placebo injected into the gastrocnemius-soleus complex (one or both legs). The first analysis was prespecified to review the effect of abobotulinumtoxinA in children previously treated with botulinum toxin versus those children new to the treatment; a second post hoc analysis evaluated the effect of abobotulinumtoxinA in children who changed botulinum toxin formulation.
Of the 241 randomized patients, 113 had previously received botulinum toxin, including 86 who had been treated with another formulation. In both analyses, muscle tone (Modified Ashworth Scale) and the Physicians Global Assessment, at week 4, improved with abobotulinumtoxinA treatment versus placebo, regardless of baseline botulinum toxin status. Placebo responses in patients new to treatment were consistently higher than in the previously treated group.
These results demonstrate similar abobotulinumtoxinA efficacy and safety profiles in children with spasticity who are new to botulinum toxin treatment and those children who were previously treated. The efficacy and safety of abobotulinumtoxinA treatment in these previously treated patients were comparable with the overall trial population, indicating that doses of 10 and 15 U/kg/leg are suitable starting doses for children with spasticity regardless of the previous botulinum toxin preparation used.
肉毒毒素的作用是短暂的，下肢痉挛的儿童需要重复注射。然而，先前接受过注射的患者中肉毒毒素的疗效在很大程度上仍未得到探索。
我们报告了一项针对门诊儿童（年龄 2 至 17 岁）痉挛性马蹄内翻足的 III 期研究的亚组分析。患者被随机分配接受单剂量的 10 U/kg/腿、15 U/kg/腿或安慰剂注射到腓肠肌-比目鱼肌复合体（一条或两条腿）。第一次分析是预先指定的，旨在回顾先前接受过肉毒毒素治疗与初次接受治疗的儿童的 abobotulinumtoxinA 效果；第二次事后分析评估了 abobotulinumtoxinA 在改变肉毒毒素配方的儿童中的效果。
在 241 名随机患者中，有 113 名之前接受过肉毒毒素治疗，包括 86 名接受过另一种制剂治疗的患者。在这两种分析中，与安慰剂相比，第 4 周时 abobotulinumtoxinA 治疗可改善肌肉张力（改良 Ashworth 量表）和医生整体评估，无论基线肉毒毒素状况如何。初次接受治疗的患者的安慰剂反应始终高于先前接受治疗的组。
这些结果表明，对于初次接受肉毒毒素治疗的痉挛性儿童和先前接受治疗的儿童，abobotulinumtoxinA 的疗效和安全性相似。这些先前接受治疗的患者的 abobotulinumtoxinA 治疗的疗效和安全性与总体试验人群相当，表明 10 和 15 U/kg/腿的剂量适用于无论先前使用何种肉毒毒素制剂的痉挛性儿童。
