ABSTRACT

Purpose: This secondary analysis of a randomized, double-blind study plus open-label extension (NCT01249417/NCT01251380) evaluated the efficacy of abobotulinumtoxinA versus placebo in improving gait pattern in children with dynamic equinus due to cerebral palsy (CP) as assessed by the observational gait scale (OGS).

Methods: Ambulatory children with CP (N = 241, aged 2–17) and dynamic equinus were randomized to treatment with abobotulinumtoxinA (10 or 15U/kg/leg) or placebo injected into the gastrocsoleus. All children received abobotulinumtoxinA in the open-label phase.

Results: In the double-blind phase, abobotulinumtoxinA significantly improved OGS total scores versus placebo at Week 4 (treatment effect vs. placebo: 10U/kg/leg: 1.5 [0.7, 2.3], p = .0003; 15U/kg/leg: 1.1 [0.3, 1.9], p = .01). In the open-label phase, treatment with abobotulinumtoxinA continued to improve the OGS score at the same magnitude as seen in the double-blind study.

Conclusion: Repeat treatment with abobotulinumtoxinA improved gait in children with dynamic equinus.

摘要

目的：这项对一项随机，双盲研究加开放标签扩展（NCT01249417 / NCT01251380）的二级分析评估了肉毒杆菌毒素A和安慰剂在改善由脑瘫（CP）引起的动态性马鼻nu的儿童步态模式方面的功效，观察步态量表（OGS）。

方法：将非卧床CP（N = 241，2–17岁）和动态马患儿随机分配至用阿霉素肉毒杆菌毒素A（10或15U / kg /腿）或安慰剂注射至胃底的方法。在开放标签阶段，所有儿童均接受了肉毒杆菌毒素A。

结果：在双盲阶段，与安慰剂相比，第4周，肉毒杆菌毒素A显着改善了OGS总评分（治疗效果与安慰剂的比较：10U / kg /腿：1.5 [ 0.7，2.3 ]，p = .0003； 15U / kg /腿：1.1 [0.3，1.9]，p＝ 0.01）。在开放标签阶段，与双盲研究相同，用肉毒杆菌毒素A治疗继续提高OGS评分。

结论：用肉毒杆菌毒素A反复治疗可改善动态马患儿的步态。