Tilton Ann, Russman Barry, Aydin Resa, Dincer Umit, Escobar Raul G, Kutlay Sehim, Lipczyk Zbigniew, Velez Juan Carlos, Grandoulier Anne-Sophie, Tse Anissa, Picaut Philippe, Delgado Mauricio R
1 LSUHSC and Children's Hospital New Orleans, New Orleans, LA, USA.
2 Shriner's Hospital for Children, Portland, OR, USA.
J Child Neurol. 2017 Apr;32(5):482-487. doi: 10.1177/0883073816686910. Epub 2017 Jan 9.
This secondary analysis of a large (n = 241), randomized, double-blind study evaluated the efficacy of 2 doses of abobotulinumtoxinA + standard of care (SOC) versus placebo + SOC in enabling children with dynamic equinus due to cerebral palsy to achieve their functional goals using Goal Attainment Scaling. Most parents/caregivers selected goals targeting aspects of gait improvement as most relevant. Mean (95% confidence interval) Goal Attainment Scaling T scores at week 4 were higher for both abobotulinumtoxinA groups versus placebo (treatment difference vs placebo: 10 U/kg/leg: 5.32 [2.31, 8.32], P = .0006, and 15 U/kg/leg 4.65 [1.59, 7.71], P = .0031). Superiority of both abobotulinumtoxinA doses versus placebo was maintained at week 12. Best goal attainment T scores were higher in the abobotulinumtoxinA groups versus placebo for the common goals of improved walking pattern, decreased falling, decreased tripping, and improved endurance. These findings demonstrate that single injections of abobotulinumtoxinA (10 and 15 U/kg/leg) significantly improved the ability of pediatric cerebral palsy patients to achieve their functional goals.
这项对一项大型（n = 241）随机双盲研究的二次分析，使用目标达成量表评估了2剂阿柏西普肉毒素A加标准护理（SOC）与安慰剂加SOC相比，在使因脑瘫导致动态马蹄足的儿童实现其功能目标方面的疗效。大多数父母/照顾者选择了以步态改善方面为目标的目标，认为这些目标最相关。在第4周时，两个阿柏西普肉毒素A组的目标达成量表T评分均值（95%置信区间）均高于安慰剂组（与安慰剂的治疗差异：10 U/kg/腿：5.32 [2.31, 8.32]，P = .0006；15 U/kg/腿：4.65 [1.59, 7.71]，P = .0031）。在第12周时，两种阿柏西普肉毒素A剂量与安慰剂相比的优势得以维持。在改善步行模式、减少跌倒、减少绊倒和提高耐力等共同目标方面，阿柏西普肉毒素A组的最佳目标达成T评分高于安慰剂组。这些发现表明，单次注射阿柏西普肉毒素A（10和15 U/kg/腿）显著提高了小儿脑瘫患者实现其功能目标的能力。
