Delgado Mauricio R, Tilton Ann, Russman Barry, Benavides Oscar, Bonikowski Marcin, Carranza Jorge, Dabrowski Edward, Dursun Nigar, Gormley Mark, Jozwiak Marek, Matthews Dennis, Maciag-Tymecka Iwona, Unlu Ece, Pham Emmanuel, Tse Anissa, Picaut Philippe
Texas Scottish Rite Hospital for Children, Dallas, Texas; University of Texas Southwestern Medical Center, Dallas, Texas;
Louisiana State University Health Center and Children's Hospital New Orleans, New Orleans, Louisiana;
Pediatrics. 2016 Feb;137(2):e20152830. doi: 10.1542/peds.2015-2830. Epub 2016 Jan 26.
Although botulinum toxin is a well-established treatment of focal spasticity in cerebral palsy, most trials have been small, and few have simultaneously assessed measures of muscle tone and clinical benefit.
Global, randomized, controlled study to assess the efficacy and safety of abobotulinumtoxinA versus placebo in cerebral palsy children with dynamic equinus foot deformity. Patients were randomized (1:1:1) to abobotulinumtoxinA 10 U/kg/leg, 15 U/kg/leg, or placebo injections into the gastrocnemius-soleus complex (1 or both legs injected). In the primary hierarchical analysis, demonstration of benefit for each dose required superiority to placebo on the primary (change in Modified Ashworth Scale from baseline to week 4) and first key secondary (Physician's Global Assessment at week 4) end points.
Two hundred and forty-one patients were randomized, and 226 completed the study; the intention to treat population included 235 patients (98%). At week 4, Modified Ashworth Scale scores significantly improved with abobotulinumtoxinA; mean (95% confidence interval) treatment differences versus placebo were -0.49 (-0.75 to -0.23; P = .0002) for 15 U/kg/leg and -0.38 (-0.64 to -0.13; P = .003) for 10 U/kg/leg. The Physician's Global Assessment treatment differences versus placebo of 0.77 (0.45 to 1.10) for 15 U/kg/leg and 0.82 (0.50 to 1.14) for 10 U/kg/leg were also significant (both Ps < .0001). The most common treatment-related adverse event was muscular weakness (10 U/Kg/leg = 2; placebo = 1).
AbobotulinumtoxinA improves muscle tone in children with dynamic equinus resulting in an improved overall clinical impression and is well tolerated.
尽管肉毒杆菌毒素是治疗脑瘫局部痉挛的一种成熟疗法，但大多数试验规模较小，很少有试验同时评估肌张力指标和临床疗效。
一项全球性、随机、对照研究，旨在评估阿柏西普肉毒杆菌毒素A对比安慰剂治疗患有动态马蹄足畸形脑瘫儿童的疗效和安全性。患者被随机分为三组（1:1:1），分别接受10 U/kg/腿、15 U/kg/腿的阿柏西普肉毒杆菌毒素A或安慰剂注射到腓肠肌-比目鱼肌复合体（一条腿或双腿注射）。在主要分层分析中，每种剂量显示出疗效需要在主要终点（从基线到第4周改良Ashworth量表的变化）和第一个关键次要终点（第4周医生整体评估）上优于安慰剂。
241名患者被随机分组，226名完成研究；意向性分析人群包括235名患者（98%）。在第4周时，阿柏西普肉毒杆菌毒素A使改良Ashworth量表评分显著改善；与安慰剂相比，15 U/kg/腿的平均（95%置信区间）治疗差异为-0.49（-0.75至-0.23；P = 0.0002），10 U/kg/腿为-0.38（-0.64至-0.13；P = 0.003）。医生整体评估与安慰剂相比，15 U/kg/腿的治疗差异为0.77（0.45至1.10），10 U/kg/腿为0.82（0.50至1.14），也具有显著性（P均<0.0001）。最常见的与治疗相关的不良事件是肌肉无力（10 U/Kg/腿组=2例；安慰剂组=1例）。
阿柏西普肉毒杆菌毒素A可改善患有动态马蹄足儿童的肌张力，从而改善整体临床印象，且耐受性良好。
