Gormley Mark E, Dabrowski Edward, Delgado Mauricio R, Tilton Ann, Christian Asare, Evans Sarah Helen, Grandoulier Anne-Sophie, Goldberg Jumaah
Gillette Children's Specialty Healthcare, St. Paul, MN, USA.
Oakland University School of Medicine & Beaumont Hospital, Grosse Point, MI, USA.
Dev Med Child Neurol. 2025 Jul 31. doi: 10.1111/dmcn.16428.
To assess the longitudinal attainment of patient-centered, function-related Goal Attainment Scaling Total (GAS T)-score after repeated abobotulinumtoxinA (AboBoNT-A) injections over a period of up to 30 months and up to 10 cycles.
In this prospective observational study, the investigators' clinical practices recruited patients aged 2 to 17 years with pediatric lower limb spasticity (PLLS). GAS T-scores were assessed for each injection, and goals could be redefined at each visit; scores of 50 reflected goal achievement. Adverse events were recorded.
Of 210 patients in the effectiveness population, 171 had cerebral palsy and 163 were previously treated with a botulinum neurotoxin. Available Gross Motor Function Classification System levels showed that 31.3% (61 out of 195) of patients were non-ambulatory. Mean (SD) cumulative GAS T-score was 51.1 (9.3) across the study duration; 75.2% achieved their primary goals. GAS T-scores were comparable between BoNT-naïve and previously treated patients and between age groups, but higher in the ambulatory than the non-ambulatory subgroup. Injection guidance techniques were used in more than 70% of patients in cycles 1 to 6. Of 242 patients in the safety population, 102 reported 392 treatment-emergent adverse events, including 15 reporting 35 treatment-related adverse events. Adverse events were generally mild to moderate.
Overall, goals were achieved as, or better than, expected in most patients. AboBoNT-A was well tolerated, with a low incidence of treatment-related adverse events. These results indicate that AboBoNT-A is an effective treatment option, with a positive risk-benefit profile, for patients with PLLS across disability levels.
评估在长达30个月、多达10个周期内重复注射阿柏西普肉毒素A（AboBoNT-A）后，以患者为中心的、与功能相关的目标达成量表总分（GAS T）的纵向达成情况。
在这项前瞻性观察研究中，研究人员的临床实践招募了2至17岁患有小儿下肢痉挛（PLLS）的患者。每次注射时评估GAS T分数，每次就诊时可重新定义目标；分数为50表示目标达成。记录不良事件。
在有效性人群的210例患者中，171例患有脑瘫，163例曾接受肉毒杆菌神经毒素治疗。可用的粗大运动功能分类系统水平显示，31.3%（195例中的61例）患者不能行走。在整个研究期间，平均（标准差）累积GAS T分数为51.1（9.3）；75.2%的患者实现了主要目标。初次使用肉毒杆菌毒素和曾接受治疗的患者之间以及不同年龄组之间的GAS T分数具有可比性，但能行走的亚组分数高于不能行走的亚组。在第1至6个周期中，超过70%的患者使用了注射引导技术。在安全性人群的242例患者中,102例报告了392起治疗中出现的不良事件，其中15例报告了35起与治疗相关的不良事件。不良事件一般为轻至中度。
总体而言，大多数患者实现了预期或更好的目标。AboBoNT-A耐受性良好，与治疗相关的不良事件发生率较低。这些结果表明，对于不同残疾水平的PLLS患者，AboBoNT-A是一种有效的治疗选择，具有积极的风险效益比。
