Dev Med Child Neurol. 2025 Jul 31. doi: 10.1111/dmcn.16428. Online ahead of print.
ABSTRACT
AIM: 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.
METHOD: 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.
RESULTS: 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.
INTERPRETATION: 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.
PMID:40745401 | DOI:10.1111/dmcn.16428
