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.
Gormley et al. (Thu,) studied this question.
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