Introduction: In view of the wide range of presentations and patient needs, it is becoming increasingly recognized that the treatment of spasticity, including the assessment of treatment goals, needs to be individualized. This prospective study assessed the longitudinal attainment of patient-centered, function-related goal attainment scale (GAS) T-scores after repeated AobotulinumtoxinA (abo-A) injections for 30 months (10 cycles) in the US. Methods: Eligible patients aged 2-17 with PLLS were recruited from investigators’ clinical practices. Prescription decisions were made independent of study enrollment. GAS T-scores were assessed for each injection cycle and goals could be re-defined at each injection visit; scores of 50 reflect goal achievement. Adverse events (AEs) were reported. Results: Of 210 patients in the effectiveness population, 77.6% (n¼163) were previously treated with a botulinum neurotoxin. Available Gross Motor Function Classification System (GMFCS) levels showed that 31.3% (n¼61/ 195) of patients were non-ambulatory (GMFCS Level IV/V). Mean cumulated GAS T-score was 51.1 (SD±9.3). Overall, 75.2% of patients achieved their primary goals. Across all cycles, the mean number of muscles injected ranged from 5.5 (±2.9) to 7.0 (±3.7); the mean number of injection sites ranged from 8.1 (±2.7) to 9.9 (±5.7), with gastrocnemius muscle injections being most common (85.7%). Injection guidance techniques were used in >70% of patients in Cycles 1‒6; electrical stimulation was most frequently used (>50%). In the safety population (n¼102/242 [42.1%]), 392 treatment- emergent AEs, which were generally mild to moderate, were reported. A total of 35 AEs (n¼15/242 patients [6.2%]) were deemed treatment related. Conclusions: Overall, goals were achieved as or better than expected in the majority of patients. Abo-A was well tolerated, with a low incidence of treatment-related AEs. These results confirm that abo-A is an effective treatment option, with a positive risk-benefit profile for PLLS.
Gormley M, Dabrowski E, Delgado MR, Tilton A, Christian A, Evans SH, et al A phase IV, prospective, observational, multicenter study evaluating the effectiveness and safety of AbobotulinumtoxinA in pediatric lower limb spasticity (PLLS). Toxicon. 2022 July;214(Suppl 1):S22-S23.