Phase IV, Prospective, Observational, Multicenter Study Evaluating the Effectiveness and Safety of AbobotulinumtoxinA in Pediatric Lower Limb Spasticity (PLLS)
Objective: This prospective study assessed the longitudinal attainment of patient-centered, function-related goal attainment scale (GAS) T-score after repeated abobotulinumtoxinA (aboBoNT-A) injections for ≤30 months (≤10 cycles) in the US.
Background: In view of the wide range of presentations and patient needs, it is becoming increasingly recognized that the treatment of spasticity needs to be individualized, including the assessment of treatment goals.
Design/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% (n=163) were previously treated with a botulinum neurotoxin. Available Gross Motor Function Classification System (GMFCS) levels showed that 31.3% (n=61/195) of patients were non-ambulatory (GMFCS IV or 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 points 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; electrostimulation was most frequent (>50%). In the safety population, n=102/242 (42.1%) reported 392 treatment-emergent AEs, which were generally mild to moderate. A total of 35 AEs in n=15/242 patients (6.2%) were deemed treatment-related.
Conclusions: Overall, goals were achieved as or better than expected in the majority of patients. AboBoNT-A was well tolerated, with a low incidence of treatment-related AEs. These results confirm that aboBoNT-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). Neurology. 2022 May 3;98(Suppl 18):3224.