Abstract Details

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.

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.