Efficacy and Safety of AbobotulinumtoxinA in Pediatric Lower Limb Spasticity: 2nd Interim Results From a Phase IV, Prospective, Observational, Multicenter Study.
Objectives: The primary objective was to assess subject-centered, function-related goal attainment (T-score) after repeated abobotulinumtoxinA (aboBoNT-A) injections. A 1st interim analysis included efficacy results from treatment cycle 1. We report updated data after as many as 5 cycles, including long-term safety results assessed for ≤18 months.
Design: This phase IV study collects real-world data on the clinical use of aboBoNT-A in patients with pediatric lower limb spasticity (PLLS), aged 2-17 years. Prescription decisions were made prior to/independent of study enrollment. Functional goals were identified at baseline by patient/parent/caregiver in consultation with investigators. Adverse events were reported.
Results: This 2nd interim analysis included 201 patients, of which 78.1% (n=157) had received prior botulinum neurotoxin (BoNT) treatment. At enrollment, 69.2% were aged 2-9. Average time to the 2nd and 3rd treatments was 24.79 (SD 12.38) and 42.58 (12.85) weeks, respectively. The cumulative GAS T-score for the total population was 51.60 (9.69). By the last treatment assessed (in this 2nd interim analysis), mean T-score for the total population (N=201) was 48.14 (8.08); BoNT-naïve (n=44) had a T-score of 52.07 (3.58) vs 47.52 (8.47) in BoNT‒non-naïve (n=157) patients aged 2-9; T-score was 46.66 (8.31) vs 52.07 (6.41), respectively, in patients aged 10-17. In the safety population (N=243), 44 TEAEs were reported in 26 patients; most were mild to moderate, with 1 severe. Extremity pain, limb discomfort, muscle swelling, and myalgia reported in 3 patients were deemed treatment related. No reported TEAEs led to study drug withdrawal or death.
Conclusions: Goal attainment outcomes reflect overachievement (T-score slightly higher than 50.0) for the overall PLLS population. AboBoNT-A was well tolerated, with a low incidence of TEAEs. These results support aboBoNT-A as an effective treatment option with a positive risk-benefit profile for patients with PLLS.
Gormley M, Dabrowski E, Tilton A, Christian A, Evans SH, Maisonobe P, et al. Efficacy and safety of abobotulinumtoxinA in pediatric lower limb spasticity (PLLS): 2nd interim results from a Phase IV, prospective, observational, multicenter study. 2021;190:S28. Available from: https://www.sciencedirect.com/science/article/pii/S0041010120308424