Efficacy and Safety of AbobotulinumtoxinA in Pediatric Lower Limb Spasticity: 2nd Interim Results From a Phase IV, Prospective, Observational, Multicenter Study.
American Journal of Physical Medicine and Rehabilitation
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, Maisonobbe P, Wietek S. Efficacy and safety of abobotulinumtoxinA in pediatric lower limb spasticity: 2nd interim results from a phase IV, prospective, observational, multicenter study. Am J Phys Med Rehab. 2021;100(4 suppl.):a162