Session Title: Live Poster Session: Pediatrics
Disclosures: Nigar Dursun, MD: Allergan (Products/Services: Yes) (Consultant/Advisory Board, Research Grant includes principal investigator, collaborator or consultant and pending grants as well as grants already received)Ipsen (Products/Services: Yes) (Consultant/Advisory Board, Research Grant includes principal investigator, collaborator or consultant and pending grants as well as grants already received)Merz (Products/Services: Yes) (Consultant/Advisory Board, Research Grant includes principal investigator, collaborator or consultant and pending grants as well as grants already received)
Objective : Assess the efficacy and safety of upper-limb abobotulinumtoxinA injections for spasticity in children with cerebral palsy (CP).
Design: Double-blind, repeat treatment (≤4 cycles over 1 year) study (NCT02106351).
Setting : International study in 32 specialist centers.
Participants : Children (2-17 years) with CP and spasticity in ≥1 upper-limb.
Interventions: 212 Children were randomized (1:1:1) to Cycle 1 injections of abobotulinumtoxinA 8U/kg, 16U/kg or 2U/kg (low-dose control) into the primary target muscle group (PTMG; elbow or wrist flexors) and additional upper-limb muscles. The PTMG could change in cycles 2-4, when children received 8U/kg or 16U/kg. All children participated in an individualized home therapy program (HETP).
Main Outcome Measures: Primary endpoint: Change in Modified Ashworth Scale from baseline to Week 6 of Cycle 1 for PTMG (MAS-PTMG). Secondary endpoints: Physician Global Assessment (PGA) and Goal Attainment Scaling (GAS) at Week 6 of Cycle 1.
Results: At Week-6, treatment with abobotulinumtoxinA at doses of 8U/kg or 16U/kg significantly reduced MAS-PTMG scores versus the 2U/kg low-dose control (treatment differences of -0.4 [p=0.0118] and -0.7 [p < 0.0001], respectively). Statistical difference versus low-dose control was maintained at Week 16. All groups improved on the Physicians Global Assessment (2.0 grade improvement with 8U/kg and 16U/kg versus 1.8 grade with 2U/kg control). Most children across the three arms achieved their GAS primary goal (70.6–75.8%). Benefits were sustained over the study with repeat abobotulinumtoxinA injections of 8U/kg or 16U/kg. Muscular weakness was the only treatment-related AE reported in >1 child/group (4.3%, 5.7% vs. 1.4%, respectively).
Conclusions: AbobotulinumtoxinA administered at doses of 8U/kg or 16U/kg significantly reduced upper-limb spasticity versus the 2U/kg control dose and was well-tolerated. Treatment (abobotulinumtoxinA plus HETP) was associated with global improvement, and therapeutic benefits were sustained with repeat injections of 8U/kg or 16U/kg over one year.
Level of Evidence: Level I
To cite this abstract in AMA style:Dursun N, Delgado M, RIO JC, Tilton AH, Bonikowski M, Oleszek J, Dabrowski E, Grandoulier A, Picaut P. Efficacy and Safety of Abobotulinumtoxina for Upper Limb Spasticity in Children with Cerebral Palsy: Results from an International, Phase 3, Pivotal Study [abstract]. PM R. 2020; 12(S1)(suppl 1). https://pmrjabstracts.org/abstract/efficacy-and-safety-of-abobotulinumtoxina-for-upper-limb-spasticity-in-children-with-cerebral-palsy-results-from-an-international-phase-3-pivotal-study/. Accessed October 23, 2021.
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PM&R Meeting Abstracts - https://pmrjabstracts.org/abstract/efficacy-and-safety-of-abobotulinumtoxina-for-upper-limb-spasticity-in-children-with-cerebral-palsy-results-from-an-international-phase-3-pivotal-study/