Therapeutics
Mauricio R. Delgado, MD
Physician
Scottish Rite Hospital and University of Texas Southwestern Medical Center
Dallas, Texas, United States
Mathieu Beneteau, BA
Biometry
Ipsen, Paris, France
Paris, Ile-de-France, France
Pascal Maisonobe, MS
Biometry
Ipsen
Paris, Ile-de-France, France
Christian Hannes, PhD
Medical director
Ipsen
München, Bayern, Germany
Primary analyses from this randomized, controlled study demonstrated that treatment with abobotulinumtoxinA (8U/kg or 16U/kg) significantly reduced upper-limb spasticity compared to the control dose. Therapeutic benefits were sustained across repeat treatment cycles in children with cerebral palsy (CP). We hypothesized that the presence of dystonia would influence both the reduction in muscle tone and the interval between injections.
This post-hoc analysis was based on a double-blind, repeat-cycle study (NCT02106351) involving children with CP aged 2-17y. Children who received abobotulinumtoxinA (8U/kg or 16U/kg) were stratified into two subgroups using the Hypertonia Assessment Tool (HAT): those with mixed spasticity and dystonia, and those with predominantly spastic presentations. Change from baseline in the Modified Ashworth Scale for the Primary Target Muscle Group (MASPTMG) was assessed for Cycle 1. Eligibility for retreatment was evaluated from Week 16 and then every 6±2 weeks until Week 52.
During Cycle 1, 139 children were treated with abobotulinumtoxinA at doses of 8U/kg or 16U/kg and had a post-baseline MAS assessment. Of these, 29.5% (n=41) exhibited a mixed spasticity and dystonia presentation, while 70.5% (n=98) presented predominantly with spasticity. Reductions in mean±SD MASPTMG scores tended to be greater in children with a mixed presentation compared to those with a spastic presentation at both Week 6 (−2.3±0.7 vs −2.0±1.1; p=0.1768) and Week 16 (−1.8±1.0 vs −1.4±1.1; p=0.0597). The median [95%CI] time to retreatment (Kaplan-Meier) was significantly longer for children with a mixed presentation than for those with a spastic presentation (30.1 [23.1–40.1] vs 18.9 [17.9–24.0] weeks; p=0.0007). Treatment-related adverse events were reported in 16% (n=11) of children with a mixed presentation and 9% (n=13) with a spastic presentation.
Children with mixed spasticity and dystonia required significantly less frequent reinjection. These findings highlight the importance of considering the presence of dystonia when planning treatment schedules.
