OnabotulinumtoxinA: Safety and Efficacy for Treating Children With Spasticity in Cerebral Palsy
At the American Academy of Neurology (AAN), researchers Heakyung Kim, Jill Meilahn, Chengcheng Liu, Henry G. Chambers, and Rozalina Dimitrova presented results evaluating the efficacy and safety of onabotulinumtoxinA (Botox; Allergan, Dublin, Ireland) for treating children with lower limb spasticity of cerebral palsy.
These researchers carried out a phase 3 clinical trial with 384 pediatric patients with cerebral palsy age 2 to 17 years. Children with an ankle plantar flexor Modified Ashworth Scale-Bohannon (MAS) ≥2 received either 4 U/kg or 8U/kg of onabotulinumtoxinA or a placebo injected into the ankle plantar flexors. All patients enrolled also received standardized physiotherapy.
OnabotulinumtoxinA decreased spasticity as measured by the MAS score. At weeks 4 and 6, the mean MAS score had decreased by 1.1 and 1.0 for both treated groups (8 U/kg and 4 U/kg respectively), which was significantly higher than the placebo group decrease of 0.8 (P = .05). OnabotulinumtoxinA 8 U/kg significantly improved clinical global impression by 1.6 points (8 U/kg) or 1.5 points (4 U/kg) versus the improvement by 1.4 points seen in the group given placebo (P = 0.023, P = .229). Both groups that received onabotulinumtoxinA significantly improved active and passive goal attainment scores compared to the placebo group, and those treated with 8 U/kg had significant improvements in measures of gait versus those treated with placebo. Adverse events were similar across treatment groups: onabotulinumtoxinA, 43.3% (n=110); placebo, 49.2% (n=63), and serious adverse events were reported by 1.2% (n=3) and 3.1% (n=4), respectively. No new safety concerns were identified.
OnabotulinumtoxinA has been used for adults with spasticity previously and interim results of the ASPIRE trial (also presented at the AAN meeting) show that over a 1-year course of treatment, onabotulinum toxin was safe and effective with high levels of patient and physician satisfaction.
David Nicholson, Chief Research and Development Officer, Allergan said, “If you see the impact it has on these children’s ability to walk, it’s absolutely amazing. Children who could hardly walk, all of the sudden can run. That’s why we develop drugs—to have that type of impact.”