The drug recently gained FDA approval for treatment of lower limb spasticity in pediatric patients without cerebral palsy, in which it is associated with improved functional outcomes.
Heakyung Kim, MD
Results from a phase 3 study of onabotulinumtoxinA (Botox; Allergan) show significant achievement of active and passive treatment goals as well as gait in pediatric patients with cerebral palsy (CP) with lower limb spasticity. The results were presented at the 2019 Child Neurology Society Annual Meeting, October 23-26, 2019 in Charlotte, North Carolina.
OnabotulinumtoxinA was recently approved for the treatment of lower limb spasticity in pediatric patients without CP. It had been previously approved to treat upper and lower limb spasticity in adults, as well as upper limb spasticity in pediatric patients.
The double-blinded, placebo-controlled study included 384 patients who were randomly assigned to receive onabotulinumtoxinA 4 U/kg (n = 126), 8 U/kg (n = 124), or placebo (n = 130) injected into ankle plantar flexors on day 1. Patients (age 2-16) who were included had CP and an ankle plantar flexor Modified Ashworth Scale-Bohannon (MAS) score of >2. All patients received weekly standardized physiotherapy (PT).
At baseline, 265 patients set active goals related to walking and moving, while 289 patients set passive goals related to symptoms of pain, spasm, or orthosis/brace wear over a 12-week examination period. Patients were assessed using the Goal Attainment Scale (GAS). A subset of 65 patients were assessed based on the Edinburgh Visual Gait (EVG).
Overall, improvements in GAS were significant for onabotulinumtoxinA plus PT versus placebo plus PT for both active and passive goals at weeks 8 and 12 for patients who received the 8 U/kg dose (P <.010). Patients who received the 4 U/kg dose plus PT saw significant improvements at week 8 (P <.047).
Patients who received either the 4 U/kg or 8 U/kg doses plus PT demonstrated a dose dependent improvement in total EVG score as well as select individual items, including foot stance and swing, compared with placebo. Notably, the 8 U/kg group reached statistical significance versus placebo at week 8 (P =.018).
In June, onabotulinumtoxinA was approved for the treatment of upper limb spasticity in pediatric patients. The approval was backed by data from 2 phase 3 studies in more than 200 patients. The trials included a double-blind, 12-week study as well as a 1-year open-label extension.
“OnabotulinumtoxinA has been demonstrated to be safe to use in the pediatric population and has a high profile of efficacy. Treatments are minimally invasive, and the dosing of the medication is easy to understand,” Heakyung Kim, MD, the A. David Gurewitsch Professor of Rehabilitation and Regenerative Medicine and Professor of Pediatrics at Columbia University Medical Center, told NeurologyLive in a previous interview.
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Kim H, Racette B, Dunn C, Mukherjee S, McCusker E, Liu C, Dimitrova R. Lower Limb Injections of OnabotulinumtoxinA: Improvement in Gait and Treatment Goal Achievement in Pediatric Patients with Cerebral Palsy. Presented at: Child Neurology Society 2019 Annual Meeting. October 23-26, 2019; Charlotte, NC. Abstract #126.