Efficacy and safety of onabotulinumtoxinA with standardized physiotherapy for the treatment of pediatric lower limb spasticity: A randomized, placebo-controlled, phase III clinical trial.

All groups received standardized physical therapy, so the trial shows the effect of adding onabotulinumtoxinA to therapy, not the effect of onabotulinumtoxinA alone. The trial used fixed doses and a fixed injection pattern into gastrocnemius, soleus, and tibialis posterior, which may not match individual clinical choices. Most children were GMFCS level I or II, and only five were level IV, so results may not apply to more severely affected children. The primary outcome measure, Modified Ashworth Scale, has been criticized because it does not account for velocity-dependent tone. The study lasted 12 weeks and tested a single treatment, so it does not show long-term effects or repeat treatment outcomes. Gait analysis was done only in a subset at selected sites, so gait findings may not apply to all participants. Secondary endpoints were not controlled for multiplicity. No independent second rater was used for the Modified Ashworth Scale, although assessors were trained.

The supplied text does not include a funding or conflict-of-interest declaration. It names onabotulinumtoxinA as BOTOX from Allergan, an AbbVie company, and describes the trial as the registrational study leading to Food and Drug Administration approval.

Do not conclude that onabotulinumtoxinA alone produced the improvements, because every child also received standardized physical therapy. Do not assume the fixed injection pattern applies to every child with cerebral palsy, because the trial used set doses and muscles and mostly included children at GMFCS levels I and II.