Prediction Model for Identifying Computational Phenotypes of Children with Cerebral Palsy Needing Neurotoxin Treatments

Abstract

Factors associated with neurotoxin treatments in children with cerebral palsy (CP) are poorly studied. We developed and externally validated a prediction model to identify the prognostic phenotype of children with CP who require neurotoxin injections. We conducted a longitudinal, international, multicenter, double-blind descriptive study of 165 children with CP (mean age 16.5 ± 1.2 years, range 12–18 years) with and without neurotoxin treatments. We collected functional and clinical data from 2005 to 2020, entered them into the BTX-PredictMed machine-learning model, and followed the guidelines, “Transparent Reporting of a Multivariable Prediction Model for Individual Prognosis or Diagnosis”. In the univariate analysis, neuromuscular scoliosis (p = 0.0014), equines foot (p < 0.001) and type of etiology (prenatal > peri/postnatal causes, p = 0.05) were linked with neurotoxin treatments. In the multivariate analysis, upper limbs (p < 0.001) and trunk muscle tone disorders (p = 0.02), the presence of spasticity (p = 0.01), dystonia (p = 0.004), and hip dysplasia (p = 0.005) were strongly associated with neurotoxin injections; and the average accuracy, sensitivity, and specificity was 75%. These results have helped us identify, with good accuracy, the clinical features of prognostic phenotypes of subjects likely to require neurotoxin injections.