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

• Published in: Toxins Vol. 15; no. 1; p. 20
• Main Authors: Bertoncelli, Carlo M., Latalski, Michal, Bertoncelli, Domenico, Bagui, Sikha, Bagui, Subhash C., Gautier, Dechelle, Solla, Federico
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 28.12.2022; MDPI
• Subjects: Accuracy; Algorithms; Artificial intelligence; Bone dysplasia; Bone surgery; Botulinum toxin; Botulinum Toxins, Type A - therapeutic use; Cerebral palsy; Cerebral Palsy - complications; Cerebral Palsy - diagnosis; Cerebral Palsy - drug therapy; Child; Children; Clinical trials; Double-Blind Method; Dystonia; Etiology; Genotype & phenotype; Hip dislocation; Humans; Longitudinal Studies; Machine Learning; Multivariate analysis; Muscle Spasticity - diagnosis; Muscle Spasticity - drug therapy; Neuromuscular Agents - therapeutic use; neurotoxin treatment; Neurotoxins; Neurotoxins - therapeutic use; Ostomy; Paralysis; Patients; Phenotypes; prediction model; Prediction models; Prognosis; Regression analysis; Scoliosis; Spasticity; Toxins; Trunk muscles; cerebral palsy; neurotoxin treatment; prediction model
• ISSN: 2072-6651; 2072-6651
• DOI: 10.3390/toxins15010020

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.