Abstracts

Rationale: Parents of children with treatment-resistant epilepsy (TRE) typically explore all available options available for seizure control, given concerns not only regarding quality of life but also development.  Recent focus in the field has turned to marijuana-based medications such as Epidiolex (highly purified cannabidiol) oral solution.  Studies with Epidiolex have demonstrated improved seizure control among children with different forms of TRE.  Studies have also focused on quality of life, including cognitive functioning using investigator designed surveys and formal questionnaires such as the VABS, QOLCE, ABAS, and PedsQL.  Prior findings have identified parental reports of improvement in the areas of alertness, behavior, language, and motor skills, mostly by parental survey.  This study focused on examining potential changes in cognitive functioning among children treated with Epidiolex as part of a larger research study examining the efficacy of Epidiolex on seizure control. Methods: Twenty two pediatric patients with TRE were included in the study.  Cognitive functioning was assessed via parent/caregiver reports using the following questionnaires: Vineland Adaptive Behavior Scales-II (Vineland-II), Child Behavior Checklist (CBCL), and Conners 3/Conners Parent Rating Scale - Long (CPRL) questionnaires.  Parents completed questionnaires at baseline prior to their children starting Epidiolex and again 12 months later.  Means on indices were compared using t-tests to determine if any changes were significant between baseline assessment and reassessment 12 months later.  Results: Patients ranged in age from 1 to 17 years. 17 of the 22 participants finished the study.  A significant change was seen on only one index: the Conners 3 Learning Problems index (p = 0.02).   Conclusions: This result represented a significant decline in parent/caregiver concerns regarding academic functioning in pediatric patients with TRE receiving a trial of Epidiolex, which is encouraging.  Although significant improvements were not seen on other measures of language, motor skills, attention, or executive functioning, this too is encouraging as no signs of significant decline were seen in these areas either, suggesting relatively stable functioning in the interim.  These preliminary results suggest that Epidiolex may benefit certain pediatric patients not only regarding seizure control but also regarding potential impact upon cognitive functioning. Funding: Research funding for this study was provided by G.W. Pharmaceuticals.