Abstracts

Rationale: Children with drug resistant epilepsy often experience neurodevelopmental problems including cognitive and behavioral deficits; risk factors include seizure activity as well as medication side effects. The safety and tolerability of Epidiolex (GW Pharmaceuticals) is being studied under an FDA granted expanded access IND. In order to assess for possible neurocognitive or neurobehavioral impacts of treatment, neuropsychological and quality of life data are included as part of the study conducted through the Massachusetts General Hospital pediatric epilepsy program. Methods: Neuropsychological and quality of life data is collected at 3 time points during the current study: 1) baseline assessment conducted 1 month prior to initiation of treatment with CBD; 2) 3 months after baseline; 3) 7 months after baseline. Neuropsychological testing is conducted at the first and third visits and a brief screening at visit two. Data collected from participants able to undergo cognitive testing at baseline included domains of general intelligence, attention, executive function, processing speed, language, visual motor skills and manual dexterity. Parent questionnaires are used at each timepoint to assess participants' behavior, adaptive skills and quality of life. Twenty-four participants with refractory epilepsy were assessed. Participants' mean age was 12.3 years. At the time this abstract is written, data from the 24 baseline assessments and 15 patients from visit 2 were collected. Details regarding seizure etiology and frequency as well as current AED treatment were recorded. Results from questionnaires The Vineland Adaptive Behavior Scales (VABS) and the Pediatric Quality of Life Inventory (PedsQL) were analyzed. Results: The PedsQL provides information about the effects of epilepsy on a child's physical, emotional, social, and school functioning. Paired-samples t-tests revealed that school-related quality of life at interim evaluation had improved (t ₍₁₂₎ = 2.51, p < 0.05). Participants' school-related problems decreased from 11.62 points (SD = 4.46) to 9.08 points (SD = 4.66). Item analysis of the school domain revealed ""Paying attention in class"" was significant. The Vineland provides information about individual's adaptive functioning in the areas of communication, daily living, socialization and motor skill domains. No significant differences were found in the domains when assessed at 3 months after baseline (and less than 2 months after reaching goal dose). Conclusions: Improvement in quality of life in school functioning, reflecting better attention skills, is associated with use of CBD for seizure control. There were no significant differences in adaptive skills between baseline and visit 2 evaluations. It is hypothesized that after 6 months of treatment participants will demonstrate improvements in more of the domains assessed.