Abstracts

Rationale: Cannabidiol (CBD) has gone through multiple phase 3 trials to confirm efficacy and safety in the treatment of epilepsy in Dravet syndrome and Lennox Gastaut syndrome. Here we present the behavior and quality of life measures reported by parents during four expanded access programs conducted at the Children’s Hospital of Philadelphia, Children’s Hospital of Pittsburgh, and Geisinger Medical Center. Methods: Patients were enrolled into either a Pennsylvania state (PA) sponsored or GW pharma sponsored, Open-label, compassionate use expanded access study of a pharmaceutical formulation of CBD (Epidiolex provided by GW pharma) as adjunctive therapy for treatment resistant epilepsy. Qualitative surveys and a validated pediatric side effect questionnaire were completed to obtain non-seizure changes in patients receiving CBD. The relationship between seizures response, side effects, and behavior changes were studied. Results: Seventy-six patients (44 male) were studied. Mean age was 10.24 years (range 2-20 years). Mean number of concomitant medications was 2.5 (range 1-3). After 3 months to 4 years of CBD treatment, parents reported via qualitative surveys and side effect questionnaire positive changes and other benefits other than decreased seizure frequency. Of the 76 patients enrolled, 57 (75%) of the patient’s parents reported a positive, non-seizure related change. Some of these changes include: improved attention, better sleep, improved mood, decreased drooling, and improved fine and gross motor skills. All patients completed multiple pediatric side effect questionnaires and Pediatric quality of life assessments. Quantitative analysis of these reports will confirm the qualitative reports. Conclusions: From this sample of patients receiving open-label CBD, there is ample qualitative data to suggest that CBD has the potential to effect more than just seizure control. These results are similar to other open-label compassionate use programs. Further controlled research is needed to determine if these effects are robust to placebo exposure and if they are maintained with long term effects. Funding: GW pharma and the State of Pennsylvania