Abstracts

Rationale: Cannabidiol (CBD) has gone through multiple phase 3 trials to confirm efficacy and safety in the treatment of epilepsy.   There remain parental and on-line views of non-seizure related benefits of medicinal cannabis-based therapies.  Determining if such adjunctive effects of CBD based preparations exist will help determine how to best use these emerging therapeutics.  Here we present the anecdotal changes reported by parents during two expanded access programs conducted at the Children’s Hospital of Philadelphia.  Methods: Patients were enrolled into either a PA state sponsored (n=16) or investigator sponsored (n=26), Open-label, compassionate use expanded access study of a pharmaceutical formulation of CBD (Epidiolex provided by GW Research LTD) as adjunctive therapy for treatment resistant epilepsy. Standard qualitative questions and a validated pediatric side effect questionnaire were completed at each clinical visit by the parents/caregiver.  We reviewed the answers to these questions and analyzed the Pediatric side effect questionnaire data to determine if there were non-seizure changes in patients receiving CBD.  We implemented summary statistics and reviewed case histories to determine non-seizure related changes.  Report of positive CBD benefit was compared to seizure calendar data.  Comparison of the two cohorts of patients will allow for internal control for parental and care giver bias. Results: Forty-two patients (23 male) were studied. Mean age was 10.57 years (range 3-18 years). Mean number of concomitant medications was 2 (range 1-3). All patients had neurodevelopmental or neurocognitive problems.  After 5 months to 3.5 years of CBD treatment, parents reported via qualitative questions and a validated side effect questionnaire positive changes and other benefits other than decreased seizure frequency. Of the 42 patients enrolled, 30 (71.4%) of the patient’s parents reported a positive, non-seizure related change. These changes included: improved attention, better sleep, improved mood, decreased drooling, and improved fine and gross motor skills.  Conclusions: This small sample of patients receiving open-label CBD (Epidiolex) reveals positive parental responses regarding their children’s outcomes.  Our data suggest that in patients with intractable seizures and cognitive disabilities that CBD has a potential greater effect than just seizure control.  These results are similar to other open-label compassionate use programs. Multiple limitations exist of this study including parental reporting bias, investigator bias, interaction of seizure and cognitive/behavioral effects.  Controlled research is needed to determine if these results are reproducible in a placebo- controlled study and if the long term cognitive/behavioral effects are maintained.  Funding: GW Research LTD and the State of Pennsylvania