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(Abst. 3.403), 2015

Cannabidiol (CBD) treatment of refractory epilepsy in tuberous sclerosis complex (TSC)
Authors: Alexandra Geffrey, Sarah F. Pollack, Jan Paolini, Evan Hess, Amy Morgan, Patricia L. Bruno, Elizabeth Thiele
Content: Rationale: Tuberous sclerosis complex (TSC) is an autosomal dominant genetic disorder characterized by benign hamartomas in multiple organ systems. Under an expanded access investigational new drug (IND) trial, cannabidiol (CBD; Epidiolex, GW Pharma) is being studied as a possible adjuvant treatment of refractory epilepsy in children. Of the subjects in the trial at the Massachusetts General Hospital, 10 had a diagnosis of definite TSC. The safety and efficacy of CBD in treating pediatric refractory epilepsy in TSC was evaluated in these 10 patients.Methods: Ten patients with refractory epilepsy and the definite diagnosis of TSC on CBD treatment under IND 119876 were included in this study. Demographic information was collected for each patient including age, sex, epilepsy history, and genetic mutations. Responder rate (>50% decrease in seizures) was calculated and side effects and subjective change in cognitive and behavioral problems were recorded over the course of 2,3,6,9 and 12 months of CBD treatment. Neuropsychological assessments were performed at baseline and after 8 months of CBD treatment.Results: Overall there was a response rate of 50, 50, 40, 60 and 66% at 2,3,6,9 and 12 months of CBD treatment, respectively. One patient became seizure free after 2 months of CBD treatment. Parents reported improvements in alertness, verbal capacity/communication, vocalizations, cognitive availability and initiation of emotional and physical connectsion as well as heightened expression of emotion. Side effects were seen in 50% of patients (n=5) and most resolved with antiepileptic drug or CBD dose adjustment.Conclusions: These data suggest that CBD is safe, well-tolerated and effective, expanding treatment options for refractory epilepsy in individuals with TSC.