Annual Meeting Abstracts: View

  • (Abst. 3.404), 2015
  • Cannabidiol treatment of refractory epileptic spasms: an open label study
  • Authors: Elena Abati, Evan Hess, Amy Morgan, Patricia L. Bruno, Elizabeth Thiele
  • Content:

    Rationale: Epileptic spasms is an epilepsy syndrome with onset in infancy and early childhood that is often associated with significant cognitive impairment and developmental delay. Epileptic spasms persisting into childhood is typically highly refractory. We are conducting an expanded access investigational new drug (IND) trial to evaluate cannabidiol (CBD) as a possible adjuvant treatment of refractory epilepsy. Of the 54 subjects enrolled, 9 had a diagnosis of infantile or epileptic spasms. The aim of this study was to determine the safety and efficacy of CBD in treating epileptic spasms.Methods: We included in this study nine patients with refractory epilepsy and a diagnosis of infantile or epileptic spasms. We collected demographic information and epilepsy history for each patient. Following a one month baseline period, patients were started on CBD (Epidiolex, GW Pharma) at a dosage of 5 mg/kg/daily, increasing by 5 mg/kg/daily each week to an initial maximal dose of 25 mg/kg/day. The number of patients who became seizure free as well as responder rate (greater than 50% reduction of seizures) were determined over the course of 3,6,9 and 12 months of CBD treatment. Side effects of the medication as well as parental report of subjective cognitive and behavioral changes were noted. Neuropsychological testing was also performed at baseline, and after 8 months of CBD treatment.Results: The patients ranged in age from 2-16 years (average 9 years). Etiologies of spasms included 15q13 duplication, CASK mutation, lissencephaly, polymicrogyria, tuberous sclerosis complex (3) and idiopathic (2). The patients had been treated with an average of 8 anticonvulsant medications, 6 had been on dietary therapy, 3 had VNS and one had prior epilepsy surgery. Overall there was a response rate to CBD of 37.5, 43, 50 and 30% at 3, 6, 9, 12 months. Three patients were seizure free after two months of treatment. Parents reported improvement in alertness, verbal capacity/communications, cognitive availabilities. Side effects were seen in 90% of patients (n=8) and most were resolved with anti-epileptics or CBD dose adjustment.Conclusions: Our results suggest that cannabidiol is safe, effective and well-tolerated, thus highlighting its potential as a new treatment option for refractory epileptic spasms and encouraging further studies.