Seizure Response to Cannabidiol in a State-Sponsored Open-Label Program (S14.006)

Objective: To assess seizure response to pharmaceutical grade CBD formulation (Epidiolex) in patients with treatment-refractory epilepsy enrolled in an open-label safety study. Introduction: CBD use for seizure management through a State-sponsored program allowed us to follow longitudinally a group of patients who have failed at least 4 different AEDs, who were experiencing on average at least 4 countable seizures per month prior to enrollment, and who were referred to the study by their managing neurologist or epilepsy specialist. Methods: In all 51 patients, pre-CBD seizure frequency was averaged over 3 months prior to initiating CBD at 5mg/kg/day for seizure control with adjustments as tolerated q2 weeks by 5mg/kg/day up to a maximum of 25mg/kg/day. Bi-weekly seizure frequency was monitored via diary. Other AEDs were adjusted as needed (especially clobazam and valproate). Standard statistical measures were used for this non-normally distributed cohort. Results: 51 patients (23 pediatric and 28 adults) had at least one follow-up visit (343 visits total); 25 (49[percnt]) were responders at the last visit (≥50[percnt] seizure reduction). There was no difference in responder rates between pediatric and adult groups (Chi-square p=0.55). 9/51 (18[percnt]) patients were no longer participating in the study at the last visit either due to lack of efficacy (7; 5 children and 2 adults) or intolerable side effects (2-diarrhea; 1 child and one adult). With up-titration of CBD, seizure control improved when compared to baseline (32, 45, 45, 43, 41[percnt] decrease when taking 5, 10, 15, 20, or 25mg/kg/d of CBD, respectively). Two patients were seizure-free at the last assessment. This trend was significant (Spearman rho=-0.316; p=0.000). Conclusions: This compassionate use open-label study indicates approximately 50[percnt] responder rate at the last visit with overall sustained improvement in seizure control over the 6-month duration of the study. Disclosure: Dr. Szaflarski has nothing to disclose. Dr. Bebin has received personal compensation for activities with Medscape. Dr. DeWolfe has nothing to disclose. Dr. Dure has nothing to disclose. Dr. Gaston has nothing to disclose. Dr. Harsanyi has nothing to disclose. Dr. Houston has nothing to disclose. Dr. McGrath has nothing to disclose. Dr. Perry has nothing to disclose. Dr. Singh has nothing to disclose. Dr. Thomas has nothing to disclose. Dr. Ver Hoef has nothing to disclose.