Seizure Outcomes With Cannabidiol (CBD) in Pediatric Versus Adult Patients With Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS): Subgroup Analysis of BECOME, a Caregiver Survey (P14-1.006)

Abstract

<h3>Objective:</h3> To characterize and quantify seizure-related outcomes in pediatric (&lt;18 years) versus adult (≥18 years) patients from the cross-sectional caregiver survey BECOME (BEhavior, COgnition, and More with Epidiolex^®). <h3>Background:</h3> A substantial proportion of caregivers reported improvements in seizure and nonseizure outcomes in the primary analysis of the survey. <h3>Design/Methods:</h3> The US-based caregivers (N=498) of patients with LGS (80%) or DS (20%) who received ≥3 months of CBD treatment (Epidiolex^®, 100 mg/mL oral solution) compared the past month with the period before CBD initiation. The online survey included multiple-choice and rank-order questions; changes were rated using a symmetrical 5- and 7-point Likert scale (from worsening to improvement). <h3>Results:</h3> Mean (standard deviation) patient age: 16 (11) years; median (IQR) CBD dose: 14 mg/kg/d (8–19 mg/kg/d); median concomitant ASMs: 4. Most caregivers of pediatric (n=315) and adult (n=183) patients reported any improvement (possible, definite minor, or definite major) in seizure frequency (84% each) and severity (77% and 75%). Worsening in seizure frequency was reported by 7% and 5% of caregivers of pediatric and adult patients, respectively; 10% and 6% reported any worsening in seizure severity. Caregivers of pediatric and adult patients reported any decrease in the frequency of specific seizure type as follows: convulsive (72% each), drop (71% each), nonconvulsive/nondrop (66% and 69%), and night-time seizures (61% and 63%). Similar proportions of caregivers of pediatric and adult patients reported improvements in seizure-free days/week for ≥1 seizure type (65% and 70%) and seizure freedom in the past month (18% and 15%). Many respondents in both groups reported reductions in rescue medication use (57% each), emergency room visits (56% and 51%), hospitalizations (55% and 50%), and occurrence of seizure-related injuries (48% and 49%). <h3>Conclusions:</h3> A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in seizure-related outcomes with CBD treatment. <b>Disclosure:</b> Dr. Saurer has received personal compensation for serving as an employee of Jazz Pharmaceuticals. Dr. Saurer has stock in Jazz Pharmaceuticals. Dr. Berg has received personal compensation for serving as an employee of COMBINEDBrain. Dr. Berg has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurocrin. Dr. Berg has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biomarin. The institution of Dr. Berg has received research support from FamilieSCN2A Foundation. Dr. Dixon-Salazar has received personal compensation for serving as an employee of Lennox-Gastaut Syndrome (LGS) Foundation. Dr. Dixon-Salazar has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neuelis. Mrs. Meskis has received personal compensation for serving as an employee of Dravet Syndrome Foundation. Ms. Danese has nothing to disclose. Dr. Le has received personal compensation for serving as an employee of Greenwich Biosciences. Dr. Le has stock in Greenwich Biosciences. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for stoke therapeutics. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Encoded Therapeutics. Dr. Perry has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for GW Biosciences. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zogenix. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurelis. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biomarin. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Taysha. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cycle Pharma. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoke Therapeutics. Dr. Perry has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Zogenix. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for GW Biosciences.