Nonseizure 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 (P14-1.008)

Abstract

<h3>Objective:</h3> To characterize and quantify nonseizure behavioral and cognitive outcomes in pediatric (&lt;18 years) vs 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> 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 7-point Likert scale (from worsening to improvement). <h3>Results:</h3> Mean (standard deviation) age of patients: 16 (11) years; median (IQR) CBD dose: 14 mg/kg/d (8–19 mg/kg/d); median concomitant ASMs: 4. A similar proportion of caregivers of pediatric (n=315) and adult (n=183) patients reported any improvement (possible, minor, or definite) in ≥1 question of the following domains: alertness, cognition, and executive function (87% and 81%) and emotional and social function (82% and 80%). A numerically greater proportion of caregivers of pediatric versus adult patients reported any improvement in ≥1 question of the language and communication (verbal patients: 81% versus 63%; nonverbal patients: 85% versus 68%), physical functioning (53% versus 33%), sleep (53% versus 48%), and daily activities (56% versus 44%) domains. Rates of worsening varied by behavior and ranged from 0% to 20% of respondents. In pediatric patients, the most common improvements were in the ability to learn new things (76%) and, in nonverbal patients, looking up or smiling at mention of their name (76%). For adult patients, the most common improvement was in alertness (70%). <h3>Conclusions:</h3> A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in outcomes beyond seizure control since initiating CBD treatment. <b>Disclosure:</b> 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. 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. Mrs. Meskis has received personal compensation for serving as an employee of Dravet Syndrome Foundation. Ms. Danese has nothing to disclose. Dr. Saurer has received personal compensation for serving as an employee of Jazz Pharmaceuticals. Dr. Saurer has stock in Jazz Pharmaceuticals. 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.