Abstract Details

Title 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

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) P14 - Poster Session 14 (11:45 AM-12:45 PM)

Poster/Presentation
Number 9-008

Objective

To characterize and quantify nonseizure behavioral and cognitive outcomes in pediatric (<18 years) vs adult (≥18 years) patients from the cross-sectional caregiver survey BECOME (BEhavior, COgnition, and More with Epidiolex^®).

Background A substantial proportion of caregivers reported improvements in seizure and nonseizure outcomes in the primary analysis of the survey.

Design/Methods 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).

Results 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%).

Conclusions 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.

Authors/Disclosures
Catherine L. Passariello, PhD (Greenwich Biosciences) PRESENTER	Dr. Passariello has received personal compensation for serving as an employee of Jazz Pharmaceuticals . Dr. Passariello has stock in Jazz Pharmaceuticals . An immediate family member of Dr. Passariello has stock in UCB Pharmaceuticals . Dr. Passariello has received intellectual property interests from a discovery or technology relating to health care.
Tracy Dixon-Salazar, PhD	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.
Anne T. Berg, PhD (Northwestern Univesity Feinberg School of Medicine)	Dr. Berg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biohaven. Dr. Berg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Berg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Encoded. Dr. Berg has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biomarin. The institution of Dr. Berg has received research support from FamilieSCN2A Foundation.
	No disclosure on file
Sherry Danese (Outcomes Insights)	No disclosure on file
Timothy B. Saurer, PhD	Dr. Saurer has received personal compensation for serving as an employee of Jazz Pharmaceuticals. Dr. Saurer has stock in Jazz Pharmaceuticals.
Ngoc Minh D. Le, MD	Dr. Le has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Le has stock in Neurona Therapeutics.
M. S. Perry, MD (Cook Childrens Medical Center)	Dr. Perry has received personal compensation in the range of $10,000-$49,999 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 Neurelis. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Marinus. Dr. Perry has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for jazz pharmaceuticals. Dr. Perry has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for pyros. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for azurity. Dr. Perry has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Perry has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoke Therapeutics. Dr. Perry has stock in Praxis Precision Medicine. Dr. Perry has stock in Biohaven. Dr. Perry has stock in Rapport. Dr. Perry has stock in Alto Neurosciences. Dr. Perry has a non-compensated relationship as a president with child neurology foundation that is relevant to AAN interests or activities. Dr. Perry has a non-compensated relationship as a President with Pediatric Epilepsy Research Consortium that is relevant to AAN interests or activities.

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