Abstract Details

Title Seizure-Related Outcomes With Real-World Use of Cannabidiol (CBD) in Lennox-Gastaut Syndrome and Dravet Syndrome: BECOME, A Caregiver Survey

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) P13 - Poster Session 13 (8:00 AM-9:00 AM)

Poster/Presentation
Number 10-006

Background The survey was developed to characterize and quantify real-world seizure and nonseizure outcomes in patients with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS).

Objective

To characterize and quantify seizure-related outcomes from a cross-sectional caregiver survey, BECOME (global outcomes survey assessing changes in BEhavior, Cognition, and More with Epidiolex^®).

Design/Methods US-based caregivers (N=498) of people with LGS (80%) or DS (20%) who were treated with CBD (Epidiolex^®, 100 mg/mL oral solution) for ≥3 months compared the past month to the period prior to CBD initiation. The survey included multiple choice and rank order questions using symmetrical 3-, 5-, and 7-point Likert scales (from worsening to improvement). Continuous variables were summarized as means, medians, and ranges and categorical variables as frequency distributions and percentages. CBD-associated adverse events can include transaminase elevations, somnolence, decreased appetite, diarrhea, pyrexia, vomiting, fatigue, rash, sleep disorders, and infections, but they were not assessed in this survey.

Results Mean (standard deviation) age: 16 (11) years (LGS, 17 [11]; DS, 12 [10]). Sex: 52% male. Median CBD dose:14 mg/kg/d. Median concomitant antiseizure medications: 4. A notable proportion of respondents reported improvements in seizure frequency (84%), seizure severity (68%), and seizure-free days per week (67%). Respondents reported improvements in specific seizure types: convulsive seizures (72%), drop seizures (71%), nonconvulsive/nondrop seizures (68%), and night-time seizures (62%). Worsening in ≥1 seizure outcome was reported by 6%–22% of respondents. Many respondents also reported reductions in use of rescue medications (57%), emergency room visits (54%), hospitalizations (53%), and occurrence of seizure-related injuries (48%). Seizure freedom (for at least the last month) was reported in 16% of patients.

Conclusions Nearly all caregivers (93%) planned to continue CBD treatment, primarily because of reduced seizure burden but also because of improvements in nonseizure related outcomes, such as emotional function, alertness, cognition, and communication.

Authors/Disclosures
Ngoc Minh D. Le, MD PRESENTER	Dr. Le has received personal compensation for serving as an employee of Greenwich Biosciences. Dr. Le has stock in Greenwich Biosciences.
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.
Sherry Danese (Outcomes Insights)	No disclosure on file
M. S. Perry, MD (Cook Childrens Medical Center)	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.
	No disclosure on file