Abstract Details

Title Non-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) P1 - Poster Session 1 (8:00 AM-9:00 AM)

Poster/Presentation
Number 10-003

Objective To characterize and quantify nonseizure behavioral and cognitive outcomes from a cross-sectional caregiver survey BECOME (global outcomes survey assessing changes in BEhavior, COgnition, and More with Epidiolex^®).

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).

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 ≥1 question for all domains: emotional functioning (82%), cognition and executive function (81%), language and communication in nonverbal (79%) and verbal patients (74%), activities of daily living (51%), sleep (51%), and physical functioning (46%). Worsening was reported in ≥1 question of each domain by 6%–26% of respondents. Most frequently reported improvements in specific questions included alertness (71%), learning new things (71%), being aware (70%), ability to engage with others (68%), paying attention (66%), happiness (66%), smiling (63%), saying sentences and phrases (58% and 60%), and calmness (56%).

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.

Authors/Disclosures
Anne T. Berg, PhD (Northwestern Univesity Feinberg School of Medicine) PRESENTER	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.
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 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 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 Longboard Pharmaceuticals. Dr. Perry has stock in Rapport. Dr. Perry has a non-compensated relationship as a president elect 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.
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.
	No disclosure on file
Ngoc Minh D. Le, MD	Dr. Le has received personal compensation for serving as an employee of Lundbeck Pharmaceuticals. Dr. Le has stock in Longboard Pharmaceuticals.
Sherry Danese (Outcomes Insights)	No disclosure on file

��ɫ��

��ɫ��