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Abstract Details

Caregiver-reported Nonseizure Outcomes with Real-world Use of Cannabidiol (CBD) in Tuberous Sclerosis Complex (TSC): Interim Results from the BECOME-TSC Survey
Epilepsy/Clinical Neurophysiology (EEG)
P8 - Poster Session 8 (5:30 PM-6:30 PM)
1-010

BECOME-TSC is an ongoing cross-sectional survey to quantify the real-world impact of CBD treatment on seizure and nonseizure outcomes in people with TSC.

To report preliminary findings on nonseizure behavioral and cognitive outcomes from the BECOME-TSC caregiver survey to assess changes in BEhavior, COgnition, and More with Epidiolex® in TSC.

People with TSC treated with CBD (Epidiolex®, 100 mg/kg oral solution) for ≥6 months were identified using electronic health records. Caregivers of these patients completed an online survey, consisting of multiple choice and rank order questions, based on the TSC-associated neuropsychiatric disorders (TAND) questionnaire, other validated measures, and previous caregiver reports, using a symmetrical Likert scale (from worsening to improvement). CBD-associated AEs were not assessed.

At the time of analysis, 12 caregivers had completed the survey. Mean (standard deviation [SD]) patient age: 16 (8) years. Gender: 58% female. Mean (SD) age at seizure onset: 17 (33) months. History of infantile spasms: 50%. Median CBD dose: 17 mg/kg/d. Most common concomitant ASMs: everolimus (42%) and clonazepam (33%). Patients were diagnosed with the following co-occurring conditions: developmental delay (92%), autism spectrum disorder (83%), anxiety disorder (42%), attention deficit hyperactivity disorder (33%), and obsessive-compulsive disorder (33%). Severe–profound intellectual disability (ID) was reported in 75% of patients, mild–moderate ID in 17%; 8% had fluent verbal language. Compared with the pre-CBD initiation period, respondents most commonly reported definite improvements (for ≥the past month) in a patient’s ability to be happy (67%), shake head for yes/no answer (63%), accomplish visuo-spatial tasks (63%), and remember things that have happened (60%). Definite worsening was reported by ≤2 respondents in domains that included using repetitive words/phrases, repetitive behaviors, impulsivity, and overactivity/hyperactivity.

These preliminary results show that a substantial proportion of caregivers of people with TSC reported improvement in TAND-related outcomes since initiating CBD.

Authors/Disclosures
Karthik Rajasekaran, PhD (Jazz Pharmaceuticals)
PRESENTER
Dr. Rajasekaran has received personal compensation for serving as an employee of Jazz Pharmaceuticals.
Sarah L. Wilson, MD (UT Houston Medical School) Dr. Wilson has nothing to disclose.
Mary Kay Koenig, MD (University of Texas Health Science Center-Houston) Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Marinus Pharmaceuticals. Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz Pharmaceuticals. Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stealth BioTherapeutics. Dr. Koenig has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Reneo Pharmaceuticals. Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acadia Pharmaceuticals. Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Koenig has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz Pharmaceuticals. The institution of Dr. Koenig has received research support from Abliva AB. The institution of Dr. Koenig has received research support from EryDel SPA. The institution of Dr. Koenig has received research support from PTC Therapeutics. The institution of Dr. Koenig has received research support from Noema Pharmaceuticals. The institution of Dr. Koenig has received research support from Marinus Pharmaceuticals. The institution of Dr. Koenig has received research support from Jazz Pharmaceuticals. The institution of Dr. Koenig has received research support from Nflexion Therapeutics. The institution of Dr. Koenig has received research support from Stealth BioTherapeutics. The institution of Dr. Koenig has received research support from Astellas Pharmaceuticals. The institution of Dr. Koenig has received research support from Reneo Pharmaceuticals.
Debopam Samanta, MD Dr. Samanta has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz.
Darcy Krueger, MD (Cincinnati Children'S Hospital Medical Center) Dr. Krueger has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NobelPharma USA. Dr. Krueger has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for RegenxBio. Dr. Krueger has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis Pharmaceuticals. Dr. Krueger has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Greenwich Biosciences. The institution of Dr. Krueger has received research support from Greenwich Bioscience. The institution of Dr. Krueger has received research support from Marinus Pharmaceuticals. The institution of Dr. Krueger has received research support from National Institutes of Health. The institution of Dr. Krueger has received research support from Food and Drug Administration. The institution of Dr. Krueger has received research support from TSC Alliance.
No disclosure on file
No disclosure on file
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.
Kelly Simontacchi (UCB Inc) The institution of Kelly Simontacchi has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Jazz Pharmaceuticals.