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Abstract Details

Caregiver-reported Seizure 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)
1-005

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 seizure-related outcomes from the BECOME-TSC caregiver survey to assess changes in BEhavior, COgnition, and More with Epidiolex® in TSC.

Caregivers of people with TSC treated with CBD (Epidiolex®, 100 mg/kg oral solution) for ≥6 months completed an online survey, consisting of multiple choice and rank order questions, based on the TSC-Associated Neuropsychiatric Disorders questionnaire, other validated measures, and previous caregiver reports, using a symmetrical Likert scale (from worsening to improvement).

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%. Most frequently reported seizure types at CBD initiation: focal onset seizures with impaired awareness (50%) and focal to bilateral tonic–clonic seizures (50%); 33% and 42% of respondents, respectively, rated these seizure types as the most frequent and severe. Median CBD dose: 17 mg/kg/d. Most common concomitant ASMs: everolimus (42%) and clonazepam (33%). Improvements in overall seizure frequency and severity were reported by 83% and 92% of respondents; 8% reported worsening in seizure frequency. Seizure freedom (for ≥the past month) was reported by 63%. Improvements were commonly reported in frequency of seizures that involve movement or stiffening of both (70%) or one side (60%) of the body, nighttime seizures (67%), status epilepticus (57%), and seizures where one is less responsive/inattentive (56%). Most respondents reported decreased rescue medication use (80%), occurrence of seizure-related injuries (75%), and hospitalizations (58%).

In this preliminary report, a substantial proportion of caregivers of people with TSC reported improvements in seizure frequency and severity and had more seizure-free days/week 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.
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.
Sarah L. Wilson, MD (UT Houston Medical School) Dr. Wilson has nothing to disclose.
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.