Abstracts

Rationale: Over 90% of people with TSC have TSC-associated neuropsychiatric disorders (TAND). Epidiolex^® is a plant-derived, highly purified, pharmaceutical formulation of CBD approved for the treatment of seizures associated with TSC. The BEhavior, COgnition, and More with Epidiolex^® in TSC (BECOME-TSC) survey was conducted to quantify the real-world effect of CBD on seizure and nonseizure outcomes in people with TSC. Preliminary analyses showed caregiver-reported improvements in seizure and nonseizure outcomes. Here we report the updated survey results for nonseizure outcomes.


Methods: Healthcare providers at TSC centers identified people with TSC treated with CBD (Epidiolex^®, 100 mg/mL oral solution) for ≥ 6 months. Caregivers completed an online survey, consisting of multiple-choice and rank-order questions, based on the TAND questionnaire, other validated measures, and previous caregiver reports, using a symmetrical Likert scale (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 were not assessed.


Results: At the time of abstract submission, 29 caregivers had completed the survey. Mean (SD) age of patients was 17 (11) years; 52% were female. The median (Q1, Q3) age at seizure onset was 4 (2, 6) months, and 62% of patients had a history of infantile spasms. The median (Q1, Q3) CBD dose was 15 (8, 23) mg/kg/d, and the most frequent concomitant antiseizure medications were clobazam (34%) and everolimus (31%). Common co-occurring neuropsychiatric conditions included developmental delay (83%), autism spectrum disorder (69%), attention deficit hyperactivity disorder (34%), and anxiety disorder (34%). Severe–profound intellectual disability (ID) was reported in 59% of patients and mild–moderate ID in 34%; 7% of patients had fluent verbal language. Compared with the period before CBD initiation, respondents most commonly reported definite improvements (in the past month) in a patient’s ability to use sentences with a noun and verb (56%), say phrases with ≥ 2 words (55%), look up or smile when someone said their name (54%), communicate wanting more of something specific (52%), and be happy (52%). Definite worsening was reported by ≤ 12% of respondents in domains including use of repetitive words/phrases, activity restriction, sleep problems, missed school, and eating difficulties. Overall, 97% of caregivers planned to continue CBD. The most important reasons for continuing CBD were reduced seizure frequency (89%), reduced seizure severity/duration (71%), and improved cognition, communication, and social function (50% each).


Conclusions: These findings suggest that CBD treatment is frequently associated with both seizure- and TAND-related improvements following treatment for ≥ 6 months. The results further support the real-world effectiveness of CBD in people with TSC, as nearly all caregivers reported planning to continue CBD treatment for their loved one.


Funding: Jazz Pharmaceuticals Inc.