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.

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.

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.