Caregiver-reported Nonseizure Outcomes with Real-world Use of Cannabidiol (CBD) in Tuberous Sclerosis Complex (TSC): Interim Results from the BECOME-TSC Survey
Sarah Wilson1, Mary Kay Koenig1, Debopam Samanta2, Darcy Krueger3, Shelly Meitzler4, Carly Kaye5, Sherry Danese6, Timothy Saurer7, Kelly Simontacchi7, Karthik Rajasekaran7
1Department of Pediatrics, Division of Child and Adolescent Neurology, McGovern Medical School, The University of Texas Health Science Center at Houston, 2Child Neurology Section, Department of Pediatrics, University of Arkansas for Medical Sciences, 3Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, 4TSC Alliance, 5TSC Alliance, TSC Alliance, 6Outcomes Insights, 7Jazz Pharmaceuticals Inc.
Objective:

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.

Background:

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.

Design/Methods:

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.

Results:

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.

Conclusions:

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

10.1212/WNL.0000000000206298