2024 American Academy of Neurology Abstract Website

Mary Kay Koenig¹, Sarah Wilson¹, Debopam Samanta², Darcy Krueger³, Shelly Meitzler⁴, Carly Kaye⁴, Sherry Danese⁵, Timothy Saurer⁶, Kelly Simontacchi⁶, Karthik Rajasekaran⁶
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, ⁴TSC Alliance, ⁵Outcomes Insights, ⁶Jazz Pharmaceuticals, Inc

Objective:

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.

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:

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).

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%. 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%).

Conclusions:

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.