Caregiver-Reported Seizure Outcomes with Real-World Use of Cannabidiol (CBD) in Tuberous Sclerosis Complex (TSC): Interim Results from the BECOME-TSC Survey

Abstract number : 2.503
Submission category : 7. Anti-seizure Medications / 7E. Other
Year : 2023
Submission ID : 1392
Source : www.aesnet.org
Presentation date : 12/3/2023 12:00:00 AM
Published date :

Authors :
Presenting Author: Mary Kay Koenig, MD – McGovern Medical School

Sarah Wilson, MD – McGovern Medical School; Debopam Samanta, MD – University of Arkansas of Medical Sciences; Darcy A. Krueger, MD, PhD – Cincinnati Children’s Hospital Medical Center; Shelly Meitzler, AA – TSC Alliance; Carly Kaye, MEd – TSC Alliance; Sherry R. Danese, MBA – Outcomes Insights; Timothy B. Saurer, PhD – Jazz Pharmaceuticals; Kelly C. Simontacchi, PhD – Jazz Pharmaceuticals; Karthik Rajasekaran, PhD – Jazz Pharmaceuticals

TSC is a neurocutaneous disorder with recurrent and treatment-resistant seizures, which are a significant and frequent cause of morbidity. A pharmaceutical formulation of highly purified CBD is approved for the treatment of seizures associated with TSC. BECOME-TSC (caregiver survey to assess changes in BEhavior, COgnition, and MOre with Epidiolex ® in TSC) is an ongoing cross-sectional survey to quantify the real-world impact of CBD on seizure and nonseizure outcomes in people with TSC. Here we report preliminary findings on seizure-related outcomes of the survey.

Using electronic health records, healthcare professionals at TSC centers identified people with TSC who were treated with CBD (Epidiolex ®, 100 mg/kg oral solution) for ≥ 6 months. Caregivers of these patients 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). Continuous variables were summarized as means, medians, and ranges, and categorical variables as frequency distributions and percentages. CBD-associated AEs, which include transaminase elevations, somnolence, decreased appetite, diarrhea, pyrexia, vomiting, fatigue, rash, sleep disorders, and infections, were not assessed.

At the time of this analysis, eight caregivers had completed the survey. Mean (standard deviation [SD]) age of patients was 14 (9) y, and 50% were female. The mean (SD) age at seizure onset was 8 (7) months, and 63% of patients had a history of infantile spasms. At CBD initiation, most patients had focal onset seizures with impaired awareness (63%) or focal to bilateral tonic–clonic seizures (38%), and 38% of respondents each rated these seizure types as the most frequent and severe. The median CBD dose was 16 mg/kg/d. The most common concomitant ASMs were everolimus (50%), clonazepam (38%), and oxcarbazepine (38%). A notable proportion of respondents reported improvements in overall seizure frequency (75%) and severity (88%); 13% of respondents reported worsening in overall seizure frequency. Seizure freedom (for ≥ the past month) was reported in 67% of patients. Improvements were commonly reported in the frequency of focal seizures (71%), nighttime seizures (71%), and status epilepticus (67%). Most respondents reported decreased rescue medication use (86%), the occurrence of seizure-related injuries (75%), emergency room visits (63%), and hospitalizations (63%).

These preliminary results show that a substantial proportion of caregivers of people with TSC reported improvements in seizure frequency and severity and had more seizure-free days per week since initiating CBD. A total of 88% of caregivers reported planning to continue CBD with reduced seizure frequency, reduced seizure severity/duration, and improved cognition as the most common reasons for continuation.

Jazz Pharmaceuticals Inc.

Anti-seizure Medications