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
Rationale:
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.
Methods:
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.
Results:
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%).
Conclusions:
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.
Funding:
Jazz Pharmaceuticals Inc.
Anti-seizure Medications