Authors :
Sarah M.L. Wilson, MD – McGovern Medical School, The University of Texas Health Science Center at Houston
Mary Kay Koenig, MD – McGovern Medical School at the University of Texas Health Science Center
Debopam Samanta, MD – University of Arkansas for Medical Sciences
Presenting Author: Darcy Krueger, MD, PhD – Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine
Linda Leary, MD – University of Texas Health Science Center
Karen Keough, MD – Child Neurology Consultants of Austin
James Wheless, BScPharm, MD, FAAP, FACP, FAAN, FAES – LeBonheur Children’s Hospital
Shelly Meitzler, N/A – TSC Alliance
Carly Kaye, N/A – TSC Alliance
Sherry R. Danese, N/A – Outcomes Insight
Timothy Saurer, PhD – Jazz Pharmaceuticals, Inc.
Kelly Simontacchi, PhD – Jazz Pharmaceuticals, Inc.
Karthik Rajasekaran, PhD – Jazz Pharmaceuticals, Inc.
Rationale: TSC is a neurocutaneous disorder with treatment-resistant seizures.
Epidiolex
® is a plant-derived, highly purified, pharmaceutical formulation of CBD approved for the treatment of seizures associated with TSC. The
BEhavior,
COgnition, and
More with
Epidiolex
® in TSC (BECOME-TSC) survey was conducted to quantify the real-world effect of CBD on seizure and nonseizure outcomes in people with TSC. Preliminary analyses showed caregiver-reported improvements in seizure and nonseizure outcomes. Here we report the updated survey results for seizure-related outcomes.
Methods: Healthcare professionals at TSC centers identified people with TSC who were treated with CBD (Epidiolex
®, 100 mg/mL oral solution) for ≥ 6 months. Caregivers 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 adverse events were not assessed.
Results: At the time of abstract submission, 29 caregivers had completed the survey. Mean (SD) age of patients was 17 (11) years; 52% were female. The median (Q1, Q3) age at seizure onset was 4 (2, 6) months; 62% of patients had a history of infantile spasms. At CBD initiation, a greater proportion of caregivers reported focal onset seizures with impaired awareness (59%) and focal to bilateral tonic–clonic seizures (28%) than other seizure types; focal onset seizures with impaired awareness were also rated as the most frequent (38%) and severe (31%). Median (Q1, Q3) CBD dose was 15 mg/kg/d (8, 23). Most common concomitant antiseizure medications were clobazam (34%) and everolimus (31%). Improvements were reported by 86% of respondents in overall seizure frequency and by 86% in seizure severity; worsening was reported by 7% and 10% of respondents, respectively. Seizure freedom for the past month was reported in 41% of patients. Improvements were most commonly reported in frequency of focal seizures (68%) and nighttime seizures (64%). Most respondents reported decreased rescue medication use (68%), seizure-related injuries (66%), hospitalizations (62%), and emergency room visits (55%). Overall, 97% of caregivers planned to continue CBD treatment. Reduced seizure frequency (89%), reduced seizure severity/duration (71%), and improved cognition, communication, and social function (50% each) were the most important reasons for continuing CBD treatment.
Conclusions:
Results of this survey show that most caregivers of patients with TSC reported improvements in patients’ overall seizure frequency and severity since initiating CBD. These findings support the real-world effectiveness of CBD in patients with TSC and epilepsy, as nearly all caregivers reported planning to continue CBD treatment for their loved one.
Funding: Jazz Pharmaceuticals Inc.