Abstracts

Rationale: LGS and DS are rare and severe epileptic encephalopathies with onset in infancy or early childhood. Highly purified pharmaceutical CBD is FDA-approved for the treatment of seizures associated with LGS, DS, and tuberous sclerosis complex in patients ≥1 year of age. To better understand real-world treatment outcomes, we developed a cross-sectional caregiver survey, BECOME (global outcomes survey assessing changes in BEhavior, Cognition, and More with Epidiolex®) to characterize and quantify real-world seizure and non-seizure outcomes in patients with LGS or DS. This first abstract reports on the seizure-related outcomes from the survey.

Methods: Survey participants were from a Greenwich Biosciences, Inc. patient caregiver database. US-based caregivers of people with LGS or DS who were treated with CBD (Epidiolex®, 100 mg/mL oral solution) for ≥3 months were asked to compare the past month to the period prior to initiation of CBD. The 20-minute survey was administered online and consisted of multiple choice and rank order questions, based on questions from validated measures and other previously published caregiver reports, using a symmetrical 5-point 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 can include transaminase elevations, somnolence, decreased appetite, diarrhea, pyrexia, vomiting, fatigue, rash, sleep disorders, and infections, but they were not assessed in this survey.

Results: A total 498 caregivers (97% parents) of people with LGS (80%) or DS (20%) completed the survey. Patients were a mean (standard deviation) age of 16 (11) years (LGS, 17 [11]; DS, 12 [10]) and 52% male. They were taking a median CBD dose of 14 mg/kg/day and a median 4 concomitant antiseizure medications. A notable proportion of respondents reported improvements in seizure frequency (84%), seizure severity (68%), and seizure-free days per week (67%). Respondents reported improvements in specific seizure types as follows: convulsive seizures (72%), drop seizures (71%), non-convulsive/non-drop seizures (68%), and night-time seizures (62%). Worsening in at least one of the seizure outcomes was reported by 6% to 22% of respondents. Many respondents also reported reductions in use of rescue medications (57%), emergency room visits (54%), hospitalizations (53%), and occurrence of seizure-related injuries (48%). Seizure freedom (for at least the last month) was reported in 16% of patients.

Conclusions: A substantial proportion of caregivers of people with LGS or DS reported improvements in seizure frequency and severity and had more seizure-free days per week since initiating CBD treatment. A total of 93% of caregivers reported planning to continue CBD treatment, primarily because of reduced seizure burden but also because of improvements in non-seizure related outcomes, such as emotional function, alertness, cognition, and communication.

Funding: Please list any funding that was received in support of this abstract.: Greenwich Biosciences, Inc.