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 second abstract reports on the non-seizure behavioral and cognitive 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 7-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 of 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. Overall, a notable proportion of respondents reported improvements in at least one question for all domains: emotional functioning (82%), cognition and executive function (81%), language and communication in non-verbal (79%) and verbal patients (74%), activities of daily living (51%), sleep (51%), and physical functioning (46%). Worsening was reported in at least one question of each domain by 6% to 26% of respondents. The most frequently reported improvements in specific questions included the following: alertness (71%), learning new things (71%), being aware (70%), ability to engage with others (68%), paying attention (66%), happiness (66%), smiling (63%), saying sentences and phrases (58% and 60%), and calmness (56%).

Conclusions: A substantial proportion of caregivers of people with LGS or DS reported improvement in emotional functioning, cognition and executive function, and language and communication 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.

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