BECOME Survey Study Highlights Real-World Benefits of CBD in Rare Epilepsies

Scott M. Perry, MD

In a survey study of patients with either Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), findings showed that treatment with add-on cannabidiol (CBD; Epidiolex) led to improvements in nonseizure outcomes, regardless of reduction in seizure frequency. Despite the limitations associated with a retrospective survey-based study design, investigators concluded that further evaluation of CBD on nonseizure outcomes in these patient populations is warranted.

Published in Epilepsy Research, the study comprised of caregivers of 498 patients with LGS (80%) or DS (20%) on CBD therapy who completed BECOME, a 20-minute, cross-sectional, online survey. On nonseizure domains of alertness, cognition, and executive function, 85% of respondents reported improvements in at least 1 survey question. This continued across several other nonseizure-related domains of emotional functioning (82%), language and communication (79% in nonverbal patients and 74% in verbal), and activities of daily living (51%).

Led by senior investigator Scott M. Perry, MD, medical director of the Cook Children’s Hospital, study results showed that a net of 46% of respondents reported positive changes in physical functioning. Overall, 51% of caregivers (LGS: 52%; DS: 49%) documented improvement in at least 1 aspect of the patient’s sleep patterns, which included time to takes to fall asleep, nighttime awakening, and the frequency of nighttime restlessness. For the physical functioning, daily activities, and sleep domains, less than 1% of respondents noted that they could not recall their impression of change.

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In the study, patients were on average 16 years of age, and were taking a median of 4 antiseizure medications (ASMs), regardless of syndrome diagnosis. For the 85% of patients who reported improvements in seizure frequency, at least 50% improvement was seen in various nonseizure-related outcomes, including cognition (80%), language (78%), and emotional (67%) domains. In patients with no clear change or worsening in seizure frequency, 56% (24 of 43) and 54% (15 of 28) of respondents, respectively, reported improvements in their cognition domain.

"This survey was not intended to assess clinical outcomes in patients with LGS or DS but provides valuable information from a caregiver perspective that could be used to design studies to evaluate these outcomes more rigorously, with fit-for-purpose clinical outcome assessment measures,” Perry et al wrote. "Our findings also underscore the need for more research to develop sensitive clinical outcome measures that reflect caregiver priorities and assess meaningful change for patients with severe developmental and epileptic encephalopathies such as LGS and DS."

In terms of caregiver burden, 71% of caregivers responded that treatment with CBD for their patient improved at least 1 aspect of their burden. Family stress level and the ability to get a good night’s sleep, reported by 53% and 49% of caregivers, respectively, were the most frequent positive changes documented. Aside from seizure reduction, nonseizure-related reasons reported by caregivers to continue treatment included improved alertness (50%), cognition (43%), sleep (30%), and language/communication (30%).

REFERENCE
1. Berg AT. Dixon-Salazar T, Meskis MA, Danese SR, Minh N, Perry MS. Caregiver-reported outcomes with real-world use of cannabidiol in Lennox-Gastaut syndrome and Dravet syndrome from the BECOME survey. Epilepsy Research. 2024;200:107280. doi:10.1016/j.eplepsyres.2023.107280