Nonseizure Outcomes With Cannabidiol (CBD) in Pediatric Versus Adult Patients With Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS): Subgroup Analysis of BECOME, a Caregiver Survey
Tracy Dixon-Salazar1, Anne T. Berg2, Mary Anne Meskis3, Sherry R. Danese4, Timothy B. Saurer5, Ngoc Minh D. Le5, M. Scott Perry6
1Lennox-Gastaut Syndrome (LGS) Foundation, 2Northwestern University Feinberg School of Medicine, 3Dravet Syndrome Foundation, 4Outcomes Insights, 5Jazz Pharmaceuticals, 6Cook Children’s Medical Center
Objective:
To characterize and quantify nonseizure behavioral and cognitive outcomes in pediatric (<18 years) vs adult (≥18 years) patients from the cross-sectional caregiver survey BECOME (BEhavior, COgnition, and More with Epidiolex®).
Background:
A substantial proportion of caregivers reported improvements in seizure and nonseizure outcomes in the primary analysis of the survey.
Design/Methods:
US-based caregivers (N=498) of patients with LGS (80%) or DS (20%) who received ≥3 months of CBD treatment (Epidiolex®, 100 mg/mL oral solution) compared the past month with the period before CBD initiation. The online survey included multiple-choice and rank-order questions; changes were rated using a symmetrical 7-point Likert scale (from worsening to improvement).
Results:
Mean (standard deviation) age of patients: 16 (11) years; median (IQR) CBD dose: 14 mg/kg/d (8–19 mg/kg/d); median concomitant ASMs: 4. A similar proportion of caregivers of pediatric (n=315) and adult (n=183) patients reported any improvement (possible, minor, or definite) in ≥1 question of the following domains: alertness, cognition, and executive function (87% and 81%) and emotional and social function (82% and 80%). A numerically greater proportion of caregivers of pediatric versus adult patients reported any improvement in ≥1 question of the language and communication (verbal patients: 81% versus 63%; nonverbal patients: 85% versus 68%), physical functioning (53% versus 33%), sleep (53% versus 48%), and daily activities (56% versus 44%) domains. Rates of worsening varied by behavior and ranged from 0% to 20% of respondents. In pediatric patients, the most common improvements were in the ability to learn new things (76%) and, in nonverbal patients, looking up or smiling at mention of their name (76%). For adult patients, the most common improvement was in alertness (70%).
Conclusions:
A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in outcomes beyond seizure control since initiating CBD treatment.