Seizure 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
Timothy B. Saurer1, Anne T. Berg2, Tracy Dixon-Salazar3, Mary Anne Meskis4, Sherry R. Danese5, Ngoc Minh D. Le1, M. Scott Perry6
1Jazz Pharmaceuticals, 2Northwestern University Feinberg School of Medicine, 3Lennox-Gastaut Syndrome (LGS) Foundation, 4Dravet Syndrome Foundation, 5Outcomes Insights, 6Cook Children’s Medical Center
Objective:

To characterize and quantify seizure-related outcomes in pediatric (<18 years) versus 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:

The 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 5- and 7-point Likert scale (from worsening to improvement).

Results:

Mean (standard deviation) patient age: 16 (11) years; median (IQR) CBD dose: 14 mg/kg/d (8–19 mg/kg/d); median concomitant ASMs: 4. Most caregivers of pediatric (n=315) and adult (n=183) patients reported any improvement (possible, definite minor, or definite major) in seizure frequency (84% each) and severity (77% and 75%). Worsening in seizure frequency was reported by 7% and 5% of caregivers of pediatric and adult patients, respectively; 10% and 6% reported any worsening in seizure severity. Caregivers of pediatric and adult patients reported any decrease in the frequency of specific seizure type as follows: convulsive (72% each), drop (71% each), nonconvulsive/nondrop (66% and 69%), and night-time seizures (61% and 63%). Similar proportions of caregivers of pediatric and adult patients reported improvements in seizure-free days/week for ≥1 seizure type (65% and 70%) and seizure freedom in the past month (18% and 15%). Many respondents in both groups reported reductions in rescue medication use (57% each), emergency room visits (56% and 51%), hospitalizations (55% and 50%), and occurrence of seizure-related injuries (48% and 49%).

Conclusions:

A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in seizure-related outcomes with CBD treatment.

10.1212/WNL.0000000000202607