Long‐Term Drop‐Seizure Response Patterns to Cannabidiol According to Genetic Status in Lennox–Gastaut Syndrome
Ji‐Hoon Na, Hee yeon Cho, Hayoon Jeon, Hyunjoo Lee, Young‐Mock LeeABSTRACT
Background
Lennox–Gastaut syndrome (LGS) is a genetically heterogeneous developmental and epileptic encephalopathy in which variability in long‐term cannabidiol (CBD) response remains incompletely understood. We evaluated whether long‐term drop‐seizure response trajectories to CBD differ according to genetic status.
Methods
This single‐center retrospective longitudinal study analyzed systematically collected clinical follow‐up data from 61 patients with LGS treated with pharmaceutical‐grade CBD; 46 patients with ≥ 24 months of follow‐up were included. Patients were classified as pathogenic/likely pathogenic variant‐positive (PV+) or variant‐negative (PV–). Drop‐seizure reduction trajectories were assessed at 3‐month intervals over 24 months.
Results
Drop‐seizure reduction improved rapidly and was sustained over 24 months in both PV+ and PV– groups, with no significant differences between groups at the prespecified comparison time points. Longitudinal response trajectories showed substantial overlap, and sustained response was observed in 76.1% of patients.
Conclusion
Long‐term drop‐seizure response to CBD in LGS was sustained but not stratified by genetic status, supporting a syndrome‐level treatment approach irrespective of genetic findings.