Long-Term Evaluation of Cannabidiol in Pediatric Drug-Resistant Epilepsy: A Real-Time Single-Center Retrospective Study
- Author:
Jong Ho CHA
1
;
Hyeryung KIM
;
Young Ho KIM
;
Seungbok LEE
;
Soo Yeon KIM
;
Byung Chan LIM
;
Jong-Hee CHAE
;
Ki Joong KIM
;
Woo Joong KIM
Author Information
- Publication Type:Original article
- From: Annals of Child Neurology 2026;34(1):66-74
- CountryRepublic of Korea
- Language:English
-
Abstract:
Purpose:Cannabidiol (CBD) is a promising treatment option for pediatric drug-resistant epilepsy (DRE). The aim of this study was to assess the tolerability and safety of CBD in a single-center retrospective cohort based on real-world clinical experience.
Methods:This study included 71 pediatric patients (median age, 8.9 years; interquartile range [IQR], 6.2 to 14.0) with Lennox–Gastaut syndrome who received purified CBD (Epidiolex, GW Pharmaceuticals) between March 2019 and July 2024. All patients had previously failed treatment with more than five anti-seizure medications (ASMs). Responder rate (≥50% seizure frequency reduction), retention rate, adverse effects (AEs), and predictors of favorable treatment response were analyzed over a median follow-up of 21.3 months (IQR, 2.8 to 38.5).
Results:The initial responder rate during the first 3 months was 45.1%, which increased to 70.8% at 18 months and 63.0% at 24 months. The retention rate at 24 months was 52.4% (33/71). Seven patients (9.9%) achieved seizure freedom beyond 24 months of CBD therapy, and five of these patients were able to reduce their concomitant ASM burden. AEs were observed in 39.4% (28/71) of patients, with the most frequent being somnolence (20 cases) and increased seizure frequency (six cases); 92.9% of AEs occurred within the first 3 months of treatment. No serious AEs requiring treatment discontinuation were identified.
Conclusion:In this real-world study, CBD demonstrated potential as an adjunctive therapy with manageable AEs. These findings highlight that CBD can reduce seizure frequency while maintaining tolerability in pediatric patients with DRE.
