Introduction

Drug-resistant epilepsy (DRE) significantly affects the development and quality of life of children and young adults.

Objective

To describe the effectiveness and safety of purified cannabidiol (CBD) in children and young adults with DRE.

Methods

A retrospective analysis of medical records of 139 children and young adults (54.7% female, median age 12.0 years) with DRE treated with purified CBD from 2018-2022 at 5 medical centers in Israel.

Results

The most common diagnosis was Lennox-Gastaut syndrome (37.4%) followed by Dravet syndrome (16.5%) and tuberous sclerosis complex (16.5%). Median purified CBD dose was 12.5 mg/kg (range 2.5-20.0) and median treatment duration was 9.0 months (range 0.5-48.0). Most patients (92.2%) experienced a reduction in seizure frequency following treatment initiation; 41.1% had >50% reduction. Fifty-three patients (38.1%) had positive effects: improved alertness (31.7%), improved speech (10.1%) and achievement of new developmental milestones (2.2%). A multivariate linear model for assessment of predictive factors for seizure reduction demonstrated that patients treated with previous CBD oils, especially those who had >50% seizure reduction on prior treatment, were also more likely to have a reduced frequency of seizures while they were treated with purified CBD (p=0.01, p<0.0001). Development, diagnosis, age, purified CBD dose (0-10 mg/kg/day vs. 10-20 mg/kg/day) and concomitant treatment with clobazam, valproic acid or everolimus did not affect seizure reduction by purified CBD. The most common adverse events were irritability (20.9%) and drowsiness (12.9%).

Conclusion

Purified CBD is well-tolerated and effective in reducing seizure frequency in children and young adults with DRE.