In a real-world study of 108 refractory epilepsy patients, 75% remained on Epidiolex at final evaluation, with 14.5% discontinuing due to adverse effects and 53% able to reduce or stop another seizure medication.
Read this if you or someone you know has refractory epilepsy and is considering or currently taking Epidiolex.
75% retentionProportion of refractory epilepsy patients who continued Epidiolex at final evaluation
What the researchers found
Of 108 patients (mean age 20.3, 52.8% female), 75% continued Epidiolex at final evaluation. The 25th percentile for discontinuation was 19 months. 46.3% experienced at least one adverse effect, but only 14.5% discontinued due to adverse effects. Top reasons for discontinuation were lack of efficacy (37%), increased seizures (22%), worsened behavior (22%), and sedation (22%). Importantly, 53% were able to discontinue or reduce at least one other antiseizure medication. GI complaints and LFT elevations were less common than in clinical trials.
Why it matters
Real-world data often differs from clinical trial results. This study shows Epidiolex performs well in everyday clinical practice, with better tolerability than trials suggested and meaningful reductions in polypharmacy.
The numbers in context
- 108 patients, mean age 20.3 years (range 2-63)
- 52.8% female
- Mean maintenance dose: 15.3 mg/kg/day
- 75% continued treatment at final evaluation
- 25th percentile discontinuation: 19 months
- 14.5% discontinued for adverse effects
- 53% reduced or stopped another ASM
- 47.2% taking concurrent clobazam
How the study worked
Single-center retrospective review of patients with refractory epilepsy taking Epidiolex. Kaplan-Meier analysis for treatment retention. Adverse event documentation and medication change tracking.
Who was studied
108 patients with refractory epilepsy taking Epidiolex (mean age 20.3, range 2-63)
What this study cannot tell us
Single center. Retrospective design. No control group. Treatment retention used as proxy for effectiveness rather than direct seizure outcomes. Clobazam interactions may confound results.
How to read the evidence
Single-center retrospective cohort. Good real-world evidence but limited by lack of control group and single site.
When this study was published
Published in 2023. Provides mature real-world data since Epidiolex FDA approval in 2018.
The bigger picture
Post-marketing real-world evidence is essential for understanding how drugs perform outside controlled trial conditions. Epidiolex's strong retention rate and polypharmacy reduction potential suggest it's earning its place in the epilepsy treatment arsenal.
Questions still open
- Which patient characteristics predict long-term Epidiolex success?
- Can early adverse effect identification prevent unnecessary discontinuation?
- Does Epidiolex retention differ by epilepsy syndrome?
Read the original research
Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex® (cannabidiol) in patients with refractory epilepsy.
Epilepsy & behavior : E&B, 141, 109159
Citation
Georgieva, Darina; Langley, James; Hartkopf, Katherine; Hawk, Lisa; Margolis, Amanda; Struck, Aaron; Felton, Elizabeth; Hsu, David; Gidal, Barry E. (2023). Real-world, long-term evaluation of the tolerability and therapy retention of Epidiolex® (cannabidiol) in patients with refractory epilepsy.. Epilepsy & behavior : E&B, 141, 109159. https://doi.org/10.1016/j.yebeh.2023.109159