In a state-funded expanded access program, 45 children with treatment-resistant epilepsy showed sustained seizure reduction and increased seizure-free days over 36 months of CBD treatment.
Pediatric neurologists, families of children with treatment-resistant epilepsy, and epilepsy policymakers.
36 months of sustained seizure reduction; +7.52 seizure-free days
What the researchers found
Over 36 months, 45 children with treatment-resistant epilepsy (various etiologies beyond Dravet/LGS) treated with adjunctive CBD (Epidiolex, up to 50 mg/kg/day) showed statistically significant reductions in seizure frequency at months 3, 6, 12, 18, 24, and 36. Seizure-free days increased by an average of 7.52 days (p<0.001). Higher doses (>25 mg/kg/day) were associated with more adverse events initially, though AE rates decreased over time.
Why it matters
Most CBD epilepsy data comes from Dravet and Lennox-Gastaut syndrome. This study extends the evidence to children with diverse seizure etiologies who failed all other treatments, showing benefit for a broader population.
The numbers in context
45 patients; 36 months; significant seizure reduction at all time points; +7.52 seizure-free days; doses up to 50 mg/kg/day; 12 children had 20 serious AEs, none CBD-related; AE rate decreased over time.
How the study worked
Multicenter expanded access program in Georgia for children aged 1-18 with treatment-resistant epilepsy who had failed available treatments and were ineligible for Dravet/LGS RCTs. CBD titrated to 25 mg/kg/day with optional increase to 50 mg/kg/day. 36-month follow-up.
What this study cannot tell us
No control group (expanded access design); relatively small sample; mixed epilepsy etiologies make subgroup analysis difficult; seizure diaries may have reporting variability; state-specific program.
How to read the evidence
Moderate: 36-month follow-up in an expanded access program, but uncontrolled and relatively small.
When this study was published
Published 2020.
The bigger picture
This is one of the longest follow-up studies of CBD for epilepsy. The sustained benefit over 3 years addresses a key clinical question: does CBD continue working long-term, or does tolerance develop? These data suggest sustained efficacy.
Questions still open
- Which specific epilepsy etiologies respond best to CBD beyond DS and LGS? Can the initial high AE rate at higher doses be managed with slower titration?
Common questions
Does CBD keep working for epilepsy over time?
Does CBD only work for Dravet and Lennox-Gastaut syndrome?
Read the original research
Long-term efficacy and safety of cannabidiol (CBD) in children with treatment-resistant epilepsy: Results from a state-based expanded access program.
Epilepsy & behavior : E&B, 112, 107474
Citation
Park, Yong D; Linder, Daniel F; Pope, Jamie; Flamini, J Robert; Moretz, Katherine; Diamond, Michael P; Long, Sarah A. (2020). Long-term efficacy and safety of cannabidiol (CBD) in children with treatment-resistant epilepsy: Results from a state-based expanded access program.. Epilepsy & behavior : E&B, 112, 107474. https://doi.org/10.1016/j.yebeh.2020.107474
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