Among 78 children and young adults with refractory epilepsy, 31% achieved 50% or greater seizure reduction at 3 months, with Dravet and Lennox-Gastaut patients responding best (70%) and clobazam co-medication improving outcomes.
Pediatric neurologists, epileptologists, and families of children with refractory epilepsy.
70% of Dravet/LGS patients achieved 50%+ seizure reduction at 3 months
What the researchers found
At 3 months, 31.4% of patients achieved 50% or greater seizure reduction and 68.6% showed some improvement. Dravet and Lennox-Gastaut patients had much higher response rates (70% achieving 50% reduction) compared to other epilepsies (22%). Clobazam co-medication increased seizure reduction. Response rates declined over time: 20% maintained 50% reduction at 24 months.
Why it matters
While CBD is approved for Dravet and Lennox-Gastaut syndromes, this study shows it may also benefit children with other severe epilepsies, though the waning response over time needs monitoring.
The numbers in context
Patients: 78. With seizure registration: 51. 50% reduction at 3 months: 31.4%. At 6 months: 31.1%. At 12 months: 28.1%. At 24 months: 20.0%. Dravet/LGS at 3 months: 70%. Other epilepsies: 22%. Any reduction at 3 months: 68.6%.
How the study worked
Retrospective cohort study of 78 patients treated with off-label cannabidiol at the Filadelfia Epilepsy Hospital, Denmark from 2016-2019. Assessed seizure frequency registration or perceived effect over up to 24 months.
What this study cannot tell us
Retrospective, uncontrolled design. Off-label use with varying doses. Only 51 of 78 patients had seizure frequency registration. No placebo comparison.
How to read the evidence
Real-world clinical data with up to 24 months follow-up, but uncontrolled retrospective design.
When this study was published
Published in 2021 with data from 2016-2019.
The bigger picture
The declining response over time (from 31% to 20% achieving 50% reduction) suggests tolerance may develop, reinforcing the need for ongoing evaluation and willingness to discontinue if benefits fade.
Questions still open
- Is the declining response over time due to tolerance or natural disease progression? Would higher CBD doses maintain efficacy? What factors predict which non-Dravet/LGS patients respond?
Common questions
Does CBD work for epilepsies other than Dravet and Lennox-Gastaut?
Does CBD effectiveness last over time?
Read the original research
Cannabidiol treatment of severe refractory epilepsy in children and young adults.
Danish medical journal, 68(5)
Citation
Zilmer, Monica; Olofsson, Kern. (2021). Cannabidiol treatment of severe refractory epilepsy in children and young adults.. Danish medical journal, 68(5).
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