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Long-term Efficacy of Cannabidiol in Children With Epilepsy

New research found over a quarter of children with treatment-refractory epilepsy sustained treatment response to CBD over four years.

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Purified cannabidiol (Epidiolex, CBD): new AED was approved in 2018 for Lennox-Gastaut and Dravet syndrome in children.1 A derivative of marijuana, it does not cause a high. Interim results of add-on CBD support efficacy in treatment resistant epilepsy2 begs the question: What is the long-term efficacy of CBD?

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In a single center, open-label prospective study,3 there were 26 participants refractory to treatment despite an average of two AEDs:

•Mean age 9 years, 50% female•23% with Dravet syndrome•15% with Lennox-Gastaut syndrome

Add-on CBD was titrated to target of 25 mg/kg/day. Lab tests/clinical assessments were taken at regular intervals. Daily seizure frequency was recorded using patient diaries. Mean CBD duration: 21 months.

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Results: 57.7% (n=15) discontinued CBD for lack of efficacy

•Most discontinued within first six months

At two years, only 34.6% (n=9) were still on CBD

>50% reduction in motor seizures (primary efficacy outcome):

•Sustained by 26.9% (n=7) over 2-4 years•11.5% (n=3) became seizure free

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Adverse events were common: 80.8% (n=21) 

Most common:

  • Decreased appetite: 38.4% (n=10)

  • Diarrhea: 34.6% (n=9)

  • Weight loss: 30.7% (n=8)

Serious adverse events in 23.1% (n=6)

  • Status epilepticus: 21% (n=3)

  • Catatonia: 7% (n=2)

  • Hypoalbuminemia: 4% (n=1)

Clinically significant weight loss in 30.7% (n=8)

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Which patients are more likely to respond to CBD?

  • Generalized seizures appear to respond better than focal or epileptic spasms

  • Best response with concomitant benzodiazepines and/or topiramate

  • Further study needed

Adverse events with CBD very common

  • Providers should be aware of rare treatment emergent status epilepticus
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26.9% of children with treatment refractory epilepsy sustained treatment response to CBD over four years

  • Most discontinued CBD due to lack of efficacy

80.8% experienced adverse events

  • Providers should be aware of rare treatment emergent status epileptics with CBD

Determinants of response to CBD need further study

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1. US Food and Drug Administration. FDA approves first drug comprised of an active ingredient derived from marijuana to treat rare, severe forms of epilepsy. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm611046.htm. Accessed January 4, 2018.

2. Szaflarski JP, Bebin EM, Comi AM, et al. Long-term safety and treatment effects of cannabidiol in children and adults with treatment-resistant epilepsies: Expanded access program results. Epilepsia. 2018;59:1540-1548.

3. Sands TT, Rahdari S, Oldham MS, et al. Long-Term Safety, Tolerability, and Efficacy of Cannabidiol in Children with Refractory Epilepsy: Results from an Expanded Access Program in the US. CNS Drugs. 2018 Nov 20 [Epub ahead of print].

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