Highlights from

AAN 2019

American Academy of Neurology annual meeting

Philadelphia, USA 4-10 May 2019

Cannabidiol effective in children with Dravet syndrome

Cannabidiol (CBD) in a dosage of 10 or 20 mg/kg/day significantly reduced convulsive seizure frequency in children and adolescents with Dravet syndrome (DS). This was the conclusion of GWPCARE, a dose-ranging, multicentre, randomised, double-blind, placebo-controlled trial. The higher dosage gave rise to more adverse events and discontinuations.

DS is an infantile-onset developmental and epileptic encephalopathy which is associated with drug-resistant seizures. In GWPCARE, a highly purified form of CBD was applied, which is on the market in the USA. A total of 199 children and adolescents with DS and drug-resistant seizures participated (mean age 9 years). They took a median of 3 antiepileptic drugs and had discontinued a median 4. Participants were randomised 1:1:1 to CBD 10 or 20 mg/kg/day (CBD10 or CBD20), or placebo as an add-on anticonvulsant therapy. The primary endpoint was the change in the number of convulsive seizure frequency during 14 weeks of treatment compared to a 4-week baseline.

In both active treatment groups, convulsive seizures were reduced more (CBD20: 46%, CBD10: 49%) than placebo (27%; P=0.0299 and P=0.0095). There were also significantly more patients with a ≥50% reduction in convulsive seizures, as well as a higher reduction in total seizures. The incidence of adverse events was similar across all groups: 90% CBD20, 88% CBD10, 89% placebo. However, serious AEs were reported in 25% CBD20, 20% CBD10, and 15% of placebo patients. Discontinuations due to AEs occurred only for CBD20 patients (7%). Elevated transaminases exceeding 3 times the upper limit of normal occurred in 19% CBD20 and 5% CBD10 patients; all elevations resolved. The authors concluded that any dose increase above 10 mg/kg/day should be guided by individual efficacy and safety.

Miller I, et al. AAN 2019.

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