For children and young adult with Dravet syndrome, treatment with cannabidiol led to a greater decrease in convulsive seizure frequency vs. placebo but was associated with more adverse events.
Patients with Dravet syndrome experience drug-resistant seizures and a high mortality rate; there is currently no approved treatment. Cannabidiol, a compound of cannabis, does not contain the psychoactive properties of marijuana. Researchers from Stanley Manne Children’s Research Institute in Chicago evaluated the efficacy of cannabidiol oral solution for the treatment of drug-resistant seizures in Dravet syndrome.
The double-blind, placebo-controlled trial randomized 120 children and young adults to either cannabidiol oral solution 20mg/kg daily or placebo, in addition to their standard antiepileptic treatment. The primary endpoint was the change in convulsive-seizure frequency over 14 weeks vs. the 4-week baseline period.
The data showed a reduction in the median frequency of convulsive seizure per month from 12.4 to 5.9 with cannabidiol vs. a reduction from 14.9 to 14.1 with placebo (adjusted mean difference: -22.8 percentage points, 95% CI: -41.1 to -5.4; P=0.01).
The percentage of patients with ≥50% decrease in convulsive-seizure frequency was 43% with cannabidiol vs. 27% with placebo (odds ratio [OR] 2.00, 95% CI: 0.93 to 4.30; P=0.08). More patients in the cannabidiol group had an improvement in at least 1 category on the 7-category Caregiver Global Impression of Change scale vs. placebo group (62% vs. 34%; P=0.02).
“The frequency of total seizures of all types was significantly reduced with cannabidiol (P=0.03), but there was no significant reduction in nonconvulsive seizures” reported lead author Orrin Devinsky, MD.
Moreover, 5% of patients in the cannabidiol group became seizure-free vs. 0% in the placebo group (P=0.08).
Adverse events, however, were more common in the cannabidiol group vs. placebo group. These included diarrhea, vomiting, fatigue, pyrexia, somnolence, and abnormal liver function tests.
For more information visit nejm.org.