RA double-blind, placebo-controlled trial found that children with Dravet syndrome had fewer seizures after taking a daily oral solution of the cannabis compound cannabidiol, which does not have the psychoactive properties of marijuana. Over a 14-week treatment with cannabidiol, convulsive seizures dropped from a monthly average of 12.4 to 5.9. In comparison, seizures in the placebo group decreased from a monthly average of 14.9 to 14.1. During the study, seizures stopped completely in 5% of patients taking cannabidiol.
"Seizures in Dravet syndrome are extremely difficult to control and they can be deadly," says study co-author Dr Linda Laux, from Stanley Manne Children's Research Institute at Ann & Robert H Lurie Children's Hospital of Chicago. "Our results are encouraging, especially considering that we don't have any antiepileptic drugs approved for Dravet syndrome in the US."
The study included 120 children and young adults with Dravet syndrome and drug-resistant seizures. They were randomly assigned to receive either cannabidiol or a placebo, in addition to standard antiepileptic treatment.
Adverse events were reported in 93% of the patients taking cannabidiol, compared to 75% of the patients in the placebo group. The most common side effects were drowsiness, diarrhea and decreased appetite.
"We will need more data to determine the long-term efficacy and safety of cannabidiol for Dravet syndrome," says Laux, who is the medical director of the Comprehensive Epilepsy Centre at Lurie Children's and assistant professor of paediatrics at Northwestern University Feinberg School of Medicine.
Abstract
Background: The Dravet syndrome is a complex childhood epilepsy disorder that is associated with drug-resistant seizures and a high mortality rate. We studied cannabidiol for the treatment of drug-resistant seizures in the Dravet syndrome.
Methods: In this double-blind, placebo-controlled trial, we randomly assigned 120 children and young adults with the Dravet syndrome and drug-resistant seizures to receive either cannabidiol oral solution at a dose of 20 mg per kilogram of body weight per day or placebo, in addition to standard antiepileptic treatment. The primary end point was the change in convulsive-seizure frequency over a 14-week treatment period, as compared with a 4-week baseline period.
Results: The median frequency of convulsive seizures per month decreased from 12.4 to 5.9 with cannabidiol, as compared with a decrease from 14.9 to 14.1 with placebo (adjusted median difference between the cannabidiol group and the placebo group in change in seizure frequency, −22.8 percentage points; 95% confidence interval [CI], −41.1 to −5.4; P=0.01). The percentage of patients who had at least a 50% reduction in convulsive-seizure frequency was 43% with cannabidiol and 27% with placebo (odds ratio, 2.00; 95% CI, 0.93 to 4.30; P=0.08). The patient’s overall condition improved by at least one category on the seven-category Caregiver Global Impression of Change scale in 62% of the cannabidiol group as compared with 34% of the placebo group (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. The percentage of patients who became seizure-free was 5% with cannabidiol and 0% with placebo (P=0.08). Adverse events that occurred more frequently in the cannabidiol group than in the placebo group included diarrhea, vomiting, fatigue, pyrexia, somnolence, and abnormal results on liver-function tests. There were more withdrawals from the trial in the cannabidiol group.
Conclusions: Among patients with the Dravet syndrome, cannabidiol resulted in a greater reduction in convulsive-seizure frequency than placebo and was associated with higher rates of adverse events.
Authors
Orrin Devinsky, J Helen Cross, Linda Laux, Eric Marsh, Ian Miller, Rima Nabbout, Ingrid E Scheffer, Elizabeth A. Thiele, Stephen Wright
[link url="https://www.luriechildrens.org/en-us/news-events/Pages/cannabidiol_reduces_seizures_in_childern_with_severe_epilepsy_466.aspx?utm_medium=lc.org&utm_source=homepage&utm_campaign=cannibidiol-dravet&utm_term=#REF!&utm_content=research"]Ann & Robert H Lurie Children’s Hospital of Chicago material[/link]
[link url="http://www.nejm.org/doi/10.1056/NEJMoa1611618"]New England Journal of Medicine abstract[/link]