Please use this identifier to cite or link to this item: http://ahro.austin.org.au/austinjspui/handle/1/16669
Title: Trial of cannabidiol for drug-resistant seizures in the dravet syndrome
Authors: Devinsky, Orrin;Cross, J Helen;Laux, Linda;Marsh, Eric;Miller, Ian;Nabbout, Rima;Scheffer, Ingrid E;Thiele, Elizabeth A;Stephen, Wright;Cannabidiol in Dravet Syndrome Study Group
Issue Date: 25-May-2017
EDate: 2017-05-25
Citation: New England Journal of Medicine 2017; 376: 2011-2020
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. (Funded by GW Pharmaceuticals; ClinicalTrials.gov number, NCT02091375 .).
URI: http://ahro.austin.org.au/austinjspui/handle/1/16669
DOI: 10.1056/NEJMoa1611618
ORCID: 0000-0002-2311-2174
PubMed URL: https://www.ncbi.nlm.nih.gov/pubmed/28538134
Type: Journal Article
Subjects: Anticonvulsants
Epilepsies, Myoclonic
Type of Clinical Study or Trial: Randomized Controlled Clinical Trial/Controlled Clinical Trial
Appears in Collections:Journal articles

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