In a related study, Michael Oldham, MD, MPH, of the University of Louisville, and colleagues reported on the long-term efficacy of Epidiolex in a small patient population enrolled in the Expanded Access program at the University of California San Francisco. The program enrolled 25 patients (52% female) aged 1 to 17 years between January 2014 and December 2014. Twenty-eight percent of patients had Dravet syndrome, and epilepsy with myoclonic absences (EMA), CDKL5, and Lennox-Gastaut syndrome accounted for 16% each. Therapy period and treatment amounts followed the same protocols as in Devinsky O et al. Median percent reduction in seizure frequency was assessed at 3 and 12 months, with treatment response considered ≥50% seizure reduction.

At 3-month follow-up, 8 patients (32%) had responded to CBD; 3 were seizure-free and 5 had a ≥50% seizure reduction. At 12-month follow-up, 10 patients (40%) had a ≥50% seizure reduction; 1 patient remained seizure-free. Twelve patients discontinued CBD due to lack of efficacy, and 1 patient due to increase in seizure frequency related to CBD.

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Overall, CBD therapy was associated with a greater than 50% reduction in seizure frequency in more than one-third of patients at 3-months. This was maintained by 40% of patients for the 12-month duration, suggesting that “CBD can be effective in controlling seizures,” Dr. Oldham said in a statement.

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  1. Devinsky O, Thiele E, Laux L, et al. Abstract 3.397. Efficacy and Safety of Epidiolex (Cannabidiol) in Children and Young Adults with Treatment-Resistant Epilepsy: Update from the Expanded Access Program. Presented at: American Epilepsy Society Annual Meeting; Dec. 4-8, 2015; Philadelphia.
  2. Oldham M, Sullivan J, Singhal N, Tilton N, Cilio M. Abstract 2.296. Long-term efficacy and tolerability of add-on cannabidiol for drug-resistant pediatric epilepsies. Presented at: American Epilepsy Society Annual Meeting; Dec. 4-8, 2015; Philadelphia.