Chapter 7 150 Long-term safety and treatment effects of cannabidiol in children and adults with treatment-resistant epilepsies: Expanded access program results. Setup In January 2014, an expanded access program was initiated to provide cannabidiol to patients with treatment-resistant epilepsy as recent findings from several phase III clinical trials showed that add-on cannabidiol was efficacious for seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. Twenty-five sites in the United States participated and data were prospectively collected both during hospital visits and using patient diaries. The article reports interim results. An institutional review board at each site approved the expanded access program protocols, and the expanded access program was funded by the sponsor, GW Research Ltd, together with the Epilepsy Foundation, the New York State Department of Health and the State of Alabama General Funds. The data collection from the sites was company-initiated and medical writing support was provided by the sponsor. Patients The expanded access program was open to patients with treatment-resistant epilepsy receiving stable doses of antiepileptic drugs for ≥ 4 weeks before enrolment. Of 607 enrolled patients, 580 patients were included in the efficacy analysis population. Patients had a median age of 13.1 years (range, 0.4-62.1). Patients were on a median of three other antiepileptic drugs in addition to cannabidiol at enrolment. Intervention Patients received oral cannabidiol, a cannabinoid binding to cannabinoid receptors CB1 and CB2. Cannabidiol was administered at a gradually increasing dose starting at 2-10 mg/kg/day until tolerability limit or till a maximum dose of 25-50 mg/kg/day was reached. The median CBD dose was 25 mg/kg/day and the median treatment duration was 48 weeks. Outcome The primary endpoint compared the change from 28-day frequency seizure to baseline. In addition, Adverse Events were monitored. At 12 weeks, add-on CBD reduced median monthly convulsive seizures by 51% and total seizures by 48% compared with baseline. The proportion of patients with ≥50%, ≥75%, and 100% reductions in convulsive seizures were 52%, 31%, and 11%, respectively. These reductions in convulsive seizures were similar through 96 weeks. The most common AEs were diarrhea (29%) and somnolence (22%) and 33% of the patients experienced serious AEs.
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