Long‐term safety and treatment effects of cannabidiol in children and adults with treatment‐resistant epilepsies: Expanded access program results

• DOI: 10.1111/epi.14477
• Publication Date: 2018-07-12T08:03:47Z
• Authors: Jerzy P. Szaflarski; Elizabeth Martina...; Anne M. Comi; Anup D. Patel; Charuta Joshi; Daniel Checketts; Jules C. Beal; Linda C. Laux; Lisa M. De Boer; Matthew H. Wong; Merrick Lopez; Orrin Devinsky; Paul D. Lyons; Pilar Pichon Zentil; Robert Wechsler
• Keywords: Concomitant; Interim analysis

Summary Objective Since 2014, cannabidiol ( CBD ) has been administered to patients with treatment‐resistant epilepsies TREs in an ongoing expanded‐access program EAP ). We report interim results on the safety and efficacy of treated through December 2016. Methods Twenty‐five US ‐based sites enrolling TRE taking stable doses antiepileptic drugs AED s) at baseline were included. During 4‐week period, parents/caregivers kept diaries all countable seizure types. Patients received oral starting 2‐10 mg/kg/d, titrated a maximum dose 25‐50 mg/kg/d. Patient visits every 2‐4 weeks 16 2‐12 thereafter. Efficacy endpoints included percentage change from median monthly convulsive total frequency, ≥50%, ≥75%, 100% reductions seizures vs baseline. Data analyzed descriptively for analysis set using last‐observation‐carried‐forward method account missing data. Adverse events AE documented each visit. Results Of 607 dataset, 146 (24%) withdrew; most common reasons lack (89 [15%]) s (32 [5%]). Mean age was 13 years (range, 0.4‐62). Median number concomitant 3 0‐10). 25 mg/kg/d; treatment duration 48 weeks. Add‐on reduced by 51% 48% 12 weeks; similar 96 Proportion 52%, 31%, 11%, respectively, weeks, rates generally well tolerated; diarrhea (29%) somnolence (22%). Significance this support previous observational clinical trial data showing that add‐on may be efficacious long‐term option .