. 2023 May 5;110:78-85.
doi: 10.1016/j.seizure.2023.05.003.
- PMID: 37331197
- DOI: 10.1016/j.seizure.2023.05.003
Free article
Abstract
Purpose: This retrospective chart review study (GWEP20052) evaluated plant-derived highly purified cannabidiol (CBD; Epidyolex®; 100 mg/mL oral solution) use without clobazam as add-on therapy in patients aged ≥2 years with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) enrolled in a European Early Access Program.
Methods: Data were extracted from patient charts covering a period starting 3 months before CBD treatment and concluding after 12 months of CBD treatment, or sooner if a patient discontinued CBD or started clobazam.
Results: Of 114 enrolled patients, data were available for 107 (92 LGS, 15 DS) who received CBD without clobazam for ≥3 months. Mean age: 14.5 (LGS) and 10.5 (DS) years; female: 44% (LGS) and 67% (DS). Mean time-averaged CBD dose: 13.54 (LGS) and 11.56 (DS) mg/kg/day. Median change from baseline in seizure frequency per 28 days over 3-month intervals varied from -6.2% to -20.9% for LGS and 0% to -16.7% for DS. Achievement of ≥50% reduction in drop (LGS) or convulsive (DS) seizures at 3 and 12 months: LGS, 19% (n = 69) and 30% (n = 53); DS, 21% (n = 14) and 13% (n = 8). Retention on CBD without clobazam (enrolled set): 94%, 80%, 69%, and 63% at 3, 6, 9, and 12 months. Adverse event (AE) incidence was 31%, most commonly somnolence, seizure, diarrhea, and decreased appetite. Two patients discontinued CBD owing to AEs, and four patients with LGS experienced elevated liver enzymes.
Conclusion: Results support favorable effectiveness and retention of CBD without concomitant clobazam for up to 12 months in clinical practice.
Keywords: Cannabidiol, Clobazam, Dravet syndrome, Lennox-Gastaut syndrome, Real-world evidence, Seizure
Copyright © 2023. Published by Elsevier Ltd.
Conflict of interest statement
Declaration of Competing Interest All authors met the ICMJE authorship criteria and had full access to relevant data. Neither honoraria nor payments were made for authorship or participation as a study investigator. The authors have stated the following potential conflicts of interest: RN is the Chair of the Scientific Committee of the French National Rare Diseases Database, has received consulting fees from Biocodex, UCB, Jazz Pharmaceuticals, Inc., Orion, Roche, and Takeda, and payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Biocodex, UCB, Jazz Pharmaceuticals, Inc., Nutricia, and Zogenix. AA is a coordinator of the European Reference Network for Rare and Complex Epilepsies, has received consulting fees from Eisai, GW Pharmaceuticals (now part of Jazz Pharmaceuticals, Inc.), Orion Pharma, UCB, and Zogenix, payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Biocodex, Eisai, Takeda, UCB, and Zogenix, and has participated on a Data Safety Monitoring board for UCB. SA has received payment to his institution for his role as Deputy Editor for Epilepsia and has received consulting fees from GRIN Therapeutics, Jazz Pharmaceuticals, Inc., Neuraxpharm, Nutricia, Orion, Supernus, Takeda, Vitaflo, and Xenon, and received payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Biocodex, BioMarin Pharmaceutical, Neuraxpharm, UCB, and Zogenix. CN and DF are full-time employees of Jazz Pharmaceuticals, Inc. who, in the course of their employment, receive stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals, plc. PP has received support for attending meetings and/or travel from Jazz Pharmaceuticals, Inc. VS has a leadership or fiduciary role at the Sociedad Española de Neuropediatria, has received payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Eisai, Jazz Pharmaceuticals, Inc., and UCB and support for attending meetings and/or travel from Jazz Pharmaceuticals, Inc., and has participated in Data Safety Monitoring and/or advisory board(s) for UCB. VV has received consulting fees and payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Angelini Pharma, Bial, Eisai, Jazz Pharmaceuticals, Inc., Novartis, Takeda, and UCB. JHC is the Chair of the Medical Advisory Board, Dravet UK and has received consulting fees from GW Pharmaceuticals (now part of Jazz Pharmaceuticals, Inc.), Takeda, UCB, and Zogenix, and payment or honoraria for lectures, presentations, speakers’ bureaus, manuscript writing or educational events from Biocodex, UCB, and Zogenix. All other authors confirmed that they had no interests which might be perceived as posing a conflict or bias.
