Abstract
What is this summary about?
Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare forms of epilepsy that start in childhood and cause life-long disability. People with LGS and DS experience many seizure types and nonseizure-related problems with cognition (ability to think and understand), learning, behaviour and communication. People with LGS and DS depend on caregivers to assist with daily activities. Although many medications are available to treat seizures associated with LGS and DS, the effect on nonseizure outcomes is unclear. A highly purified (defined as a substance that has undergone a special process to remove impurities) oral (by mouth) solution of cannabidiol (CBD; Epidiolex®) from plants is approved by the United States (US) Food and Drug Administration (FDA) for the treatment of seizures associated with LGS, DS and tuberous sclerosis complex in people who are at least 1 year old. In clinical studies, treatment with CBD reduced the number of seizures.
Common side effects (secondary, typically unintended effects of the medication) were increased liver enzyme levels, sleepiness, decreased appetite, diarrhea, fever, vomiting, tiredness, rash, problems with sleep and infections. CBD may also improve nonseizure outcomes, such as cognition, behavior and quality of life. The BEhavior, COgnition and More with Epidiolex® (BECOME) survey was designed to find out whether caregivers of people with LGS or DS taking CBD noticed changes in nonseizure and seizure outcomes.
What are the key takeaways?
Caregivers of people with LGS or DS taking CBD noticed improvements in cognition, language and communication, emotional and social functioning, physical functioning, sleep and ability to do daily activities, as well as a reduction in the number and severity of seizures. People with reduction in seizures were more likely to experience nonseizure benefits, but some people without seizure reduction also experienced improvements in nonseizure outcomes. Caregivers reported that both seizure and nonseizure effects were important when thinking about treatment options.
What were the main conclusions reported by the researchers?
The BECOME survey provides a unique caregiver perspective on nonseizure outcomes that may be affected by CBD. These results support further study of CBD for nonseizure outcomes in people with LGS or DS.
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Financial disclosure
The survey was sponsored by Jazz Pharmaceuticals, Inc (formerly GW Pharmaceuticals companies), Palo Alto, CA, USA. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Writing disclosure
The authors received medical writing support from Ritu Pathak, PhD, graphics/design support from Stefanie Howard, and editorial support from Dena McWain of Ashfield MedComms, an Inizio company, which was funded by Jazz Pharmaceuticals, Inc.
Acknowledgments
The authors would like to thank the participants of this survey for their valuable time and help.
Competing interests disclosure
ATB received speakers fees from BioMarin and advisory panel fees from Biogen, Biohaven and Encoded Therapeutics; TD-S is the Executive Director of the LGS Foundation and a consultant for the Chan Zuckerberg Initiative and Neurelis; MAM is the Executive Director of the Dravet Syndrome Foundation; SRD has nothing to disclose; NMDL was an employee of Jazz Pharmaceuticals, Inc at the time this study was conducted and may hold stock and/or stock options in Jazz Pharmaceuticals, plc; MSP received safety monitoring committee fees from Stoke Therapeutics; consulting fees from BioMarin, Encoded Therapeutics, Greenwich Biosciences (now part of Jazz Pharmaceuticals, Inc), Neurelis, Stoke Therapeutics, Taysha and Zogenix; speakers bureau fees/advisory board fees from Zogenix and Bright Minds; research funds paid to Cook Children's Medical Center from Encoded Therapeutics, Greenwich Biosciences (now part of Jazz Pharmaceuticals, Inc), Marinus, Ovid, Stoke Therapeutics and Zogenix; speakers fees from nobelPharma; and advisory board fees from Eisai. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript apart from those disclosed.