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Published on: December 29, 2021
Effectiveness and tolerability of cannabidiol in paediatric epilepsy: a one-year multisite prospective study
Andreas Halman1, Jeremy L Freeman2, Michael Fahey3
1School of Population and Global Health, University of Melbourne, Melbourne, VIC, Australia.
Insights
Cannabidiol shows promise for paediatric epilepsy, with many patients experiencing reduced seizures and hospitalizations. However, high discontinuation rates due to ineffectiveness or side effects necessitate further research for optimal patient selection.
Area of Science:
- Neurology
- Pharmacology
- Cannabinoid Research
Background:
- Epilepsy impacts millions globally, with drug-resistant forms posing significant challenges.
- Cannabidiol (CBD), a non-psychoactive cannabis compound, presents potential therapeutic benefits for paediatric epilepsy.
- Existing evidence on CBD efficacy in paediatric epilepsy is limited, necessitating further investigation.
Purpose of the Study:
- To evaluate the effectiveness and safety of purified cannabidiol in paediatric epilepsy patients.
- To assess changes in seizure frequency, hospitalizations, and overall epilepsy severity.
- To identify factors influencing treatment discontinuation and patient response.
Main Methods:
- A multi-site, open-label, observational study involving 103 paediatric epilepsy patients.
- Patients received purified cannabidiol through a compassionate access program.
- Data collected at baseline, 3, and 12 months included seizure frequency, hospitalizations, medication use, adverse events, and validated epilepsy severity scales (Global Assessment of Severity of Epilepsy, CGI-I).
Main Results:
- 46% of patients discontinued treatment by 12 months, primarily due to lack of effectiveness (31%) or adverse events (7%).
- Among continuing patients, 40% showed significant improvement (CGI-I 'much improved' or higher) at 12 months.
- Sustained reductions were observed in epilepsy severity, seizure frequency, medication use, status epilepticus, emergency visits, and hospitalizations.
- Adverse events were reported by 31% of patients, mostly mild to moderate.
Conclusions:
- Cannabidiol treatment demonstrated sustained improvements in multiple epilepsy-related measures in paediatric patients.
- High discontinuation rates underscore the need for further research to identify patient subgroups most likely to benefit from CBD.
- Cannabidiol shows potential as an adjunctive therapy for paediatric epilepsy, but careful patient selection is crucial.
Abstract:
Epilepsy affects ∼50 million people worldwide, with drug-resistant forms occurring in over a third of cases and resulting in major health and quality-of-life burdens. Cannabidiol, a non-psychoactive cannabis compound, shows promise in paediatric epilepsy, but evidence remains limited for its use. This multi-site open-label observational study in Australia involved 103 paediatric epilepsy patients receiving purified cannabidiol via a compassionate access scheme. Clinicians provided data at baseline, three and twelve months, including seizure frequency, hospitalisations, medication use and adverse events. Epilepsy severity was assessed using the Global Assessment of Severity of Epilepsy instrument and overall improvement was measured with the Clinical Global Impression Improvement (CGI-I) scale. A total of 46% of patients who commenced treatment discontinued before twelve months, mainly due to lack of effectiveness (n = 31) and adverse events (n = 7). Among those continuing, 40% were rated at least "much improved" on the CGI-I scale at 12 months with sustained reductions in epilepsy severity, seizures, medication use, status epilepticus, emergency visits and hospitalisations. Adverse events were reported by 31%, mostly mild to moderate. In conclusion, cannabidiol treatment was associated with sustained improvements across multiple measures; however, frequent discontinuations highlight the need for further research to better identify patients most likely to respond.
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