Assessing Real World Efficacy, Safety, and 18-Month Retention Rates of Cannabidiol in Individuals With Drug Resistant Epilepsies
Nicole Chemaly, Mathieu Kuchenbuch, Emma Losito, Anna Kaminska, Delphine Coste-Zeitoun, Giulia Barcia, Isabelle Desguerre, Marie Hully, Rima Nabbout
Department of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France
Université de Paris Cité, Paris, France
Laboratory of Translational Research for Neurological Disorders, INSERM MR1163, Imagine Institute, Paris, France
Affiliationer
Background
Highly purified Cannabidiol (CBD, epidiolex) is approved for treating Lennox–Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis (TSC), with off-label use in drug-resistant epilepsies. The aim of this study is to evaluate efficacy, safety, and long-term retention of CBD in a large pediatric population.
Methods
We prospectively included all consecutive patients initiating CBD for drug-resistant epilepsy at our pediatric tertiary center from 2019 to 2021. Efficacy and safety were assessed via parental questionnaires on seizure and non-seizure outcomes at 1, 2, and 6 months, comparing each item to prior clinic visits. Retention rate was evaluated at 18 months.
Results
We included 103 patients (mean age 11.2 years), with 47% receiving CBD for label-approved syndromes (23 DS, 15 LGS, 10 TSC) and 53% off-label. At 1 month, 54% of caregivers reported reduced seizure frequency and duration, with a positive overall impression in 62%. By months 2–6, 48% maintained seizure improvement, alongside sustained gains in communication (60%), alertness (54%), and motor skills (44%). Retention rates were 97% at 1 month, 90% at 2 months, 82% at 6 months, 66% at 12 months, and 55% at 18 months. Off-label use (p = 0.02), male gender (p = 0.049), and older age at initiation (p = 0.03) were associated with higher retention.
Conclusions
CBD reduced seizures and improved non seizures’ morbidities in drug-resistant epilepsy. Strong 18-month retention rate, including off-label use, highlights efficacy and tolerability, underscoring the need for further real-world data in various rare epilepsy syndromes beyond its market approval.
