Long-Term Study Finds CBD Reduces Seizures and Improves Daily Functioning in Children With Drug-Resistant Epilepsy

The prospective trial, published in the European Journal of Neurology, followed 103 pediatric patients treated at a tertiary center over a two-year period. Nearly half had conditions approved for CBD treatment such as Dravet syndrome, Lennox–Gastaut syndrome, or tuberous sclerosis, while the majority used it off-label for other forms of epilepsy. The mean age was 11.2 years. After one month of treatment, 54% of caregivers reported reduced seizure frequency and duration, and 62% noted an overall positive impression. By months two through six, nearly half of participants maintained seizure improvements, with additional gains in communication (60%), alertness (54%), and motor skills (44%).

The long-term data showed strong treatment adherence: retention rates were 97% at one month, 90% at two months, 82% at six months, 66% at a year, and 55% at 18 months. Notably, off-label use, male gender, and older age at initiation were linked to higher retention rates.

While side effects such as appetite changes, sleep disturbances, and behavioral issues were reported, major adverse events were uncommon. Researchers emphasized the importance of caregiver-reported outcomes, noting that improvements in communication, alertness, and behavior were as meaningful to families as seizure reduction itself.

The authors conclude:

Our study provides valuable insights into the real-world data on the role of CBD in the treatment of pediatric drug-resistant epilepsies, reinforcing its potential beyond currently approved indications, highlighting both the efficacy and possible factors that influence its long-term retention. CBD demonstrated good tolerability, with few major side effects. Notably, its therapeutic effects, particularly in seizure and non-seizure outcomes, may take up to 6 months to manifest, emphasizing the importance of sustained treatment before considering discontinuation. Our findings also emphasize the critical role of caregivers’ involvement in treatment evaluation, especially for pediatric patients with intellectual disability. By integrating parental input, clinicians can better assess a child’s response to CBD beyond seizure control, capturing more comprehensive improvements in emotional and behavioral functioning. As the role of CBD in managing drug-resistant epilepsy continues to evolve, our study contributes to a deeper understanding of how to optimize treatment strategies, ensuring that patients receive the full benefit of this promising therapy.