(A-109) Effectiveness and Tolerability of Cannabidiol in Children with Drug-Resistant Epilepsy: A Real-world Multicentric Ambispective Study in India

Background: Cannabidiol (CBD) was licensed in 2023, for use in India by CDSCO as an add-on therapy for children with drug-resistant epilepsy. Despite several studies conducted in other countries, the effectiveness data may not be directly applicable to the Indian population due to long treatment lag, preponderance of structural etiology, and availability of its synthetic formulation.

Objectives: To evaluate the effectiveness, and tolerability of CBD as an adjunct therapy in children with DRE in real-world settings.

Methods: This ambispective, observational, multicentric study was conducted at various hospitals in India for a duration of 12 months. It includes clinically diagnosed children over one year of age with drug-resistant epilepsy, specifically those with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and Tuberous sclerosis complex (TSC), who have not achieved seizure control despite treatment with at least two antiseizure medications, either as monotherapy or in combination. The study assessed patients prescribed CBD as an adjunct to usual care and those receiving usual care alone. Only children whose parents or caregivers were ready to consent to participate were included in the study cohort. The primary outcomes were the proportion of patients achieving >50% seizure reduction, and the secondary outcome included adverse events associated with the drug. Data were analyzed using frequency (n), percentages, and mean/median values.

Results: The study cohort comprised 122 children prescribed CBD and 105 patients on usual care with a median age of 5.7 (range: 2-16) years. In the CBD group, a total of 29 (23.8%) experienced a reduction in seizure frequency of >50%, independent of the epilepsy subtype. About 1/3 of patients discontinued therapy within the observation period due to adverse effects or lack of effectiveness. Adverse events were reported in 45 (36.1%) cases. The most common of them were somnolence 18 (40%), diarrhea 12 (26%), decreased appetite 8 (18%), and lethargy 7 (16%). While, in Usual care, only 13 (12%) experienced a reduction in seizure frequency of >50%. Cannabidiol was more effective in combination with clobazam; however, sedation was observed more frequently in these cases.

Conclusions: our study suggested that cannabidiol is usually considered safe and effective in the population. The findings call for long-term efficacy and safety study of CBD in Children with Drug-resistant epilepsy.