Retrospective review finds similar CBD response across pediatric epilepsy…

BACKGROUND: The pharmaceutical formulation of highly purified cannabidiol (CBD; Epidiolex) is approved by the the US Food and Drug Administration for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis in patients one year of age and older. Among the large volume of drug-resistant cases treated at pediatric Level 4 Epilepsy Centers, many patients fall outside of these categories but are still prescribed CBD for off label use. This study sought to characterize the real-world experience with CBD of a large single pediatric comprehensive epilepsy center and to compare response to CBD within and outside of its FDA-approved indications. METHODS: A retrospective chart review was performed of patients with a prescription for CBD at our institution between January 2020 and January 2022, excluding patients who were previously included in our center's 2019 analysis. Patients were not included in the analysis if initially prescribed CBD elsewhere, follow-up was outside our institution, medication was not started, patient expired during the study period, or diagnostic data was not available. Between-group differences were assessed via Wilcoxon rank-sum and Fisher exact tests, as appropriate. RESULTS: Of 223 patients with a CBD prescription during the study period, 144 patients had sufficient data available for review. Of these patients, 81 (56%) carried diagnoses of Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis, and 63 (44%) carried other diagnoses outside of FDA-labeled indications. Median (interquartile range) age at initiation was 10.0 years (5.0, 13.0), and median (interquartile range) number of concurrent anti-seizure medications was 3.0 (2.0, 3.0). There was no significant difference in the proportion of patients treated concurrently with vagus nerve stimulation or ketogenic diet between the two groups. The FDA-labeled (63%) group had a significantly higher proportion of patients with tonic seizures compared to the non-FDA labeled (29%) group (P < 0.001), otherwise there were no significant differences in baseline seizure types. Overall, 53 patients (65%) in the FDA-labeled group and 40 (63%) of the non-FDA labeled group had a documented responder rate at least 50% on CBD (P = 0.9). The most common side effects reported were nausea/diarrhea/gastrointestinal upset (19%), agitation (7.6%), insomnia (4.9%), weight loss (6.3%), and secretions/drooling (2.8%, seen only in patients on concurrent clobazam). CONCLUSIONS: Among our cohort of treatment-resistant pediatric epilepsy, purified CBD was efficacious in a broad range of pediatric epilepsies and was well tolerated as an adjunctive anti-seizure medication. This medication may be considered in pharmacoresistant pediatric epilepsies in conditions beyond its FDA-labeled indications.