Abstracts

Rationale: Cannabidiol (CBD) is a relatively new antiseizure medication that was FDA approved in 2018 for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome and in 2020 for tuberous sclerosis. CBD continues to be studied in other epilepsies, particularly genetic epilepsies. Further data continues to be gathered in cannabidiol's real world efficacy for seizures. We present a retrospective review of outcomes of pediatric patients who were prescribed CBD for epilepsy at a pediatric tertiary care center.

Methods: We performed a retrospective review of patients younger than 19 years of age who were prescribed CBD for epilepsy at CHMC in Omaha, Nebraska. Of the patients included in the study, we extracted demographic and clinical information from the medical records. Epilepsy syndromes and etiologies were recorded when available and classified per the 2017 ILAE guidelines. Dose of CBD used along with the date of initiating and discontinuing the therapy were recorded. If CBD was discontinued, provider notes were reviewed to determine the reasoning. We categorized efficacy of CBD therapy based on seizure frequency as follows: not improved (same seizure frequency), mildly improved (when seizures were mildly or somewhat decreased, or if numbers provided, < 50% reduction in seizures), or greatly improved (when seizures were phrased as significantly improved, or if numbers provided, >50% reduction in seizures). The efficacy of CBD therapy was compared between genetic and not genetic etiologies, and outcomes were statistically analyzed via two-tailed t-test.

Results: A total of 32 patients were included in the study. The mean age (± SD) of patients at the time of initiating CBD therapy was 9.0 ± 5.5 years, and 15 (46.9%) were female. Based on reasons for prescribing CBD, 28 were for LGS, 1 was for Dravet syndrome, and 3 were initially LGS but then later found to have different epilepsy syndromes with genetic etiology. The mean daily weight-based dose of CBD given was 17.6 ± 9.5 mg/kg/day. Following CBD therapy in patients identified to have genetic etiology of epilepsy, 3 (23%) had no improvement, 4 (30.7%) had mild improvement, and 6 (46.1%) had great improvement (Table 1). In patients without a genetic etiology, 6 (31.6%) had no improvement, 1 (5.2%) had mild improvement, and 12 (63.1%) had great improvement (Table 1). Furthermore, among non-genetic who did not have any improvement 5 (83.3%) were structural and 1 (16.7%) was unknown. Among the 12 who had great improvement, 6 (50%) were unknown, 5 (41.67%) were structural and 1 (8.3%) was metabolic. CBD was discontinued in 6 (18.8%) after a mean of 1.3 (range, 0.4-2.5) years of therapy, with reasons being the medication not being effective (n=2), increased appetite (1), agitation/irritability (1), vomiting (1), and change in diagnosis (1).

Conclusions: Cannabidiol appears to be effective for the treatment of LGS in pediatric patients with both genetic and other etiologies of epilepsy. The majority of patients with non-genetic causes of LGS who did not improve with CBD had a structural etiology for their epilepsy. Therapeutic doses of CBD seemed to be well tolerated with minimal side effects in the pediatric population.

Funding: None