Cannabidiol(Epidiolex,CBD) is a relatively new antiseizure medication that was FDA approved in 2018 for seizures with Lennox-Gastaut-syndrome(LGS), Dravet-syndrome or tuberous sclerosis. CBD continues to be studied in other epilepsies, particularly genetic epilepsies.

We performed a retrospective review of patients prescribed CBD younger than 19 who follow with Children’s Hospital in Omaha, Nebraska. We extracted demographic and clinical information from the medical records. Epilepsy syndromes and etiologies were recorded and classified per the 2017 ILAE guidelines. Records of CBD dose along with start and end of therapy were recorded when available. If discontinued, provider notes were reviewed to determine the reasoning. Given provider variability of notes, we categorized efficacy based on seizure frequency as follows: not improved, mildly improved(<50% reduction), or greatly improved(>50% reduction). The efficacy of CBD therapy was compared between genetic and not genetic etiologies.

A total of 32 patients were included in the study. Following CBD therapy in patients identified to have genetic etiology of epilepsy, 3 patient(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 patients(31.6%)had no improvement, 1(5.2%)had mild improvement, and 12(63.1%)had great improvement(Table 1). Furthermore, among the non-genetic, 6 patients 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%)of the patients 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).

Cannabidiol appears to be effective for the treatment of  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.