Abstracts

Rationale: Previous studies have demonstrated that purified cannabidiol (CBD) is a tolerable and effective treatment for childhood-onset epilepsies, Dravet Syndrome and Lennox-Gastaut syndrome (LGS), and for medically refractory epilepsy (MRE). Epidiolex, a plant-derived pharmaceutical formulation of highly purified CBD, became approved by the FDA in June 2018 and available to the market in the fall of 2018. The purpose of this study was to perform an initial analysis of the early effects of Epidiolex since its availability outside clinical trials. Methods: A retrospective chart review was used to analyze the early benefits and side effects of Epidiolex. From our large, multi-site regional epilepsy program, there were 45 patients with intention to treat with Epidiolex. We excluded patients who were on Epidiolex before 2018 from earlier trials. Patients who did not receive the medication, did not yet start, or did not have follow-up appointments were also excluded from the study. Of the 45 patients, 12 patients (27%) had started Epidiolex and had a follow-up since starting the medication.  Results: Of the 12 patients prescribed Epidiolex, 6 (50%) were male and 6 (50%) were female. The median age was 13.5 years (range 3-17 years). 7 patients (58%) were diagnosed with LGS, 1 patient (8%) with Doose Syndrome, 2 (17%) had static encephalopathy, and 2 patients had other diagnoses. The most commonly reported seizure type, experienced by 9 patients (75%), was tonic seizures. Other seizure types reported were generalized tonic-clonic (33%), myoclonic (33%), absence (25%), clonic (17%), drop (17%), and focal (8%). 1 patient (8%) chose to discontinue the medication due to lack of efficacy and difficulty focusing. Of the remaining 11 patients, 6 (55%) reported reduced seizure frequencies, 2 patients (18%) reported shorter duration of episodes, and 2 patients (18%) reported reduced intensity and improved control of their seizures. Other benefits experienced were more regulated bowel movements (9%) and better control of a comorbid movement disorder (9%). 2 patients (18%) reported no benefits or changes since starting Epidiolex. Side effects experienced were lethargy (18%) and slower cognition (9%), and 8 patients (73%) did not report any side effects. The average number of previously used antiepileptic drugs (AEDs) was 4.6. The average number of AEDs used concurrently with Epidiolex was 2.9. Two patients (18%) had taken CBD oil before starting Epidiolex. Conclusions: The most common early benefit of Epidiolex experienced was reduced seizure frequency. Side effects experienced were lethargy and cognitive impairments, although the majority of patients tolerated the medication well and did not report any side effects. Although this study is based on self-reporting and a small sample size, it serves as a pilot study for a larger study to quantify the effects of Epidiolex on quality of life.  Funding: No funding