Pediatric Neurology Resident University Hospitals Rainbow Babies and Children's Hospital Cleveland, Ohio
Rationale: Epidiolex (cannabidiol) is an antiepileptic drug approved by the Federal Drug Administration (FDA) in June 2018 for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients who are two years old and older. Many pediatric antiepileptic drugs are prescribed for compassionate use outside the scope of the FDA indication. There is a paucity of studies to date that examine the use, safety and efficacy of the drug beyond its approved use indications. The purpose of this study was to review the scope of use, side effects, efficacy and cognitive effects of Epidiolex in a single tertiary children’s hospital. Additionally, this study sought to identify characteristics of patients for whom there was efficacy for seizures, as well as improvement in cognition, as an unintended side effect. Methods: This was a retrospective chart review of children ages 2-21 years old in the Division of Pediatric Neurology and Epilepsy at Rainbow Babies and Children’s Hospital in Cleveland, Ohio for whom the medication, Epidiolex, was initiated for intractable epilepsy between November 1, 2018 and December 3, 2019. Results: Of the 50 children treated with Epidiolex for intractable epilepsy, 10% had a diagnosis of Dravet syndrome, 32% had Lennox-Gasteaut syndrome, and 58% had “other” diagnoses. Of the “other” diagnoses, 41% had identifiable genetic causes, and 10% had autoimmune/inflammatory etiology. Thirty children (60%) had improvement in seizure frequency, duration, or both. Seven children (14%) had worsening of seizure duration, frequency, or both. The remaining had no change in seizures or data was not available. When comparing those who responded to Epidiolex, to those who did not respond, the responders were more likely to be female (p = 0.0361) and were on fewer prior anti-epileptic drugs (p = 0.0503). A total of 27 patients (54%) experienced at least one adverse effect. The top reported adverse effects were GI upset (10%), somnolence (10%), worsening seizure frequency (14%), behavioral issues (8%), no change in seizures (6%), and transaminitis (4%). Eighteen patients (36%) discontinued Epidiolex for the top reasons of worsened seizure frequency (7), behavioral issues (4), somnolence (2), and GI upset (2). An unintended positive side effect of the medication was a subjective report of improved cognition in 20 patients (40%). Conclusions: In its first 15 months of clinical availability at a single tertiary care children’s hospital, Epidiolex is being used outside of its approved indications of Dravet and Lennox-Gasteaut syndromes. When compared to the original trials, our cohort had greater than expected efficacy and fewer adverse effects of Epidiolex, but higher rates of discontinuation of the drug. Improved cognitive effects were an unintended positive finding, and two patients continued the medicine because of this effect, despite no impact on their seizure control. Funding: Please list any funding that was received in support of this abstract.: This study did not receive any funding.