Authors :
Presenting Author: Emilly Matias, MS – University of Miami
Neelesh Pandey, BS – University of Miami
Claire Shi, BS – University of Miami
Klenisson Brenner, MD – University of Miami
Paula Kostreni, MD – University of Miami
Stella Ghevondyan, BS – UC San Diego
Nikol Agadzhanian, MSc – Yale University, Yale School of Medicine
Jayanth Tallapalli, – Yale University
Rohith Nelakurthi, MD – Yale
Aline Herlopian, MD – Yale
Hyunmi Choi, MD, MS – Columbia University
Kamil Detyniecki, MD – University of Miami
Rationale:
Purified pharmaceutical cannabidiol (CBD, Epidiolex®) was FDA-approved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) for patients 1 year of age and older. However, a large number of drug-resistant epilepsy cases treated at Level 4 Epilepsy Centers, fall outside of these categories and are prescribed cannabidiol for off label use. This study sought to characterize the real-world experience with CBD of a large adult comprehensive epilepsy center, and to compare treatment responses within and outside its FDA approved indications. Methods:
We conducted a retrospective chart review of epilepsy patients receiving CBD from November 2018 to April 2025. Demographic data, comorbidities, medication history, reasons for discontinuation, and adverse events were collected. Retention rates and seizure freedom outcomes were calculated. This is a preliminary analysis of the first 50 patients. Results:
The cohort was 48% male, with a mean seizure onset age of 9.5 years (SD 9.95, range 0–49). Most patients (84%) had focal epilepsy, while 12% had mixed, and 4% were unclear. Approximately half of the patients (52%) had some degree of static encephalopathy. Epilepsy syndromes were identified in 90% of patients, with 74% having Focal and 10% Lennox–Gastaut syndrome. The median age at CBD initiation was 33.5 years, and the median treatment duration was 41.4 months. Patients had previously tried a mean of 6.66 anti-seizure medications (ASMs) and were taking 2.89 concurrent ASMs. The most frequently used prior ASM was clobazam (70%). In the end, 52% discontinued CBD, primarily due to intolerability (24%) or lack of efficacy (22%). Adverse events were reported in 72% of patients. The most common were gastrointestinal issues (30%), sedation (26%), and psychiatric symptoms (16%). Adverse events, leading to discontinuation, were less frequent: sedation (8%), gastrointestinal issues (6%), and psychiatric symptoms (6%). Retention rates were 90% at 3 months, 80% at 6 months, 63.3% at 12 months, 60.4% at 24 months, and 56.3% at 36 months. Overall, 18.75% of patients achieved seizure freedom for at least 6 months while 12.5% achieved seizure freedom for 12 months.
Conclusions:
Purified Pharmaceutical cannabidiol (CBD) provides modest seizure control in refractory epilepsy of multiple types. While adverse events were common, they led to discontinuation in only a quarter of patients. These real-world findings can provide valuable insights into its potential use in other epilepsy types beyond its current FDA-labeled indications. Funding:
Jazz Pharmaceuticals.