Use of Purified Cannabidiol in Refractory Pediatric Epilepsy: Beyond Lennox Gastaut Syndrome, Dravet Syndrome, and Tuberous Sclerosis Complex
Abstract number :
3.438
Submission category :
7. Anti-seizure Medications / 7E. Other
Year :
2024
Submission ID :
466
Source :
www.aesnet.org
Presentation date :
12/9/2024 12:00:00 AM
Published date :
Authors :
Presenting Author: Madeline Kahan, MD – Children's Hospital Los Angeles
Sucheta Joshi, MD, MS, FAES – Children's Hospital Los Angeles
Elizabeth Hagopian, BA – Children's Hospital Los Angeles
My H. Vu, MS – Children's Hospital Los Angeles
Michele Van Hirtum-Das, MD – Children's Hospital Los Angeles
Latanya Agurs, MD – Children's Hospital Los Angeles
Deborah L. Holder, MD – Cedars-Sinai Guerin Children's
Rationale: The pharmaceutical formulation of highly purified cannabidiol (CBD, Epidiolex®) is approved by the FDA for the treatment of seizures associated with Lennox Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis (TSC) in patients 1 year of age and older. Among the large volume of drug-resistant cases treated at pediatric Level 4 Epilepsy Centers, many patients fall outside of these categories, but are still prescribed cannabidiol for off label use. This study sought to characterize the real-world experience with CBD of a large single pediatric comprehensive epilepsy center, and to compare response to CBD within and outside of its FDA-approved indications.
Methods: A retrospective chart review was performed of patients with a prescription for CBD at Children’s Hospital Los Angeles between January 2020 and January 2022, excluding patients who were previously included in our center’s 2019 analysis. Patients were not included in the analysis if initially prescribed CBD elsewhere, follow-up was outside our institution, medication was not started, or if diagnostic data was not available. Between-group differences were assessed via Wilcoxon Rank-sum and Fisher’s Exact tests, as appropriate.
Results: Of 223 patients with a CBD prescription during the study period, 149 patients had sufficient data available for review. Of these patients, 88 (59%) carried diagnoses of LGS, DS, or TSC, and 61 (41%) carried other diagnoses outside of FDA-labeled indications. Median (IQR) age at initiation was 10.2 years (5.5, 14.2), and median (IQR) number of concurrent antiseizure medications was 3.0 (2.0, 3.0). There was no significant difference in the proportion of patients treated concurrently with VNS or ketogenic diet between the two groups. The FDA-labeled (61%) group had a significantly higher proportion of patients with tonic seizures compared to the non-FDA labeled (16%) group (p < 0.001), otherwise there were no significant differences in baseline seizure types. Overall, 49 patients (61%) in the FDA-labeled group and 36 (60%) of the non-FDA labeled group had a documented reduction in seizure frequency of at least 50% on CBD (p > 0.9). Among each group, the median (IQR) number of other antiseizure medications weaned was 1.0 (0.0, 1.0) (p = 0.3). The most common side effects reported were nausea/diarrhea/gastrointestinal upset (16%), agitation (8%), insomnia (3.6%), weight loss (2.9%), and secretions/drooling (2.9%, seen only in patients on concurrent clobazam).
Conclusions: Among our cohort of refractory pediatric epilepsy, purified CBD was efficacious in a broad range of pediatric epilepsies, and was well tolerated as an adjunctive antiseizure medication. This medication may be considered in pharmacoresistant pediatric epilepsies in conditions beyond its FDA-labeled indications.
Funding: None
Anti-seizure Medications