Use of Purified Cannabidiol in Treatment-Resistant Pediatric Epilepsy: Beyond Lennox-Gastaut Syndrome, Dravet Syndrome, and Tuberous Sclerosis Complex.

Kahan M.; Lin T.; Joshi S.; Hagopian E.; Vu M.; Van Hirtum-Das M.; Agurs L.; Holder D. · Pediatric neurology · 2026

**Background & Methods** This was a retrospective chart review of 144 pediatric patients with treatment-resistant epilepsy prescribed purified cannabidiol (CBD; Epidiolex) at a single Level 4 Epilepsy Center between January 2020 and January 2022. The study compared treatment response in patients with FDA-approved indications (Lennox-Gastaut syndrome, Dravet syndrome, tuberous sclerosis complex; n=81) versus off-label diagnoses (n=63), with median age at initiation of 10 years and concurrent use of 3 anti-seizure medications. **Key Findings** • **Comparable efficacy across indications**: 65% of FDA-labeled group and 63% of non-FDA labeled group achieved ≥50% seizure reduction (P=0.9), demonstrating CBD efficacy in pediatric epilepsies beyond approved diagnoses. • **Seizure type differences**: FDA-labeled group had significantly higher proportion of tonic seizures (63% vs. 29%, P<0.001); otherwise baseline seizure types were not significantly different between groups. • **Broad tolerability profile**: CBD was well-tolerated as adjunctive therapy with similar side effect profiles between groups, supporting potential consideration in pharmacoresistant epilepsies outside labeled indications. **Dosage & Administration** Not reported. **Safety & Adverse Effects** The most common adverse effects were nausea/diarrhea/gastrointestinal upset (19%), agitation (7.6%), weight loss (6.3%), insomnia (4.9%), and secretions/drooling (2.8%, only in patients concurrently using clobazam). No serious adverse events or treatment discontinuations due to safety were reported. **Evidence Quality** This study is limited by its retrospective design at a single institution, which restricts generalizability and introduces selection bias. The 35% exclusion rate (79 of 223 patients with prescriptions) due to incomplete data or lost follow-up may further bias results. Lack of standardized seizure counting methods, variable follow-up duration, and absence of control group limit causal inference. Documentation of responder status relied on clinical documentation rather than standardized outcome measures. As a retrospective observational study, this provides level 3-4 evidence and cannot establish causation. However, the large single-center cohort and inclusion of non-FDA-approved indications provide valuable real-world data suggesting CBD efficacy beyond labeled uses, warranting prospective studies to confirm these preliminary findings.

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