Study register · detailKohortenstudie · Epilepsy · 2026
Adjunctive cannabidiol in intractable pediatric epilepsy: A retrospective study on tolerability, efficacy, and safety across genetic and nongenetic etiologies.
Shim et al.·MedicineImpact 1.7
Clear benefitGRADEVery low2 citations
Samplen = 29 Pat.
DurationMedian 14.3 months follow-up
EndpointSeizure reduction ≥50 %
Blindingn.a.
DesignKohortenstudie
Cannabinoidcbd
Routeoral
”Key finding
In 79,3% of patients a reduction in seizure frequency of ≥50% was achieved, 34,5% achieved a ≥75% reduction without generalized motor seizures; one patient achieved seizure freedom; high retention rate (>86% at 12 and 24 months) with mild, manageable side effects.
Summary
Retrospective cohort n=29 pediatric treatment-refractory epilepsy (diverse genetic/non-genetic etiologies), adjunctive CBD median 14,2 mg/kg/d over a median 14,3 months follow-up. Retention rate >86% at 12/24 months. At 12 months: 79,3% achieved ≥50% seizure reduction, 34,5% achieved ≥75% reduction without generalized motor seizures, 1 patient (GABRB3 variant) seizure-free. Adverse events in 37,9% (mostly somnolence/lethargy, mild, manageable by ASM adjustment); discontinuation in n=3 (pneumonia, lethargy, seizure aggravation).
P
PopulationPediatric patients (6–24 years) with treatment-refractory epilepsy of various genetic and non-genetic etiologies, n=29, treated at Korea University Hospitals
I
InterventionAdjunctive cannabidiol (CBD), median maintenance dose 14,2 mg/kg/d, oral, as add-on therapy to existing antiepileptic drugs
O
Outcome79,3 % of patients achieved a ≥50% reduction in seizure frequency after 12 months; 34,5 % achieved a ≥75% reduction without generalized motor seizures; one patient achieved seizure freedom; retention rate >86 % at 12 and 24 months
Confidence in the evidence
Very lowLowModerateHigh
Very low
The lowest GRADE level, the effect estimate remains uncertain.
This retrospective cohort study evaluated the tolerability, efficacy, and safety of adjunctive cannabidiol (CBD) therapy in pediatric-onset intractable epilepsy across diverse genetic and nongenetic etiologies. Twenty-nine patients aged 6 to 24 years, treated at Korea University Hospitals between April 2019 and May 2024, were included. The median follow-up duration was 14.3 months. Confirmed genetic etiologies included SCN1A-related epilepsy (6.9%); GABRB3-, SCN2A-, KCNT1-, KIF1A-, and COL4A1-related epilepsies (3.4% each); Angelman syndrome and Down syndrome (3.4% each). Presumed genetic etiologies included hemimegalencephaly (3.4%) and cortical dysplasia (6.9%). Acquired causes included hypoxic brain injury (6.9%) and CNS infection (10.3%). In 41.4% of cases, the etiology was unidentified; among them, 58.3% had a history of infantile spasms. At CBD initiation, patients were receiving a median of 5 antiseizure medications, most commonly valproic acid (93.1%), clobazam (82.8%), and levetiracetam (75.9%). The median maintenance dose of CBD was 14.2 mg/kg/d. The retention rate was above 86% at both 12 and 24 months. At 12 months, 79.3% achieved a >/=50% reduction in seizure frequency, and 34.5% achieved a >/=75% reduction without generalized motor seizures. One patient with a GABRB3 variant achieved seizure freedom. Adverse events occurred in 37.9%, most commonly somnolence and lethargy. These were mild and resolved with antiseizure medication adjustments. CBD was discontinued in 3 patients due to pneumonia, lethargy, or seizure aggravation. CBD therapy demonstrated favorable retention, efficacy, and safety profiles in pediatric-onset intractable epilepsy across a spectrum of etiologies.