Study register · detailKlinische Studie · Epilepsy · 2025
Long-term efficacy and safety of cannabidiol in patients with treatment-resistant focal epilepsies treated in the Expanded Access Program.
Patel et al.·EpilepsiaImpact 4.1
Clear benefitGRADEModerate6 citations
Samplen = 140 Pat.
Duration144 weeks
EndpointSeizure frequency
Blindingoffen
DesignKlinische Studie
Cannabinoidcbd
Routeoral
”Key finding
CBD was associated with sustained seizure reduction of 44–87% over 144 weeks, independent of epilepsy type.
Summary
Long-term EAP data on CBD (Epidiolex®) in focal epilepsies (n=140; 33 TSC, 107 other focal epilepsies). Median CBD dose 25 mg/kg/d (TSC) or 23 mg/kg/d (non-TSC). Over 144 weeks: median reduction in focal seizures 51–87% (TSC) or 46–75% (non-TSC); total seizures 44–87% (TSC) or 45–71% (non-TSC). Responder rates comparable. AEs in 91% (TSC) and 96% (non-TSC).
P
PopulationPatients with treatment-resistant focal epilepsies (TSC n=33, non-TSC n=107), median age 11,9 and 17,8 years respectively, total group n=140
I
InterventionPlant-derived highly purified cannabidiol (Epidiolex, 100 mg/mL oral), starting dose 2–10 mg/kg/day, titration up to max. 25–50 mg/kg/day
O
OutcomeMedian reduction in focal seizures of 51–87% (TSC) or 46–75% (non-TSC) versus baseline over 144 weeks; similar responder rates in both groups
Confidence in the evidence
Very lowLowModerateHigh
Moderate
The third of four GRADE levels, the effect estimate is probably reliable.
Downgraded for
Risk of bias
Quality profile
Sample size★★★★★
BlindingOpen-label
Effect sizeClear benefit
Citations / year★★★★★
Authors
Patel AD, Szaflarski JP, Lyons PD, Boffa M, Greco T, Saurer TB, Rajasekaran K, Simontacchi KC, Thiele EA
Objective: Cannabidiol (CBD) treatment was associated with long-term seizure reduction in patients with various treatment-resistant epilepsies who participated in the CBD Expanded Access Program. A phase 3 trial showed CBD effectively treats tuberous sclerosis complex (TSC)-associated seizures, which are primarily of focal origin. However, the effectiveness of CBD in non-TSC focal epilepsies is not well reported. Here, we evaluate CBD treatment outcomes among patients with a variety of focal epilepsies, including
Tsc. Methods: Patients received plant-derived highly purified CBD (Epidiolex; 100 mg/mL oral solution) doses starting at 2-10 mg/kg/day and titrated up to each patient's limit or to a maximum of 25-50 mg/kg/day. CBD effectiveness was assessed by the median percentage change from baseline in monthly frequency of focal and total seizures and by responder rates through 144 weeks of treatment. Safety data were reported for the full follow-up period.
Results: Of 140 patients with focal epilepsies, 33 (24%) had TSC and 107 (76%) had other focal epilepsies, including cortical dysplasia (14%), frontal lobe epilepsy (10%), and malformation of cortical development (9%). Median age was 11.9 years (range = 2-31) in the TSC group and 17.8 years (range = 2-73) in the non-TSC group. Median CBD daily dose was comparable between TSC and non-TSC groups (25 and 23 mg/kg/day, respectively). CBD treatment was associated with a median reduction from baseline of 51%-87% in focal seizures and 44%-87% in total seizures in the TSC group and 46%-75% and 45%-71% in the non-TSC group, respectively. Responder rates were similar for both groups. Adverse events occurred in 91% of the TSC group and 96% of the non-TSC group.
Significance: Open-label CBD treatment was associated with sustained seizure reduction through 144 weeks, with a consistent safety profile in patients with treatment-resistant focal epilepsies, regardless of epilepsy type.