Evidence map›Paper›PMID 40673944›Full record

Trial reportEpilepsia2025

Long-term efficacy and safety of cannabidiol in patients with treatment-resistant focal epilepsies treated in the Expanded Access Program.

Anup D Patel, Jerzy P Szaflarski, Paul D Lyons, Michael Boffa, Teresa Greco, Timothy B Saurer, Karthik Rajasekaran, Kelly C Simontacchi, Elizabeth A Thiele

Abstract readClinical Trial, Phase III
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Epilepsia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed.

  1. Trial
  2. Article
  3. Article
  4. Review
  5. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Anup D PatelNationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0001-9313-1541
Jerzy P SzaflarskiUniversity of Alabama at Birmingham, Birmingham, Alabama, USA.ORCID https://orcid.org/0000-0002-5936-6627
Paul D LyonsWinchester Neurological Consultants, Winchester, Virginia, USA.
Michael BoffaJazz Pharmaceuticals, Inc., Palo Alto, California, USA.
Teresa GrecoJazz Pharmaceuticals, Inc., Gentium Srl, Villa Guardia, Italy.
Timothy B SaurerJazz Pharmaceuticals, Inc., Palo Alto, California, USA.
Karthik RajasekaranJazz Pharmaceuticals, Inc., Palo Alto, California, USA.
Kelly C SimontacchiJazz Pharmaceuticals, Inc., Palo Alto, California, USA.
Elizabeth A ThieleMassachusetts General Hospital, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0003-3431-4713

Funding

Jazz Pharmaceuticals, Inc.
6 · The paper itself

Abstract

objectiveCannabidiol (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.

methodsPatients 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.

resultsOf 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.

Indexed as

AnticonvulsantsCannabidiolDrug Resistant EpilepsyEpilepsies, PartialAdolescentAdultChildChild, PreschoolFemaleFollow-Up StudiesHumansMaleMiddle AgedTreatment OutcomeTuberous SclerosisYoung AdultAnticonvulsantsCannabidiolantiseizure medicationcannabidiolcortical dysplasiafocal epilepsiesfocal seizurestreatment‐resistant epilepsy

Identifiers

PMID40673944
PMCPMC12605695

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.