Evidence map›Paper›PMID 40968578›Full record

ArticleEuropean journal of neurology2025

Assessing Real World Efficacy, Safety, and 18-Month Retention Rates of Cannabidiol in Individuals With Drug Resistant Epilepsies.

Nicole Chemaly, Mathieu Kuchenbuch, Emma Losito, Anna Kaminska, Delphine Coste-Zeitoun, Giulia Barcia, Isabelle Desguerre, Marie Hully, Rima Nabbout

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In one paragraph

Article in European journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
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  4. 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.

Nicole ChemalyDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.ORCID 0000-0002-2333-9408
Mathieu KuchenbuchDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.ORCID 0000-0002-5944-2204
Emma LositoDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.
Anna KaminskaUnit of Pediatric Neurophysiology, Necker Enfants Malades Hospital, AP-HP, Paris, France.ORCID 0000-0003-3805-6651
Delphine Coste-ZeitounUnit of Pediatric Neurophysiology, Necker Enfants Malades Hospital, AP-HP, Paris, France.ORCID 0000-0003-4063-5345
Giulia BarciaUniversité de Paris Cité, Paris, France.ORCID 0000-0001-6657-5040
Isabelle DesguerreDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.ORCID 0000-0002-4074-5862
Marie HullyDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.ORCID 0000-0002-1742-3590
Rima NabboutDepartment of Pediatric Neurology, Reference Center for Rare Epilepsies, Hôpital Necker-Enfants Malades, Member of ERN EpiCARE, Paris, France.ORCID 0000-0001-5877-4074

Funding

Agence Nationale de la Recherche ANR-10-IAHU-01Agence Nationale de la Recherche ANR-23-RHUS-0002FAMASwiss Philanthropy Foundation
6 · The paper itself

Abstract

backgroundHighly purified Cannabidiol (CBD, epidiolex) is approved for treating Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis (TSC), with off-label use in drug-resistant epilepsies. The aim of this study is to evaluate efficacy, safety, and long-term retention of CBD in a large pediatric population.

methodsWe prospectively included all consecutive patients initiating CBD for drug-resistant epilepsy at our pediatric tertiary center from 2019 to 2021. Efficacy and safety were assessed via parental questionnaires on seizure and non-seizure outcomes at 1, 2, and 6 months, comparing each item to prior clinic visits. Retention rate was evaluated at 18 months.

resultsWe included 103 patients (mean age 11.2 years), with 47% receiving CBD for label-approved syndromes (23 DS, 15 LGS, 10 TSC) and 53% off-label. At 1 month, 54% of caregivers reported reduced seizure frequency and duration, with a positive overall impression in 62%. By months 2-6, 48% maintained seizure improvement, alongside sustained gains in communication (60%), alertness (54%), and motor skills (44%). Retention rates were 97% at 1 month, 90% at 2 months, 82% at 6 months, 66% at 12 months, and 55% at 18 months. Off-label use (p = 0.02), male gender (p = 0.049), and older age at initiation (p = 0.03) were associated with higher retention.

conclusionsCBD reduced seizures and improved non seizures' morbidities in drug-resistant epilepsy. Strong 18-month retention rate, including off-label use, highlights efficacy and tolerability, underscoring the need for further real-world data in various rare epilepsy syndromes beyond its market approval.

Indexed as

AnticonvulsantsCannabidiolDrug Resistant EpilepsyAdolescentChildChild, PreschoolEpilepsies, MyoclonicFemaleHumansLennox Gastaut SyndromeMaleProspective StudiesTreatment OutcomeAnticonvulsantsCannabidioldevelopmental and epileptic encephalopathyDravet syndromegender differencesLennox–Gastaut syndromeoff‐labelretention ratetuberous sclerosis

Identifiers

PMID40968578
PMCPMC12446575

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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.