{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,21]],"date-time":"2026-10-21T14:04:28Z","timestamp":1792591468501,"version":"4.5.0"},"reference-count":17,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T00:00:00Z","timestamp":1770163200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background\/Objectives: Epilepsy represents one of the most common chronic neurological disorders in children, with a considerable proportion of patients exhibiting resistance to pharmacotherapy despite the advent of novel antiseizure medications (ASMs) in recent decades. This retrospective study assesses the off-label administration of cenobamate\u2014a newly approved antiseizure medication (ASM) for focal seizures in adults\u2014in a cohort of paediatric patients with drug-resistant epilepsy at a single neurology centre. Methods: Clinical outcomes were reviewed retrospectively for 18 children who received cenobamate for at least 6 months. Results: Eighteen paediatric patients with drug-resistant epilepsy received cenobamate therapy at a neurology centre. The mean age was 164.6 months, and each patient had previously trialled an average of 8.7 antiseizure medications. During a follow-up period of up to 29 months, 39% of participants achieved complete seizure freedom, while five additional patients experienced a seizure reduction exceeding 80%. Concomitant clobazam use was common among the cohort. Adverse events were reported in 78% of patients, predominantly somnolence, though these were generally transient or manageable. One patient developed a temporary exacerbation of seizures, which resolved following a dosage adjustment. Many patients were able to reduce or discontinue other ASMs during the observation period. Conclusions: Cenobamate demonstrated acceptable tolerability in this paediatric cohort, and seizure improvements were observed in a subset of patients. Further clinical trials are warranted to comprehensively evaluate the efficacy and safety profile of cenobamate in this patient population.<\/jats:p>","DOI":"10.3390\/jcm15031218","type":"journal-article","created":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T12:51:56Z","timestamp":1770209516000},"page":"1218","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Adjunctive Use of Cenobamate in Paediatric Drug-Resistant Epilepsy: A Real-World, Single-Centre Experience"],"prefix":"10.3390","volume":"15","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5010-2732","authenticated-orcid":false,"given":"Barbara","family":"Oleksy","sequence":"first","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Agata","family":"Lipiec","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alicja","family":"Goszcza\u0144ska-Ciuchta","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanna","family":"\u017bebrowska","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Magdalena","family":"Bosak","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical College, Jagiellonian University, 31-007 Cracow, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksandra","family":"Ku\u017aniar-Pa\u0142ka","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hanna","family":"Mazurkiewicz","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"El\u017cbieta","family":"Lipi\u0144ska","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tomasz","family":"Mazurczak","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1329-846X","authenticated-orcid":false,"given":"El\u017cbieta","family":"Stawicka","sequence":"additional","affiliation":[{"name":"Clinic of Paediatric Neurology, Institute of Mother and Child, Kasprzaka 17A, 01-211 Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,2,4]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"e20163908","DOI":"10.1542\/peds.2016-3908","article-title":"Incidence and Prevalence of Childhood Epilepsy: A Nationwide Cohort Study","volume":"139","author":"Aaberg","year":"2017","journal-title":"Pediatrics"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1069","DOI":"10.1111\/j.1528-1167.2009.02397.x","article-title":"Definition of drug resistant epilepsy: Consensus proposal by the ad hoc Task Force of the ILAE Commission on Therapeutic Strategies","volume":"51","author":"Kwan","year":"2010","journal-title":"Epilepsia"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"2491","DOI":"10.1111\/epi.17350","article-title":"Timing of referral to evaluate for epilepsy surgery: Expert Consensus Recommendations from the Surgical Therapies Commission of the International League Against Epilepsy","volume":"63","author":"Jehi","year":"2022","journal-title":"Epilepsia"},{"key":"ref_4","doi-asserted-by":"crossref","unstructured":"Verrotti, A., Iapadre, G., Di Francesco, L., Zagaroli, L., and Farello, G. (2020). Diet in the treatment of epilepsy: What we know so far. Nutrients, 12.","DOI":"10.3390\/nu12092645"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1007\/s40263-021-00827-8","article-title":"The Pharmacology and Clinical Efficacy of Antiseizure Medications: From Bromide Salts to Cenobamate and Beyond","volume":"35","author":"Klein","year":"2021","journal-title":"CNS Drugs"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1007\/s40263-021-00819-8","article-title":"Pharmacology of Cenobamate: Mechanism of Action, Pharmacokinetics, Drug\u2013Drug Interactions and Tolerability","volume":"35","author":"Roberti","year":"2021","journal-title":"CNS Drugs"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/S1474-4422(19)30399-0","article-title":"Safety and efficacy of adjunctive cenobamate (YKP3089) in patients with uncontrolled focal seizures: A multicentre, double-blind, randomised, placebo-controlled, dose-response trial","volume":"19","author":"Krauss","year":"2020","journal-title":"Lancet Neurol."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/j.seizure.2025.05.002","article-title":"Efficacy and safety of Cenobamate: A multicenter, retrospective evaluation of real-world clinical practice","volume":"130","author":"Bosak","year":"2025","journal-title":"Seizure Eur. J. Epilepsy"},{"key":"ref_9","doi-asserted-by":"crossref","unstructured":"Makridis, K.L., Bast, T., Prager, C., Kovacevic-Preradovic, T., Bittigau, P., Mayer, T., Breuer, E., and Kaindl, A.M. (2022). Real-World Experience Treating Pediatric Epilepsy Patients With Cenobamate. Front. Neurol., 13.","DOI":"10.3389\/fneur.2022.950171"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"4712","DOI":"10.1111\/epi.18586","article-title":"Real-world use of cenobamate in pediatric focal epilepsies and developmental epileptic encephalopathies: A multicenter retrospective series","volume":"66","author":"Jamardo","year":"2025","journal-title":"Epilepsia"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"108679","DOI":"10.1016\/j.yebeh.2022.108679","article-title":"Adjunctive use of cenobamate for pediatric refractory focal-onset epilepsy: A single-center retrospective study","volume":"130","author":"Varughese","year":"2022","journal-title":"Epilepsy Behav."},{"key":"ref_12","unstructured":"National Institutes of Health, National Cancer Institute (2026, January 29). Common Terminology Criteria for Adverse Events (CTCAE) v5.0, Available online: https:\/\/dctd.cancer.gov\/research\/ctep-trials\/for-sites\/adverse-events."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1038\/clpt.1981.154","article-title":"A method for estimating the probability of adverse drug reactions","volume":"30","author":"Naranjo","year":"1981","journal-title":"Clin. Pharmacol. Ther."},{"key":"ref_14","unstructured":"International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) (1994). Clinical Safety Data Management: Definitions and Standards for Expedited Reporting (E2A), ICH."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1007\/s40263-023-01027-2","article-title":"New GABA-Targeting Therapies for the Treatment of Seizures and Epilepsy: I. Role of GABA as a Modulator of Seizure Activity and Recently Approved Medications Acting on the GABA System","volume":"37","author":"Perucca","year":"2023","journal-title":"CNS Drugs"},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"109156","DOI":"10.1016\/j.yebeh.2023.109156","article-title":"The cenobamate-clobazam interaction- evidence of synergy in addition to pharmacokinetic interaction","volume":"142","author":"Osborn","year":"2023","journal-title":"Epilepsy Behav."},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"e83","DOI":"10.1111\/epi.18426","article-title":"Lack of effectiveness and seizure worsening with cenobamate in pediatric patients with Dravet syndrome","volume":"66","author":"Cagigal","year":"2025","journal-title":"Epilepsia"}],"container-title":["Journal of Clinical Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2077-0383\/15\/3\/1218\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,2,4]],"date-time":"2026-02-04T13:03:52Z","timestamp":1770210232000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2077-0383\/15\/3\/1218"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,2,4]]},"references-count":17,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2026,2]]}},"alternative-id":["jcm15031218"],"URL":"https:\/\/doi.org\/10.3390\/jcm15031218","relation":{},"ISSN":["2077-0383"],"issn-type":[{"value":"2077-0383","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,2,4]]}}}