{
  "abstract": "Background and Importance Epilepsy is a neurological disorder with diverse aetiologies and comorbidities, including neurodevelopmental and autism spectrum disorders. Approximately 50% of paediatric-onset epilepsies are drug-resistant (DRE) including Dravet syndrome (DS), Lennox–Gastaut syndrome (LGS) and Tuberous Sclerosis Complex (TSC). Dravet Syndrome is characterised by drug-resistant seizures, progressive cognitive and motor deficits and often pathogenic variants in SCN1A gene. Lennox–Gastaut syndrome typically presents with seizure between 1–7 years and has genetic or acquired aetiologies. Tuberous Sclerosis Complex (TSC) is a multisystemic disorder with drug-resistant seizures due to TSC1/TSC2 mutations.In Italy, Cannabidiol (CBD) (Epidyolex) has been approved by AIFA since 20191 as add-on therapy for DS and LGS patients older than 2 years (in combination with clobazam) and for TSC patients older than 1 year.Aim and Objectives To evaluate the efficacy, tolerability, and safety of add-on CBD in paediatric and young adult patients with DREMaterial and Methods Clinical and pharmacological data of patients treated with add-on CBD at the Regional Paediatric Epilepsy Centre, Salesi Hospital (Ancona, Italy) between September 2019 and December 2023 were analysed. Efficacy was defined as ≥50% reduction in seizure frequency. Adverse events (AEs) were classified as minor or major. Quality of life (QoL) was assessed through caregiver reports.Results Thirty-six patients were included: four with DS, seven with TSC, nineteen with LGS, and six with other DRE. Mean age at add-on CBD initiation was 26 years and 4 months; at treatment onset, 50% of patients were in the paediatric age range (<18 years). The mean treatment duration was 15 months, with an average of 3.25 concomitant antiseizure drugs. Overall, 53% (n=19) achieved a clinically relevant seizure reduction, most of whom (n=13) had LGS. Discontinuation occurred in 47% (n=17), due to lack of efficacy (n=12) or AEs (n=5). Adverse events were reported in 58% of patients, predominantly minor and transient. QoL improvement was reported in 53% of patients, including better sleep quality in 30.5%.Conclusion and Relevance In this real-world cohort, add-on CBD demonstrated clinically relevant efficacy with acceptable tolerability and safety in paediatric and young adult patients with DRE, particularly in LGS. These findings support its role as a valuable therapeutic option in refractory epilepsies.References and/or Acknowledgements 1. www.aifa.gov.it/documents/20142/1540069/123_EPIDYOLEX_DS_pp_scheda_innovativita_GRADE.pdfConflict of Interest No conflict of interest",
  "authors": [
    {
      "affiliations": [
        "University of Camerino- Faculty of Pharmacy, Experimental Medicine and Public Health, Camerino, Italy"
      ],
      "name": "C Polidori"
    }
  ],
  "title": "4CPS-011 Paediatric drug-resistant epilepsies: efficacy, tolerability, and safety of add-on cannabidiol",
  "uid": "b656fec5-66f0-5ade-823f-f00334437856"
}