From syndrome to precision therapy: current and emerging therapeutic options for selected pediatric focal epilepsies
INTRODUCTION: Pediatric focal epilepsies comprise a heterogeneous group of disorders, ranging from self-limited childhood syndromes to severe drug-resistant forms associated with structural, genetic, immune, or unknown etiologies. Despite newer antiseizure medications, pediatric evidence remains largely extrapolated from heterogeneous focal-onset seizure trials, with limited syndrome- or localization-specific data. AREAS COVERED: A structured iterative narrative search of PubMed, Scopus, and Embase was completed in May 2026 and supplemented by section-specific searches and manual reference screening. This review summarizes current and emerging pharmacotherapeutic options for selected representative pediatric focal epilepsies using a syndrome- and localization-oriented approach. We discuss self-limited focal epilepsies of childhood, frontal lobe epilepsy, and temporal lobe epilepsy, highlighting conventional antiseizure medications, newer agents, and investigational or repurposed treatments. Particular attention is given to drug resistance, comorbidities, rational polytherapy, and integration of pharmacological treatment with genetic diagnosis, neuroimaging, and surgical evaluation. EXPERT OPINION: Management is moving from a purely seizure-based approach toward individualized, mechanism-informed care. Newer antiseizure medications expand therapeutic options, particularly in drug-resistant focal epilepsies, but should not delay etiological work-up or timely referral for epilepsy surgery. Future progress will require pediatric condition-specific trials, genotype-driven stratification, real-world data, and targeted therapies.
Authors
- Federico Vigevano (ORCID: https://orcid.org/0000-0001-7513-0051)
- Giovanni Battista Dell’Isola (ORCID: https://orcid.org/0000-0002-2302-1029)
- Lorenzo Perilli (ORCID: https://orcid.org/0000-0002-8630-6718)
- Alberto Verrotti
- Salvatore Savasta
- Pietro Ferrara
- Pasquale Striano
Institutions
- University of Siena (IT)
- University of Cagliari (IT)
- University of Perugia (IT)
- Saint Camillus International University of Health and Medical Sciences (IT)
- Applied Scientific Research (United States) (US)
- Campus Bio Medico University Hospital (IT)
- National Hospital for Neurology and Neurosurgery (GB)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele (IT)
- University College London (GB)
- University of Genoa (IT)
Publication Details
- Journal
- Expert Opinion on Pharmacotherapy
- Published
- 2026-08-27
- DOI
- https://doi.org/10.1080/14656566.2026.2725871
- Primary Topic
- Epilepsy research and treatment
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ministero della Salute