# Treatment of pediatric epilepsy

**Authors:** Junxiao Li, Tinghong Liu, Chang Liu, Jie Deng, Shijie Wu, Suhui Kuang, Xiaotong Li, Zhirong Wei, Shuli Liang

PMC · DOI: 10.1002/ped4.70043 · Pediatric Investigation · 2026-01-29

## TL;DR

This paper reviews treatment options for pediatric epilepsy, including medications, surgery, diet, and emerging therapies like gene therapy.

## Contribution

The paper highlights the potential of disease-modifying therapies and personalized medicine in treating pediatric epilepsy.

## Key findings

- Minimally invasive surgery showed a 68% seizure-free rate at 2 years in pediatric cases.
- Ketogenic diet can reduce seizures by more than 50% but carries metabolic risks.
- Gene therapy and antisense oligonucleotides show promise in early trials for treating epilepsy.

## Abstract

Pediatric epilepsy is a neurological disorder arising from various etiologies, including structural, genetic, immune, infectious, metabolic, and unknown causes. Anti‐seizure medications remain the primary treatment; however, in cases of drug‐resistant epilepsy, surgical interventions, ketogenic diet, and emerging therapies have become increasingly effective options. Disease‐modifying treatments, such as antisense oligonucleotides and adeno‐associated virus–mediated gene replacement, have shown promise in some epilepsy treatments, with early trials reporting moderate seizure reduction. Minimally invasive surgical approaches, including magnetic resonance–guided laser interstitial thermal therapy, have also demonstrated favorable outcomes, showing a 68% seizure‐free rate at 2 years in the largest pediatric series. Although the ketogenic diet is effective in some patients, demonstrating superiority over conventional management for >50% seizure reduction, long‐term use may be associated with metabolic risks; careful monitoring is warranted. Future treatment strategies are expected to emphasize personalized medicine through the integration of genetic, electrophysiological, and neuroimaging data to optimize therapeutic decision‐making and enable targeted interventions based on the underlying etiology.

Anti‐seizure medications are the first‐line treatment for the vast majority of children with epilepsy, with the advantages of non‐invasive wide adaptability. Surgery is the main treatment for drug‐resistant epilepsy and lesion‐related epilepsy, which can cure some cases of epilepsy in children. A ketogenic diet is often an add‐on therapy. Gene therapy and disease modification therapy may bring new hope to pediatric epilepsy.

## Linked entities

- **Diseases:** epilepsy (MONDO:0005027)

## Full-text entities

- **Genes:** PVALB (parvalbumin) [NCBI Gene 5816] {aka D22S749}, TSC2 (TSC complex subunit 2) [NCBI Gene 7249] {aka LAM, PPP1R160, TSC4}, SCN1A (sodium voltage-gated channel alpha subunit 1) [NCBI Gene 6323] {aka DEE6, DEE6A, DEE6B, DRVT, EIEE6, FEB3}, ALDH7A1 (aldehyde dehydrogenase 7 family member A1) [NCBI Gene 501] {aka ATQ1, EPD, EPEO4, PDE}, SCN1B (sodium voltage-gated channel beta subunit 1) [NCBI Gene 6324] {aka ATFB13, BRGDA5, DEE52, EIEE52, GEFSP1}, MTOR (mechanistic target of rapamycin kinase) [NCBI Gene 2475] {aka FRAP, FRAP1, FRAP2, RAFT1, RAPT1, SKS}, SLC2A1 (solute carrier family 2 member 1) [NCBI Gene 6513] {aka CSE, DYT17, DYT18, DYT9, EIG12, GLUT}, TSC1 (TSC complex subunit 1) [NCBI Gene 7248] {aka LAM, TSC}, CAMK2A (calcium/calmodulin dependent protein kinase II alpha) [NCBI Gene 815] {aka CAMKA, CaMKIINalpha, CaMKIIalpha, MRD53, MRT63}
- **Diseases:** PDE (MESH:C536254), sleep disturbance (MESH:D012893), trauma (MESH:D014947), drop attacks (MESH:D013575), cyclin-dependent kinase-like 5 deficiency disorder (MESH:C538124), blood loss (MESH:D016063), folate or biotinidase deficiency (MESH:D028921), anxiety (MESH:D001007), irritability (MESH:D001523), chromosomal abnormalities (MESH:D002869), tumors (MESH:D009369), learning disabilities (MESH:D007859), CCT (MESH:C535338), DRE (MESH:D000069279), diarrhea (MESH:D003967), stroke (MESH:D020521), focal epilepsy (MESH:D004828), hemorrhage (MESH:D006470), neurogenetic disorders (MESH:D020271), infantile epileptic spasm syndrome (MESH:D013036), developmental and epileptic encephalopathies (MESH:C562695), vomiting (MESH:D014839), Seizure (MESH:D012640), metabolic disorders (MESH:D008659), genetic defects (MESH:D030342), LGS (MESH:D065768), neurodevelopmental deficits (MESH:D009461), hyperthermia (MESH:D005334), brain lesions (MESH:D001927), hypothermia (MESH:D007035), mitochondrial gene defects (MESH:C565376), abnormal neurodevelopment (MESH:D000014), Epileptic Seizures (MESH:D004827), hippocampal sclerosis (MESH:D000092223), hypercalcemia (MESH:D006934), vascular disorders (MESH:D002561), infection (MESH:D007239), MRgLITT (MESH:D016609), cortical dysplasia type II (MESH:C537067), epilepsy syndrome (MESH:D000073376), hepatic ketogenesis (MESH:D056486), KD (MESH:D009080), executive dysfunction (MESH:D006331), cortical dysplasia (MESH:D054220), constipation (MESH:D003248), depression (MESH:D003866), DISEASE-MODIFYING (OMIM:603855), cognitive deficits (MESH:D003072), neurodevelopmental impairment (MESH:D009422), memory deficits (MESH:D008569), infectious (MESH:D003141), central nervous system infections (MESH:D002494), neuropsychological deterioration (MESH:D000075902), necrosis (MESH:D009336), TSC (MESH:D014402), developmental delay (MESH:D002658), Dravet syndrome (MESH:D004831)
- **Chemicals:** Eslicarbazepine (MESH:C571001), Clobazam (MESH:D000078306), TC (MESH:D013667), Pyridoxine (MESH:D011736), beta-hydroxybutyrate (MESH:D020155), brivaracetam (MESH:C482793), ethosuximide (MESH:D005013), ketones (MESH:D007659), sirolimus (MESH:D020123), clonazepam (MESH:D002998), oxcarbazepine (MESH:D000078330), phenobarbital (MESH:D010634), Pyrimidine (MESH:C030986), GABA (MESH:D005680), Felbamate (MESH:D000078328), everolimus (MESH:D000068338), sodium (MESH:D012964), AMPA (MESH:D018350), Ganaxolone (MESH:C105051), ASMs (-), zonisamide (MESH:D000078305), valproate (MESH:D014635), acetoacetate (MESH:C016635), carbohydrate (MESH:D002241), acetone (MESH:D000096), pipecolic acid (MESH:C031345), phenytoin (MESH:D010672), lamotrigine (MESH:D000077213), carbamazepine (MESH:D002220), cenobamate (MESH:C000654784), Lacosamide (MESH:D000078334), Cannabidiol (MESH:D002185), glucose (MESH:D005947), perampanel (MESH:C551441), levetiracetam (MESH:D000077287), lysine (MESH:D008239), vitamin B6 (MESH:D025101), gabapentin (MESH:D000077206), topiramate (MESH:D000077236), Vigabatrin (MESH:D020888)
- **Species:** Adeno-associated virus (species) [taxon 272636], Mus musculus (house mouse, species) [taxon 10090], Homo sapiens (human, species) [taxon 9606]

## Full text

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## Figures

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## References

99 references — full list in the complete paper: https://tomesphere.com/paper/PMC12921632/full.md

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Source: https://tomesphere.com/paper/PMC12921632