M. Yavuz, Ü. Yılmaz, F. Gülağız, Y. Güzin, S. Pekuz and A. Ünalp (2026). Efficacy, safety, and growth outcomes of ketogenic diet therapy in children under two years of age. European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 61, 68-77. https://doi.org/10.1016/j.ejpn.2026.03.007

PubMed 41905333

[Abstract]
OBJECTIVE: This study aims to evaluate the efficacy, safety and impact on growth of ketogenic diet therapy (KDT) in infants under two years of age with drug-resistant epilepsy.

METHODS: A retrospective analysis was conducted on 36 infants who received KDT. Demographic data, seizure frequency, adverse effects, dietary adherence, and growth metrics were assessed at baseline and during follow-up intervals.

RESULTS: In the intent-to-treat analysis, all patients who initiated the ketogenic diet were included, and the responder rates at 1, 3, 6, 12, 18, and 24 months were 52.8%, 58.3%, 58.3%, 52.8%, 38.9%, and 36.1%, respectively. Among those continuing KDT, one-third remained seizure-free at Month 24. Metabolic etiologies showed higher early responsiveness. Dyslipidemia was the most frequent adverse event but improved significantly over time. Adverse effects such as nephrolithiasis and gastrointestinal symptoms were common but manageable. Growth demonstrated a mild but statistically significant decline in Z-scores. Medication burden decreased over time, and 23.3% of patients who maintained long-term adherence ultimately became medication-free.

CONCLUSION: KDT is an effective and generally well-tolerated treatment for drug-resistant epilepsy in infants, providing meaningful seizure control, reduced medication use, and an acceptable safety profile when carefully monitored.