Real-world outcomes of ketogenic diet therapy in children with drug-resistant epilepsy in a prospective cohort study

一项前瞻性队列研究:生酮饮食疗法对药物难治性癫痫患儿的真实世界疗效

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Abstract

The ketogenic diet (KD) is an established non-pharmacological treatment for drug-resistant epilepsy (DRE) in children, but data from large prospective cohorts in China remain limited. We conducted a prospective observational study of children with DRE whose caregivers elected KD treatment compared with those who continued conventional therapy. Seizure frequency, EEG findings, cognitive assessments, and adverse events were monitored over six months. A total of 136 children were enrolled (73 KD; 63 controls). At six months, children receiving KD showed a higher proportion of seizure reduction ≥ 50% compared with controls. EEG evaluations revealed improvements in background activity and reduction in epileptiform discharges, and cognitive assessments demonstrated gains in specific domains. Adverse effects were generally mild and manageable with dietary adjustments. In this single-center prospective cohort, KD was associated with improved seizure control, EEG patterns, and cognitive performance over six months compared with conventional therapy. While limited by non-randomized design and short follow-up, these findings provide real-world evidence supporting KD as a feasible and safe adjunctive therapy for pediatric DRE in China. Longer randomized studies are warranted to establish causality and evaluate long-term outcomes.

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