Objective To investigate the efficacy and safety of the ketogenic diet (KD) treatment in refractory epilepsy (RE) with myoclonic seizures as the predominant seizure type. Methods Clinical data were collected from 34 pediatric patients with RE characterized by myoclonic seizures who underwent KD treatment at the Department of Neurology, Jinan Children's Hospital from January 2021 to October 2023. The classic KD protocol was applied, and seizure frequency and adverse reactions were monitored at 1, 3, and 6 months post-treatment. Results Among the 34 patients, intention-to-treat (ITT) analysis revealed seizure-free rates of 11.8%, 17.6%, and 14.7% at 1, 3, and 6 months KDT, respectively. Seizure control response rates were 41.2%, 52.9%, and 38.2%. Proximal protocol (PP) analysis showed the KDT treatment achieved seizure-free rates of 11.8%, 25%, and 5% at 1, 3, and 6 months, respectively, with seizure control efficacy rates of 41.2%, 75%, and 86.7%. Subgroup analysis: Among 34 patients, 16 had epilepsy syndrome (ES) and 18 had myoclonic epilepsies (MEs). Comparing efficacy rates, the ES group showed significantly higher efficacy than the MEs group at 1 month (P=0.045), with no significant differences observed at 3 or 6 months. Adverse reactions occurred in some patients: 6 developed gastrointestinal adverse events, 2 experienced somnolence, and 2 had urinary system adverse reactions during KD treatment. Conclusion KD treatment is an effective treatment for RE with predominant myoclonic seizures. Short-term application demonstrates good safety, but adherence issues require attention.
