F. Zhao, W. Hu, W. Zhang, Y. Lu, J. Li and H. Zhang (2026). Effectiveness, tolerability, and retention of the ketogenic diet for infantile epileptic spasms syndrome: a single-center cohort study. Frontiers in neurology, 17, 1701141. https://doi.org/10.3389/fneur.2026.1701141

PubMed 41821635

[Abstract]
OBJECTIVE: This study aimed to investigate the effectiveness, tolerability, and retention of the ketogenic diet (KD) in patients with infantile epileptic spasms syndrome (IESS).

METHODS: In this single-center prospective cohort study, baseline data were collected from the Children's Hospital Affiliated to Shandong University. Follow-up assessments were conducted at 3, 6, and 12 months after initiating KD. Outcomes included seizure frequency, adverse reactions, and retention rates. Survival analysis was performed to examine the association between retention rates and follow-up duration.

RESULTS: A total of 74 patients with IESS were admitted. The seizure response rates were 56.8% (42/74), 41.9% (31/74), and 25.7% (19/74) at 3, 6, and 12 months, respectively. The corresponding seizure-free rates were 13.5% (10/74), 13.5% (10/74), and 8.1% (8/74), respectively. Retention rates at 3, 6, and 12 months were 70.2% (52/74), 43.2% (32/74), and 25.7% (19/74), respectively. Survival analysis indicated that retention rates decreased over time, most markedly within the first 3 months. Responders to the KD exhibited significantly higher retention rates than non-responders throughout the 12 months (HR = 0.35, 95% CI: 0.19-0.64; p < 0.001). Adverse reactions were reported in 20.3% of patients, with gastrointestinal symptoms being the most common (16.2%), including constipation (6.8%), diarrhea (5.4%), and vomiting (4.0%).

CONCLUSION: The KD demonstrated favorable effectiveness and an acceptable safety profile in patients with IESS, particularly in those who did not respond to first-line antiseizure medications (ASMs). The higher retention rates among responders supports its utility in children with IESS. Therefore, early initiation of the KD should be considered for IESS patients who do not respond to first-line ASMs.