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Evaluating the therapeutic efficacy of a modified ketogenic diet in children with autism spectrum disorder: a randomized controlled trial

AI Summary
  • Modified ketogenic diet plus rehabilitation produced significantly greater short-term reductions in ABC and CARS scores than rehabilitation alone (P=0.027 and P=0.003).
  • Randomised controlled trial of 62 young children with ASD, 31 per group, two-month intervention with hospital and home-based rehabilitation.
  • Acceptable short-term feasibility reported, with caregivers managing home diet and hospital providing lunches during training days.
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BMC Pediatr. 2026 Sep 11;26(1):893. doi: 10.1186/s12887-026-07708-3.

ABSTRACT

BACKGROUND: Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder marked by persistent impairments in social communication and interaction, together with restricted and repetitive behavior or interests. Recent epidemiological data indicate that the prevalence of ASD has continued to increase worldwide. Current behavioral and pharmacological interventions remain limited in efficacy, particularly for associated comorbidities. In this context, metabolic interventions, especially the ketogenic diet (KD), have attracted growing interest as a potential therapeutic strategy.

METHODS: A total of 62 young children with ASD admitted to Hefei Maternal and Child Health Hospital between January 2024 and January 2025 were prospectively enrolled and randomly assigned to a treatment group or a control group (31 per group). Both groups underwent hospital-based rehabilitation training on weekdays and home-based rehabilitation on weekends over a 2-month period. The treatment group additionally received a modified KD, comprising ketogenic nutritional powder and a structured dietary plan, with lunch provided at the hospital on training days while breakfast, dinner, weekend dietary implementation, and home monitoring were managed by caregivers. The control group followed a regular diet along with the same rehabilitation program. Changes in ASD-related symptoms were assessed using the Autism Behavior Checklist (ABC) and Childhood Autism Rating Scale (CARS).

RESULTS: After two months of intervention, both groups showed significant reductions in ABC and CARS scores compared with baseline (P < 0.05). Between-group comparison of individual change scores showed larger reductions in the treatment group for both ABC (U = 324.000, P = 0.027) and CARS (U = 273.500, P = 0.003).

CONCLUSIONS: In this cohort of young children with ASD, the modified ketogenic diet combined with rehabilitation training was associated with greater short-term reductions in ABC and CARS scores than rehabilitation alone. The intervention demonstrated acceptable short-term feasibility.

TRIAL REGISTRATION: The Project was registered on China Clinical Trial Registry (ChiCTR) with the identifier ChiCTR2300075057 ,Registered 24 August 2023, https://www.chictr.org.cn/ .

PMID:42763398 | DOI:10.1186/s12887-026-07708-3

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