Comparison of clinical efficacy and gut microbiota characteristics in children with ASD treated with fecal microbiota transplantation and ketogenic diet.

Yu Wang; Lihong Wang; Zhifeng Cai; Lianhu Yu; Yanshuo Guo; Linlin Zhang; Mengna Zhu; Zhiyue Liu; YongHeng Zhao; Lili Liu; Aihua Cao

doi:10.1186/s12888-026-08547-3

PubMed · 42687165

Comparison of clinical efficacy and gut microbiota characteristics in children with ASD treated with fecal microbiota transplantation and ketogenic diet.

Yu Wang·Lihong Wang·Zhifeng Cai·Lianhu Yu·Yanshuo Guo·Linlin Zhang·Mengna Zhu·Zhiyue Liu·YongHeng Zhao·Lili Liu·Aihua Cao

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Abstract

OBJECTIVE: Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder characterized by impairments in social communication and interaction, along with restricted, repetitive patterns of behavior. It is often accompanied by gastrointestinal dysfunction and gut microbiota dysbiosis. Fecal Microbiota Transplantation (FMT) and the Ketogenic Diet (KD) are interventions targeting the gut microbiota for ASD. METHODS: 30 participants were diagnosed with ASD according to DSM-5 and ADOS-2. ASD core symptoms were evaluated with CARS and ABC. Gut microbiota composition was analyzed by shotgun metagenomic sequencing. RESULTS: Both groups demonstrated significant improvements in core symptoms. In the FMT group, the mean CARS score significantly decreased from 34.87 to 33.53 (p < 0.01); in the KD group, it declined from 35.13 to 33 (p < 0.01). The mean ABC score reduced from 79.93 to 69.33 (p = 0.064) in the FMT group and from 63.07 to 42.73 (p < 0.01) in the KD group. Following the intervention, no statistically significant changes were observed in α-diversity or β-diversity within either group. LEfSe analysis revealed distinct post-intervention microbial signatures: FMT significantly enriched butyrate-producing taxa (Wujia chipingensis, Eubacterium sp. MSJ-33, and Butyrivibrio crossotus), while KD elevated Blautia massiliensis and decreased propionate metabolism -associated taxa (Veillonella sp. S12025-13 and Veillonella nakazawae). KEGG enrichment analysis revealed that KD enriched propionate metabolism (Fold enrichment = 3.747, q = 0.010) and aromatic compound degradation (Fold enrichment = 3.591, q = 0.010). CONCLUSIONS: Both interventions significantly improved clinical symptoms among children with ASD, potentially through distinct patterns of gut microbiota modulation. CLINICAL TRIALS NUMBER: NCT06348433 (03/21/2024).

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Child Child, Preschool Female Humans Male Autism Spectrum Disorder Diet, Ketogenic Fecal Microbiota Transplantation Gastrointestinal Microbiome Treatment Outcome Autism spectrum disorder Fecal microbiota transplantation Gut microbiota Ketogenic Diet Short-chain fatty acids Comparative Study Journal Article Randomized Controlled Trial

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Yu Wang, Lihong Wang, Zhifeng Cai, Lianhu Yu, Yanshuo Guo, Linlin Zhang, Mengna Zhu, Zhiyue Liu, YongHeng Zhao, Lili Liu, Aihua Cao. 2026-09-02. Comparison of clinical efficacy and gut microbiota characteristics in children with ASD treated with fecal microbiota transplantation and ketogenic diet.. https://doi.org/10.1186/s12888-026-08547-3

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Autism spectrum disorder subject Child subject Child, Preschool subject Comparative Study subject Fecal Microbiota Transplantation subject Female subject Gastrointestinal Microbiome subject Gut microbiota subject Humans subject Journal Article subject Male subject Randomized controlled trials study

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