Comparison of clinical efficacy and gut microbiota characteristics in children with ASD treated with fecal microbiota transplantation and ketogenic diet.
Both fecal microbiota transplantation and ketogenic diet significantly improved core symptoms in children with ASD, potentially through distinct patterns of gut microbiota modulation.
Key Findings
Results
FMT significantly reduced CARS scores in children with ASD.
Mean CARS score decreased from 34.87 to 33.53 in the FMT group (p < 0.01)
Sample size: 30 participants total diagnosed with ASD according to DSM-5 and ADOS-2
CARS (Childhood Autism Rating Scale) was used to evaluate ASD core symptoms
The FMT group was one of two intervention arms in the study
Results
Ketogenic diet significantly reduced both CARS and ABC scores in children with ASD.
Mean CARS score declined from 35.13 to 33.00 in the KD group (p < 0.01)
Mean ABC score reduced from 63.07 to 42.73 in the KD group (p < 0.01)
The ABC (Aberrant Behavior Checklist) reduction of approximately 20 points represents a substantial improvement
Both CARS and ABC improvements reached statistical significance in the KD group
Results
FMT did not produce a statistically significant improvement in ABC scores.
Mean ABC score reduced from 79.93 to 69.33 in the FMT group (p = 0.064)
The reduction of approximately 10 points did not reach statistical significance
By contrast, the KD group showed a statistically significant ABC reduction (p < 0.01)
The FMT group had a higher baseline ABC score (79.93) compared to the KD group (63.07)
Results
Neither intervention produced statistically significant changes in gut microbiota diversity measures.
No statistically significant changes were observed in α-diversity or β-diversity within either group following intervention
Gut microbiota composition was analyzed by shotgun metagenomic sequencing
This finding held for both the FMT and KD groups
Despite the lack of overall diversity changes, specific taxon-level differences were identified
Results
FMT and ketogenic diet produced distinct post-intervention microbial signatures as revealed by LEfSe analysis.
KD also enriched aromatic compound degradation with Fold enrichment = 3.591, q = 0.010
These functional pathway changes were specific to the KD group
The decrease in Veillonella species (propionate-associated taxa) alongside enrichment of propionate metabolism pathways suggests complex metabolic remodeling
Background
ASD in the study population was accompanied by gastrointestinal dysfunction and gut microbiota dysbiosis, motivating the choice of both interventions.
ASD is characterized by impairments in social communication and interaction, along with restricted, repetitive patterns of behavior
Both FMT and KD were selected as interventions targeting the gut microbiota for ASD
Participants were diagnosed according to DSM-5 and ADOS-2 criteria
The study registered under NCT06348433 (03/21/2024)
What This Means
This research suggests that two very different dietary and microbiome-based treatments — fecal microbiota transplantation (FMT, which transfers gut bacteria from a healthy donor) and the ketogenic diet (a very high-fat, low-carbohydrate diet) — can both lead to measurable improvements in the core symptoms of autism spectrum disorder (ASD) in children. The study enrolled 30 children with ASD and assessed their symptoms using standardized rating scales before and after treatment. Both groups showed reductions in autism severity scores, though the ketogenic diet group showed stronger and more statistically consistent improvements across both symptom measures used.
Interestingly, despite both treatments improving symptoms, they appeared to work through different changes in the gut microbiome. FMT increased the abundance of bacteria known for producing butyrate, a beneficial short-chain fatty acid, while the ketogenic diet altered bacteria involved in propionate metabolism and the breakdown of aromatic compounds. Neither treatment significantly changed the overall diversity of gut bacteria, suggesting the benefits may come from shifts in specific bacterial species or their metabolic activities rather than broad diversity changes.
This research suggests that the gut-brain connection may be an important pathway for influencing ASD symptoms, and that multiple approaches targeting the gut microbiome could be viable. However, the study was small (30 children total), and further larger trials are needed to confirm these findings and to better understand which children might benefit most from each approach.
Wang Y, Wang L, Cai Z, Yu L, Guo Y, Zhang L, et al.. (2026). Comparison of clinical efficacy and gut microbiota characteristics in children with ASD treated with fecal microbiota transplantation and ketogenic diet.. BMC psychiatry. https://doi.org/10.1186/s12888-026-08547-3