Fecal Microbiota Transplantation for Autism Spectrum Disorder in Children: Results from a Prospective Open-Label Controlled Observational Study.

Researchers studied whether fecal microbiota transplantation (transferring healthy gut bacteria) could help children with autism. 30 children participated - 15 received the treatment, 15 didn't. The main autism test didn't show improvement, but parents reported better autism behaviors, fewer behavioral problems, and improved gut symptoms in treated children. Some benefits lasted up to 18 months. Three children had mild side effects that resolved on their own.

More rigorous studies are needed to confirm these promising but preliminary results.

This prospective open-label controlled study investigated fecal microbiota transplantation (FMT) in 30 children with autism spectrum disorder (15 intervention, 15 control). While the primary endpoint of ADOS score improvement was not met at 8 weeks, the intervention group showed significant improvements in CARS autism ratings, parent global impressions, behavioral problems, and gastrointestinal symptoms compared to controls. Some benefits persisted at 6 months, with sustained improvements in CARS, gastrointestinal symptoms, and parent ratings continuing up to 18 months within the intervention group. Three mild, self-limited adverse events occurred.

The researchers emphasize these are exploratory findings requiring larger randomized controlled trials due to lack of blinding and potential selection bias.

Results suggest potential therapeutic role for FMT in autism, particularly for gastrointestinal symptoms and parent-reported behaviors. However, lack of improvement in gold-standard ADOS scores and methodological limitations mean clinical application is premature. Larger, blinded randomized controlled trials with standardized protocols are essential before considering FMT as a therapeutic option for autism.

Open-label design without blinding introduces significant bias risk. Small sample size (n=30) limits statistical power. Possible selection bias acknowledged. Single-center study limits generalizability. Lack of standardized FMT protocols. Authors explicitly state findings should be considered exploratory.

: Autism spectrum disorder (ASD) is a prevalent neurodevelopmental disorder with an increasing global incidence. Gut microbiota dysbiosis is believed to be playing a role in ASD pathogenesis. Fecal microbiota transplantation (FMT) is emerging as a potential therapeutic strategy to alleviate ASD-related and gastrointestinal symptoms, but data in pediatric ASD populations remain limited.: We conducted a prospective, single-center, open-label controlled study to evaluate the efficacy of colonoscopic FMT in children with ASD. Participants were allocated to two groups: an intervention group that underwent a single FMT procedure and a control group.

Gastrointestinal Symptoms Rating Scale (GSRS), Autism Diagnostic Observation Schedule (ADOS), Childhood Autism Rating Scale (CARS), Child Behavior Checklist (CBCL), and Parent Global Impression (PGI-R) scales were assessed for both groups at baseline and at set time points.: 30 participants were enrolled, with 15 in each group. At 8 weeks, no significant between-group differences were observed for the prespecified primary endpoint, change in ADOS scores. The intervention group showed significantly greater improvements in CARS (< 0.001), PGI-R (< 0.001), CBCL Internalizing Problems (= 0.001), and GSRS (= 0.037) compared with controls; CARS and PGI-R improvements persisted at 6 months. Within the intervention group, sustained improvements were noted in CARS, GSRS, and PGI-R up to 18 months.

No serious adverse events were observed; three mild, self-limited adverse events were recorded following FMT.: Colonoscopic FMT was associated with significant short-term improvements in gastrointestinal and caregiver-reported ASD symptoms (CARS), but not in ADOS scores. Some effects persisted long-term. However, due to a lack of blinding and possible selection bias, these findings should be interpreted as exploratory. Larger randomized controlled trials are needed to confirm efficacy and optimize protocols.