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Published on: April 22, 2015
Fecal Microbiota Transplantation for Autism Spectrum Disorder in Children: Results from a Prospective Open-Label
Dominykas Varnas1, Arnas Kunevičius2, Aurelijus Burokas2
1Clinic of Children's Diseases, Faculty of Medicine, Vilnius University, 03101 Vilnius, Lithuania.
Insights
Fecal microbiota transplantation (FMT) shows promise for improving gastrointestinal and caregiver-reported autism spectrum disorder (ASD) symptoms in children, though not core diagnostic measures. Further research is needed to confirm these findings.
Area of Science:
- Neuroscience
- Gastroenterology
- Microbiology
Background:
- Autism spectrum disorder (ASD) is a growing global concern.
- Gut microbiota dysbiosis is implicated in ASD.
- Limited data exists on fecal microbiota transplantation (FMT) for pediatric ASD.
Purpose of the Study:
- To evaluate colonoscopic FMT efficacy in children with ASD.
- To assess impact on gastrointestinal and ASD-related symptoms.
Main Methods:
- Prospective, single-center, open-label controlled study.
- 30 children with ASD underwent FMT or served as controls.
- Assessed using GSRS, ADOS, CARS, CBCL, and PGI-R scales.
Main Results:
- No significant difference in ADOS scores between groups at 8 weeks.
- FMT group showed improved CARS, PGI-R, CBCL Internalizing Problems, and GSRS.
- Improvements in CARS and PGI-R persisted at 6 months; CARS, GSRS, PGI-R sustained up to 18 months in the FMT group.
- No serious adverse events; three mild events reported.
Conclusions:
- Colonoscopic FMT improved short-term GI and caregiver-reported ASD symptoms, but not ADOS scores.
- Some benefits were long-term.
- Findings are exploratory due to lack of blinding and potential bias; larger RCTs are required.
Abstract:
Background and Objectives: 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. Materials and Methods: 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. Results: 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 (p < 0.001), PGI-R (p < 0.001), CBCL Internalizing Problems (p = 0.001), and GSRS (p = 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. Conclusions: 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.
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