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Updated: Nov 28, 2025
Preparation and Maintenance of Bioexclusion IsoPositive Cage Experiment for Human Fecal Transplantation into Germ-Free Mice
Published on: February 28, 2025
Power calculations for detecting differences in efficacy of fecal microbiota donors
1OpenBiome, 2067 Massachusetts Avenue, Cambridge, MA, 02140, USA.
Abstract:
Fecal microbiota transplantation (FMT) is a recommended therapy for recurrent Clostridioides difficile infection and is being investigated as a potential therapy for dozens of other indications, notably inflammatory bowel disease. The immense variability in human stool, combined with anecdotal reports from FMT studies, have suggested the existence of "donor effects", in which stool from some FMT donors is more efficacious than stool from other donors. In this study, simulated clinical trials were used to estimate the number of patients that would be required to detect donor effects under a variety of study designs. In most cases, reliable detection of donor effects required more than 100 patients treated with FMT. These results suggest that previous reports of donor effects need to be verified with results from large clinical trials and that patient biomarkers may be the most promising route to robustly identifying donor effects.
Insights
Fecal microbiota transplantation (FMT) shows promise for various conditions, but detecting "donor effects" requires large trials. Patient biomarkers may offer a more reliable way to identify effective FMT donors.
Area of Science:
- Microbiology
- Gastroenterology
- Clinical Trials
Background:
- Fecal microbiota transplantation (FMT) is a proven therapy for recurrent *Clostridioides difficile* infection.
- FMT is under investigation for numerous other conditions, including inflammatory bowel disease.
- Variability in stool composition and anecdotal evidence suggest potential
- "donor effects" in FMT efficacy.
Purpose of the Study:
- To estimate the sample size needed to detect donor effects in FMT using simulated clinical trials.
- To evaluate different study designs for their ability to identify significant donor effects.
Main Methods:
- Simulated clinical trials were designed to model FMT studies.
- Statistical methods were employed to determine the patient numbers required for detecting donor effects across various scenarios.
Main Results:
- Detecting reliable donor effects in FMT typically requires over 100 patients per trial.
- The study highlights the challenges in confirming donor effects with current study designs.
Conclusions:
- Previous reports of FMT donor effects require validation through large-scale clinical trials.
- Identifying patient biomarkers is a promising strategy for robustly detecting donor effects in FMT.
