Moayyedi et al. from Canada recently published a paper entitled Fecal Microbiota Transplantation Induces Remission in Patients with Active Ulcerative Colitis in a Randomized, Controlled Trial. Authors investigated the safety and efficacy of fecal microbiota transplantation (FMT) to treat active ulcerative colitis (UC) without infectious diarrhea; they say it is the largest placebo-controlled, randomized trial for FMT to treat any disease.
Patients were examined by flexible sigmoidoscopy and then given weekly treatment for 6 weeks: either FMT from healthy anonymous donors, or a placebo (water enema). The primary outcome was remission of UC.
Halfway through recruitment, the data monitoring and safety committee recommended discontinuing the trial for futility because they ascertained the primary endpoint was not likely to be achieved. However, patients already enrolled were allowed to complete the trial and the final results turned out more favorably than first anticipated.
70 patients completed the trial; 24% of the FMT group and 5% of the placebo group were in remission by the end of the study and there was no difference in adverse events. Researchers found no measurable difference in quality of life scores after the treatment.
A possible donor effect was observed, as 7 of 9 patients in remission after FMT had received stool from the same donor. Patients taking concurrent immunosuppressant therapy may have derived a greater benefit from FMT, and those with a more recent diagnosis appeared to be more responsive.
Microbiota analysis showed more diversity in the treatment group compared to the placebo group after 6 weeks; patients in the active therapy group were more similar to their donor sample than a control fecal sample.
This trial is an important addition to the literature on FMT for conditions other than C. difficile. It highlights the need for 1) stratification of different UC patients in clinical trials, and 2) investigation of specific donor effects that influence FMT success.
Moayyedi P, et al. (2015) Fecal Microbiota Transplantation Induces Remission in Patients with Active Ulcerative Colitis in a Randomized, Controlled Trial. Gastroenterology, doi: 10.1053/j.gastro.2015.04.001