& Aims: Several case series have reported the effects of fecal microbiota transplantation (FMT) for ulcerative colitis (UC). We assessed the efficacy and safety of FMT for patients with UC in a double-blind randomized trial.
Patients with mild to moderately active UC (n=50) were assigned to groups that underwent FMT with feces from healthy donors or were given autologous fecal microbiota (control); each was administered via naso-duodenal tube at the start of the study and 3 weeks later. The study was performed at the Academic Medical Center in Amsterdam from June 2011 through May 2014. The composite primary endpoint was clinical remission (simple clinical colitis activity index scores =2) combined with =1 point decrease in the Mayo endoscopic score at week 12. Secondary endpoints were safety and microbiota composition by phylogenetic microarray in fecal samples.
Thirty-seven patients completed the primary endpoint assessment. In the intention-to-treat analysis, 7/23 patients who received fecal transplants from healthy donors (30.4%) and 5/25 controls (20.0%) achieved the primary endpoint (P=.51). In the per protocol analysis, 7/17 patients who received fecal transplants from healthy donors (41.2%) and 5/20 controls (25.0%) achieved the primary endpoint (P=.29). Serious adverse events occurred in 4 patients (2 in the FMT group) but these were not considered to be related to the FMT. At 12 weeks the microbiota of responders in the FMT group was similar to that of their healthy donors; remission was associated with proportions of Clostridium clusters IV and XIVa.
In this phase 2 trial, there was no statistically significant difference in clinical and endoscopic remission between patients with UC who received fecal transplants from healthy donors and those who received their own fecal microbiota, which may be due to limited numbers. However, the microbiota of responders had distinct features from that of nonresponders, warranting further study. ClinicalTrials.gov no: NCT01650038