Fecal microbiota transplant (FMT) improved disease activity and inflammation in children with ulcerative colitis (UC) better than placebo, according to a study in Gastroenterology.
The study included 25 patients (age range, 4-17 years) with active UC who were randomly assigned to either FMT (n=13) or a placebo (n=12). During the trial, approximately 54% of patients in the FMT group received anti-tumor necrosis factor (n=3) or immunomodulator (n=4) agents vs 17% of patients in the placebo arm (n=1 for each). A total of 7 patients initially randomized to placebo later crossed over to the open-label FMT group.
A higher proportion of patients in the FMT arm reached the composite clinical endpoint of improvement in pediatric UC activity index, C-reactive protein, or fecal calprotectin (92% vs 50%; risk ratio [RR], 1.8; 95% CI, 1.1-3.7). Bacterial taxa associated with achievement of the composite clinical outcome included Alistipes spp and Escherichia spp.
The majority of patients assigned to FMT (75%) maintained a clinical response up to 1 year. There was a trend toward higher beta-diversity from baseline to week 6 in the FMT group compared with the placebo arm.
During the 6-week treatment period, a higher proportion of patients in the FMT group experienced ≥1 adverse event (83.3% vs 41.7%; P =.68). A total of 3 patients in the FMT group and 1 patient in the placebo arm experienced worsening colitis that required hospitalization with intravenous methylprednisolone.
A secondary analysis that compared all recipients of FMT, including those who crossed over to the active intervention after placebo, also showed a higher proportion of FMT-patients achieving the composite clinical endpoint at week 6 (84% vs 50%; RR, 1.7; 95% CI, 1.0-3.4).
Limitations of this study included the lack of endoscopic outcomes as well as the study’s failure to reach its recruitment target, resulting in a small cohort.
The investigators noted that despite the study’s limitations, the “trial offers the first pilot randomized controlled trial evidence that FMT may have an important role in improving symptoms and inflammatory indices in pediatric UC.”
Pai N, Popov J, Hill L, et al. Results of the first pilot randomized controlled trial of fecal microbiota transplant in pediatric ulcerative colitis: lessons, limitations, and future prospects. Published online May 4, 2021. Gastroenterol. doi: 10.1053/j.gastro.2021.04.067