FMT of no benefit in systemic sclerosis patients with lower GI tract involvement

Faecal microbiota transplantation (FMT) with standardized anaerobic cultivated human intestinal microbiome is safe but does not appear to be efficacious in patients with systemic sclerosis experiencing lower gastrointestinal (GI) tract symptoms, according to a phase II study.

The study included 67 adult systemic sclerosis patients (mean age 58.91 years, 93 percent women, 75 percent anticentromere antibody positive) with and moderate-to-severe bloating or diarrhoea. These patients were randomly assigned to receive intestinal infusions of anaerobic cultivated human intestinal microbiome (n=33) or placebo (n=34) at weeks 0 and 2, stratified by worst symptom.

Researchers evaluated the change in worst lower gastrointestinal tract symptom (bloating or diarrhoea) from week 0 to week 12, the primary study endpoint, using the University of California Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 scoring system in the intention-to-treat population. They also assessed safety at weeks 0, 2, 4, 6, and 12 in all participants who received at least one infusion.

At week 12, the change in worst lower gastrointestinal tract symptom did not significantly differ between patients who received FMT and those who received placebo (–0.13 vs –0.33; average marginal effect, 0.20, 95 percent confidence interval, –0.12 to 0.52; p=0.22).

Adverse events (AEs) occurred in 48 percent patients in the FMT group and in 56 percent of those in the placebo group. Most AEs were mild and short-lived gastrointestinal tract symptoms. During gastroscopy, one patient was found to have a duodenal perforation.