Fecal microbiota transplantation for graft-versus-host disease in children and adults: methods, clinical effects, safety

Aim.  Was to evaluate clinical efficacy, adverse events and changes in the gut microbiome after fecal microbiota transplantation (FMT) in patients with gastrointestinal (GI) form of graft-versus-host disease (GVHD). Materials and methods.  The prospective single-center study in R.M. Gorbacheva institute included 27 patients with GI GVHD after allogeneic stem cell transplantation. 19 patients received FMT, 8 patients received placebo. Clinical scales for GI autoimmune diseases were used to evaluate response. Microbiome alterations were assessed with multiplex PCR. Results.  After FMT higher overall bacterial mass ( р =0.00088), higher bacterial numbers of  Bifidobacterium  spp. ( р =0.021),  Escherichia coli  ( р =0.049) and  Bacteroides fragilis  gr. ( р =0.000043) compared to placebo group. Also higher bacterial mass was observed in patients with clinical response ( р =0.0057). The bacterial mass after procedure in non-responders was compared to the placebo group ( р =0.31). Partial response of GVHD was achieved faster in the FMT group compared to placebo (median 4 days vs 48 days,  p =0.014). Complete response was observed in 8 (42%), 14 (74%) and 16 (84%) at 30, 60 and 90 days respectively, while in the placebo group only 0%, 1 (13%) and 4 (50%) achieved complete response at the same time points. The incidence and severity of adverse events was comparable between FMT and the placebo group. Conclusion.  FMT in patients with refractory GI GVHD was associated with favorable clinical outcomes and recovery in certain marker bacterial populations. Multiplex PCR can be used to assess an engraftment of a donor microbiota. FMT in GI GVHD was not associated with life-threatening adverse events, but further studies are required to validate clinical efficacy.