Efficacy and Safety of Fecal Microbiota Transplantation in the Treatment of Acute and Chronic Steroid-Refractory Graft-Versus-Host Disease after Hematopoietic Stem Cell Transplantation

Despite major improvements in allogeneic hematopoietic stem cell transplantation over the last decades, steroid-refractory (SR) acute (a) and chronic (c) graft-versus-host disease (GVHD) cause high mortality. SR-GVHD is a therapeutic challenge. Increasing evidence reveals that gut microbiota play a significant role in GVHD. Several small sample clinical trials indicates fecal microbiota transplantation (FMT) is the viable solution for SR-GVHD.In this retrospective survey, we reported outcome data from 29 patients aged 16-61 years who had underwent a total of 44 FMTs (range, 1-3) as salvage-therapy for SR-GVHD. Patients were classified as having SR-aGVHD (n=18, all grade II or III, all gastrointestinal tract involvement) or SR-cGVHD (n=11, all moderate or severe, progressive involvement of the gastrointestinal tract, lungs, liver, skin and mouth). The median number of prior systemic GVHD-therapies was 3 for both SR-aGVHD (range, 1-4) and SR-cGVHD (range, 1-5). The overall response rate (ORR) was 55.6% (10/18) in SR-aGVHD including 7 CRs (38.9%), while for SR-cGVHD the ORR was 54.5% (6/11, all partial responses). Among the complications of FMT with a possible cause-effect relationship there were: 11 fever, 3 fecal bacterial culture positive, 2 constipation. No serious adverse events clearly related to FMT occurred.FMT may constitute a promising new treatment option for SR-aGVHD and SR-cGVHD that should be validated in a prospective trial.