Clinical analysis of fecal microbiota transplantation for refractory acute gastrointestinal graft-versus-host disease after allogeneic hematopoietic stem cell transplantation: a five-year retrospective study.

Abstract

Refractory acute gastrointestinal graft-versus-host disease (GI-aGVHD) is a severe complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT), which is associated with significantly increased mortality. Fecal microbiota transplantation (FMT) has emerged as a promising therapeutic strategy for this condition, and its efficacy and safety have garnered substantial attention in clinical research. To explore the clinical efficacy and safety of FMT for refractory acute GI-aGVHD after allo-HSCT. Data from 22 patients with refractory GI-aGVHD who received FMT and 20 patients who did not undergo FMT, all diagnosed with refractory GI-aGVHD after allo-HSCT and treated at Henan Cancer Hospital between December 2017 and March 2023, were retrospectively analyzed to evaluate short-term efficacy, safety, and long-term survival outcomes. FMT significantly reduced the median diarrhea volume (1300 versus 450 mL, P = 0.017) and frequency (10 versus 5.5 times/d, P = 0.049) in the FMT group, whereas the control group showed no significant improvement in either parameter (diarrhea volume: 1350 versus 900 mL, P > 0.05; frequency: 10 versus 9 times/d, P > 0.05). The FMT group exhibited significantly higher complete response (CR) (31.8% versus 5%, P = 0.047) and overall response rate (ORR) (59.1% versus 25%, P = 0.033) compared with the control group. Subgroup analyses showed that patients with isolated intestinal involvement (ORR = 100%) and those without bloody stool before FMT (ORR = 78.6%) had superior responses to FMT. The FMT group achieved a median progression-free survival (PFS) of 30 (10-2113) days and a median overall survival (OS) of 150 (51-2130) days, with the 100-day, 1-year and 3-year PFS rates of 40.9%, 28.1%, and 28.1%, respectively, and the 100-day, 1-year and 3-year OS rates of 59.1%, 50.0% and 34.3%. In contrast, the control group had notably poorer survival outcomes: median PFS was 23 (7-1820) days, median OS was 80 (20-1901) days, while the 100-day, 1-year, and 3-year PFS rates remained 5% across all time points, and the 100-day, 1-year, and 3-year OS rates were 20.0%, 10.0%, and 10%. FMT is effective and safe as a clinical treatment for refractory GI-aGVHD after allo-HSCT, and the effectiveness of FMT can be used as a prognostic indicator for long-term survival in refractory GI-aGVHD.