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Safety and efficacy of fecal microbiota transplantation in the treatment of graft-versus-host disease

Abstract

This article evaluates the efficacy and safety of FMT in the treatment of GVHD after HSCT using a systematic literature search to conduct a meta-analysis constructed of studies involving GVHD patients treated with FMT. 23 studies were included, among which 2 prospective cohort studies, 10 prospective single arm studies, 2 retrospective single arm studies, 2 case series and 7 case reports, comprise a total of 242 patients with steroid-resistant or steroid-dependent GVHD secondary to HSCT who were treated with FMT. 100 cases achieved complete responses, while 61 cases showed partial responses, and 81 cases presented no effect after FMT treatment. The estimate of clinical remission odds ratio was 5.51 (95% CI 1.49–20.35) in cohort studies, and the pooled clinical remission rate is 64% (51–77%) in prospective single arm studies and 81% (62–95%) in retrospective studies, case series and case reports. Five (2.1%) patients had FMT-related infection events, but all recovered after treatment. Other adverse effects were mild and acceptable. Microbiota diversity and composition, donor type, and other related issues were also analyzed. The data proves that FMT is a promising treatment modality of GVHD, but further validation of its safety and efficacy is still needed with prospective control studies.

Clinical trial registration: Registered in https://www.crd.york.ac.uk/PROSPERO/CRD42022296288

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Fig. 1: Flow diagram of the search strategy and included studies according to PRISMA.
Fig. 2: Forest plot of the cohort studies describing the therapeutic effect (complete response and partial response) of fecal microbiota transplantation (FMT) to treat graft-versus-host disease.
Fig. 3: Forest plot of the prospective single arm studies describing the therapeutic effect (complete response and partial response) of fecal microbiota transplantation to treat graft-versus-host disease.
Fig. 4: Forest plot of the combination of retrospective studies (2), case series (2) and case reports (7) describing the effect of fecal microbiota transplantation to treat graft-versus-host disease.

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Acknowledgements

We would like to thank Prof Karolina Skonieczna-Żydecka for her consultancy.

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These authors contributed equally to this work. XQ had contributions to the search of the databases and the implementation of the statistical analysis, the quality assessment of the studies, the acquisition and summarize of the information of the studies and writing of the paper. LW had contributions to the quality assessment of the studies and the writing of the paper. TY contributed to the acquisition and summarize of the information of the studies as well as writing of the paper. RL, YF contributed to the revision of the paper and the connection of authors of included studies. JB, GY contributed to the study conception and design, revision of the paper, interpretation of the results and discussion of the review. All authors read and approved the final paper.

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Correspondence to Jarosław Biliński or Guibin Yang.

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Qiao, X., Biliński, J., Wang, L. et al. Safety and efficacy of fecal microbiota transplantation in the treatment of graft-versus-host disease. Bone Marrow Transplant (2022). https://doi.org/10.1038/s41409-022-01824-1

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