Introduction and objective
The therapeutic role of fecal microbiota transplantation in ulcerative colitis varies across different reports. This study aims to evaluate the long-term safety and efficacy of a strategy called step-up fecal microbiota transplantation for ulcerative colitis.
Two clinical trials (NCT01790061, NCT02560727) for moderate-to-severe ulcerative colitis (Mayo score range 6–12) were performed from November 2012 to July 2017. Both studies were pooled for analysis on the safety and efficacy of fecal microbiota transplantation in patients with ulcerative colitis over a 1-year follow-up. The step-up fecal microbiota transplantation strategy included step 1: single fecal microbiota transplantation; step 2: two or more fecal microbiota transplantations; and step 3: fecal microbiota transplantations followed by immunosuppressants. Long-term clinical efficacy and adverse events were assessed, and multiple factors related to fecal microbiota transplantation were evaluated.
Of 134 eligible patients in this real-word study, 81.3% (109/134) were included for analysis. The follow-up ranged from 1 to 5 years. Fecal microbiota transplantation-related adverse events were observed in 17.4% (43/247) of fecal microbiota transplantation procedures including one serious adverse event (myasthenia gravis) and 56 non-serious adverse events. Multivariable logistic regression analysis showed that both the method of preparation of microbiota from stool using the automatic system and the delivery method of colonic transendoscopic enteral tubing were associated with a lower rate of fecal microbiota transplantation-related adverse events (p = 0.023, p = 0.017, respectively). In total, 74.3% (81/109) and 51.4% (56/109) of patients achieved clinical response at 1 month and 3 months after step-up fecal microbiota transplantation, respectively.
Fecal microbiota transplantation should be a safe and promising therapy for ulcerative colitis. The improved fecal microbiota preparation and colonic transendoscopic enteral tubing might reduce the rate of adverse events in ulcerative colitis.
ClinicalTrials.gov NCT01790061, NCT02560727.
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The authors thank the following investigators who participated in this study: Jun Wang (Institute of Microbiology, Chinese Academy of Science), Yongzhan Nie (The Fourth Military Medical University), Kaichun Wu (The Fourth Military Medical University), and Daiming Fan (The Fourth Military Medical University). The authors also thank Dr. Heena Buch for her kind assistance with language improvement.
This study was funded by the publicly donated Intestine Initiative Foundation; Jiangsu Province Creation Team and Leading Talents Project (Zhang F); National Natural Science Foundation of China (81670495, 81600417); Top-Notch Talent Research Projects (LGY2017080); Project of National Health and Family Planning Commission (201502026); and the National Clinical Research Center for Digestive Diseases, Xi’an, China (2015BAI13B07).
Conflict of interest
Faming Zhang invented the concept of GenFMTer and transendoscopic enteral tubing and related devices. Xiao Ding, Qianqian Li, Pan Li, Ting Zhang, Bota Cui, Guozhong Ji, and Xiang Lu have no conflicts of interest that are directly relevant to the content of this study.
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Declaration of Helsinki and its later amendments or comparable ethical standards. This study was reviewed and approved by the Second Affiliated Hospital of the Nanjing Medical University Institutional Ethical Review Board.
Consent to participate
Informed consent was obtained from all individual participants included in the study.
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Ding, X., Li, Q., Li, P. et al. Long-Term Safety and Efficacy of Fecal Microbiota Transplant in Active Ulcerative Colitis. Drug Saf 42, 869–880 (2019). https://doi.org/10.1007/s40264-019-00809-2