Farncombe Family Digestive Health Research Institute, Department of Medicine, McMaster University, Hamilton, Ontario, Canada.
Farncombe Family Digestive Health Research Institute, Department of Medicine, McMaster University, Hamilton, Ontario, Canada.
Gastroenterology. 2015 Jul;149(1):102-109.e6. doi: 10.1053/j.gastro.2015.04.001. Epub 2015 Apr 7.
BACKGROUND & AIMS: Ulcerative colitis (UC) is difficult to treat, and standard therapy does not always induce remission. Fecal microbiota transplantation (FMT) is an alternative approach that induced remission in small series of patients with active UC. We investigated its safety and efficacy in a placebo-controlled randomized trial.
We performed a parallel study of patients with active UC without infectious diarrhea. Participants were examined by flexible sigmoidoscopy when the study began and then were randomly assigned to groups that received FMT (50 mL, via enema, from healthy anonymous donors; n = 38) or placebo (50 mL water enema; n = 37) once weekly for 6 weeks. Patients, clinicians, and investigators were blinded to the groups. The primary outcome was remission of UC, defined as a Mayo score ≤2 with an endoscopic Mayo score of 0, at week 7. Patients provided stool samples when the study began and during each week of FMT for microbiome analysis. The trial was stopped early for futility by the Data Monitoring and Safety Committee, but all patients already enrolled in the trial were allowed to complete the study.
Seventy patients completed the trial (3 dropped out from the placebo group and 2 from the FMT group). Nine patients who received FMT (24%) and 2 who received placebo (5%) were in remission at 7 weeks (a statistically significant difference in risk of 17%; 95% confidence interval, 2%-33%). There was no significant difference in adverse events between groups. Seven of the 9 patients in remission after FMT received fecal material from a single donor. Three of the 4 patients with UC ≤1 year entered remission, compared with 6 of 34 of those with UC >1 year (P = .04, Fisher's exact test). Stool from patients receiving FMT had greater microbial diversity, compared with baseline, than that of patients given the placebo (P = .02, Mann-Whitney U test).
FMT induces remission in a significantly greater percentage of patients with active UC than placebo, with no difference in adverse events. Fecal donor and time of UC appear to affect outcomes. ClinicalTrials.gov Number: NCT01545908.
溃疡性结肠炎（UC）难以治疗，标准疗法并不总能诱导缓解。粪便微生物群移植（FMT）是一种替代方法，在小系列活动性 UC 患者中诱导缓解。我们在一项安慰剂对照随机试验中研究了它的安全性和有效性。
我们对无感染性腹泻的活动性 UC 患者进行了一项平行研究。研究开始时，参与者接受了灵活的乙状结肠镜检查，然后随机分为 FMT 组（50 mL，来自健康匿名供体的灌肠；n = 38）或安慰剂组（50 mL 水灌肠；n = 37），每周一次，持续 6 周。患者、临床医生和研究人员对分组情况均不知情。主要结局是在第 7 周时 UC 缓解，定义为 Mayo 评分≤2，内镜 Mayo 评分 0。患者在研究开始时和每周接受 FMT 期间提供粪便样本，进行微生物组分析。数据监测和安全委员会因无效而提前终止试验，但已入组的所有患者均被允许完成研究。
70 例患者完成了试验（安慰剂组 3 例脱落，FMT 组 2 例脱落）。接受 FMT 的 9 例患者（24%）和接受安慰剂的 2 例患者（5%）在 7 周时缓解（风险差异有统计学意义 17%；95%置信区间，2%-33%）。两组间不良反应无显著差异。9 例 FMT 后缓解的患者均接受了来自单一供体的粪便物质。UC ＜1 年的 4 例患者中有 3 例缓解，UC ＞1 年的 34 例患者中有 6 例缓解（P =.04，Fisher 确切检验）。与安慰剂组相比，接受 FMT 的患者粪便微生物多样性较基线时显著增加（P =.02，Mann-Whitney U 检验）。
FMT 诱导活动性 UC 患者缓解的比例显著高于安慰剂，不良反应无差异。粪便供体和 UC 发病时间似乎影响结局。临床试验注册号：NCT01545908。
