Kao Dina, Roach Brandi, Silva Marisela, Beck Paul, Rioux Kevin, Kaplan Gilaad G, Chang Hsiu-Ju, Coward Stephanie, Goodman Karen J, Xu Huiping, Madsen Karen, Mason Andrew, Wong Gane Ka-Shu, Jovel Juan, Patterson Jordan, Louie Thomas
Division of Gastroenterology, Department of Medicine, University of Alberta, Edmonton, Alberta, Canada.
Division of Infectious Diseases, Department of Medicine, University of Calgary, Calgary, Alberta, Canada.
JAMA. 2017 Nov 28;318(20):1985-1993. doi: 10.1001/jama.2017.17077.
Fecal microbiota transplantation (FMT) is effective in preventing recurrent Clostridium difficile infection (RCDI). However, it is not known whether clinical efficacy differs by route of delivery.
To determine whether FMT by oral capsule is noninferior to colonoscopy delivery in efficacy.
DESIGN, SETTING, AND PARTICIPANTS: Noninferiority, unblinded, randomized trial conducted in 3 academic centers in Alberta, Canada. A total of 116 adult patients with RCDI were enrolled between October 2014 and September 2016, with follow-up to December 2016. The noninferiority margin was 15%.
Participants were randomly assigned to FMT by capsule or by colonoscopy at a 1:1 ratio.
The primary outcome was the proportion of patients without RCDI 12 weeks after FMT. Secondary outcomes included (1) serious and minor adverse events, (2) changes in quality of life by the 36-Item Short Form Survey on a scale of 0 (worst possible quality of life) to 100 (best quality of life), and (3) patient perception on a scale of 1 (not at all unpleasant) to 10 (extremely unpleasant) and satisfaction on a scale of 1 (best) to 10 (worst).
Among 116 patients randomized (mean [SD] age, 58 [19] years; 79 women [68%]), 105 (91%) completed the trial, with 57 patients randomized to the capsule group and 59 to the colonoscopy group. In per-protocol analysis, prevention of RCDI after a single treatment was achieved in 96.2% in both the capsule group (51/53) and the colonoscopy group (50/52) (difference, 0%; 1-sided 95% CI, -6.1% to infinity; P < .001), meeting the criterion for noninferiority. One patient in each group died of underlying cardiopulmonary illness unrelated to FMT. Rates of minor adverse events were 5.4% for the capsule group vs 12.5% for the colonoscopy group. There was no significant between-group difference in improvement in quality of life. A significantly greater proportion of participants receiving capsules rated their experience as "not at all unpleasant" (66% vs 44%; difference, 22% [95% CI, 3%-40%]; P = .01).
Among adults with RCDI, FMT via oral capsules was not inferior to delivery by colonoscopy for preventing recurrent infection over 12 weeks. Treatment with oral capsules may be an effective approach to treating RCDI.
clinicaltrials.gov Identifier: NCT02254811.
粪便微生物群移植（FMT）在预防艰难梭菌反复感染（RCDI）方面有效。然而，尚不清楚临床疗效是否因给药途径而异。
确定口服胶囊粪菌移植在疗效上是否不劣于结肠镜给药。
设计、地点和参与者：在加拿大艾伯塔省的3个学术中心进行的非劣效性、非盲、随机试验。2014年10月至2016年9月共纳入116例RCDI成年患者，随访至2016年12月。非劣效性界值为15%。
参与者按1:1的比例随机分配接受胶囊粪菌移植或结肠镜粪菌移植。
主要结局是粪菌移植后12周无RCDI的患者比例。次要结局包括：（1）严重和轻微不良事件；（2）通过36项简短问卷调查得出的生活质量变化，范围为0（最差生活质量）至100（最佳生活质量）；（3）患者感受评分，范围为1（一点也不难受）至10（极其难受），以及满意度评分，范围为1（最佳）至10（最差）。
在116例随机分组的患者中（平均[标准差]年龄为58[19]岁；79例女性[68%]），105例（91%）完成试验，其中57例患者随机分配至胶囊组，59例至结肠镜组。在符合方案分析中，胶囊组（51/53）和结肠镜组（50/52）单次治疗后预防RCDI的比例均为96.2%（差异为0%；单侧95%置信区间为-6.1%至无穷大；P < .001），符合非劣效性标准。每组各有1例患者死于与粪菌移植无关的基础心肺疾病。胶囊组轻微不良事件发生率为5.4%，结肠镜组为12.5%。两组间生活质量改善情况无显著差异。接受胶囊治疗的参与者中，将其体验评为“一点也不难受”的比例显著更高（66%对44%；差异为22%[95%置信区间为3%-40%]；P = .01）。
在患有RCDI的成年人中，口服胶囊粪菌移植在预防12周内反复感染方面不劣于结肠镜给药。口服胶囊治疗可能是治疗RCDI的一种有效方法。
clinicaltrials.gov标识符：NCT02254811。
