Rigorous donor selection for FMT in active ulcerative colitis: KEY lessons from a randomized controlled trial halted for futility.
Abstract
BACKGROUND AND AIMS
Rigorous donor preselection on microbiota level, strict anaerobic processing, and repeated FMT administration were hypothesized to improve FMT induction of remission in UC.
METHODS
The RESTORE-UC trial was a multi-centric, double-blind, sham-controlled, randomized trial. Patients with moderate to severe UC (defined by total Mayo 4-10) were randomly allocated to receive four anaerobic-prepared allogenic or autologous donor FMTs. Allogenic donor material was selected after a rigorous screening based on microbial cell count, enterotype, and the abundance of specific genera. The primary endpoint was steroid-free clinical remission (total Mayo ≤2, no sub-score >1) at week 8. A pre-planned futility analysis was performed after 66% (n=72) of intended inclusions (n=108). Quantitative microbiome profiling (n=44) was performed at weeks 0 and 8.
RESULTS
In total, 72 patients were included of which 66 received at least one FMT (allogenic-FMT n=30 and autologous-FMT n=36). At week 8, respectively 3 and 5 patients reached the primary endpoint of steroid-free clinical remission (p=0.72), indicating no treatment difference of at least 5% in favour of allogenic-FMT. Hence, the study was stopped due to futility. Microbiome analysis showed numerically more enterotype transitions upon allogenic-FMT compared to autologous-FMT and more transitions were observed when patients were treated with a different enterotype than their own at baseline (p=0.01). Primary response was…
How strong is the evidence?
Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 2 of 5 (High · randomized trial). Randomized trials of a reasonable size are the strongest single-study design. Reported size: 108 participants or records.
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