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Ustekinumab for fistulising perianal Crohn's disease: a randomised placebo-controlled trial from the GETAID

In brief

Ustekinumab leads to combined remission in 62% versus 25% with placebo

In a double-blind trial of 32 patients with draining perianal fistulas, 62% of those receiving ustekinumab achieved both clinical and MRI remission at 12 weeks compared with 25% on placebo. Most participants had failed anti-TNF therapy, and the advantage waned by week 48, leaving long-term benefit uncertain.

Journal
Gut (Q1)
Published
24 July 2026
Study design
Randomized controlled trial
Evidence level
Level 1, High (CEBM 1b)
Authors
Pauline Wils, Stéphane Nancey, Elodie Messmer, Arnaud Bourreille, Anthony Buisson, Ludovic Caillo, et al.
PMID
42498622
DOI
10.1136/gutjnl-2026-339158

Why clinicians should know about it

Abstract

BACKGROUND: Fistulising perianal Crohn's disease (CD) remains a debilitating condition, with few randomised controlled trials conducted so far. OBJECTIVE: To evaluate the efficacy and safety of ustekinumab (UST) in patients with CD with active draining perianal fistulas (NCT04496063) in a randomised placebo-controlled trial. DESIGN: In this double-blind, multicentre Groupe d'Etudes Therapeutiques des Affections Inflammatoires Digestives trial, patients enrolled with active fistulising perianal CD were randomised 1:1 to receive UST (6 mg/kg intravenously at baseline followed by 90 mg subcutaneously at week 8, then every 8 weeks) or placebo, after standardised surgical management. The primary endpoint was combined clinical remission (absence of drainage from all external fistula openings) and radiological remission (absence of abscesses >2 cm on blinded central MRI) at week 12. At week 12, placebo non-responders could switch to UST, and UST non-responders could be intensified during the open-label phase. RESULTS: 32 patients were randomised (UST: 16; placebo: 16) across 10 French centres and 69% had prior anti-tumour necrosis factor failure. At week 12, combined remission was achieved in 62% with UST versus 25% with placebo (OR=5.1 (95% CI 1.07-24.4)). Clinical remission occurred in 62.5% vs 31% (OR=3.75 (95% CI 0.84 to 16.8)) and radiological remission in 87.5% vs 75% (OR=2.7 (95% CI 0.34 to 21.1)). Nine placebo-patients switched to UST during the open-label phase. Combined remission rates at week 48 were 50% in the placebo arm and 31% in the UST arm, respectively. CONCLUSION: These findings suggest greater short-term clinical benefit with UST compared with placebo and support its use in patients with active fistulising perianal CD. TRIAL REGISTRATION NUMBER: NCT04496063.

Abstract as published, via PubMed.

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For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.