Endpoints for Perianal Crohn's Disease Trials: Past, Present and Future

Crohns Colitis. 2021 Aug 2;15(8):1387-1398. doi: 10.1093/ecco-jcc/jjab026.

Bénédicte Caron 1, Ferdinando D'Amico 2 3, Silvio Danese 2 3, Laurent Peyrin-Biroulet 1


Author information

1Department of Gastroenterology and Inserm NGERE U1256, Nancy University Hospital, University of Lorraine, Vandoeuvre-lès-Nancy, France.

2Department of Biomedical Sciences, Humanitas University, Pieve Emanuele, Milan, Italy.

3Department of Gastroenterology, IBD Center, Humanitas Clinical and Research Center, IRCCS, Rozzano, Milan, Italy.


Background and aims: Since the 1980s, many studies have evaluated the efficacy of therapies to improve the outcomes of patients with perianal Crohn's disease. We performed a systematic review to describe the evolution of endpoints in perianal fistulizing Crohn's disease. Efficacy outcomes, definitions and measurement tools were assessed.

Methods: Electronic databases were searched up to November 1, 2020. All published randomized placebo-controlled trials enrolling patients with perianal fistula and Crohn's disease were eligible for inclusion. Ongoing randomized clinical trials were also described.

Results: Nineteen randomized controlled trials were included. Clinical efficacy endpoints were reported in all trials. Clinical response was the most frequent primary endpoint [6/19 studies, 31.6%], followed by clinical remission in four studies [21%]. Clinical response was defined as closure of at least 50% of fistulas, while remission was defined as closure of all fistulas. A combined clinical and radiological primary endpoint was used to assess fistula healing in four studies [21%]. The Perianal Disease Activity Index was a primary endpoint in only one study [5.5%]. In addition, eight ongoing controlled trials were identified. Combined clinical and radiological remission was the most frequent primary endpoint in these studies [4/8, 50%].

Conclusion: In this systematic review, significant changes in outcomes used in randomized clinical trials of perianal Crohn's disease were observed. Radiological endpoints are increasingly used in perianal fistulizing Crohn's disease trials.

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