A Systematic Review on Long-Term Efficacy Outcome Measures in Crohn’s Disease Patients

Abstract

Introduction The traditional long-term treatment goal of Crohn’s disease(CD) is maintenance of corticosteroid free clinical remission. Additional treatment targets, such as biochemical, endoscopic, and patient reported remission, are advocated. The relapsing-remitting nature of CD posts a challenge to the timing of target assessment. Cross-sectional assessment on predetermined moments disregards the health state in between measurements.. In this systematic review, we provide an overview of outcomes used to assess long-term efficacy in clinical trials in CD. Methods A systematic search of PubMed and EMBASE databases was performed to identify clinical trials in luminal CD reporting on maintenance treatment (strategies) since 1995. Two independent reviewers selected eligible articles for full text retrieval, and assessed if long-term corticosteroid free clinical, biochemical, endoscopic or patient reported efficacy outcomes were used. Results The search resulted in 2452 hits and 82 articles were included. Clinical activity was used in 80 studies (98%) as long-term efficacy outcome, in 21 (26%) of these concomitant corticosteroid use was taken into account. CRP was used in 32 studies (41%), faecal calprotectin in 15 studies (18%), endoscopic activity in 34 studies (41%), and patient reported outcome in 32 studies (39%). In seven studies, clinical, biochemical, endoscopic activity and patient’s perspective were measured. In most studies cross-sectional measures or multiple measurements over time were used. Conclusion In none of the published clinical trials in CD sustained remission on all treatment targets was reported. Cross-sectional outcomes on predetermined moments were widely applied, leading to lack of information regarding sustained corticosteroid free remission for this relapsing-remitting chronic disease.

Abdominal pain in patients with inflammatory bowel disease in remission: A prospective study on contributing factors

“…Abdominal pain is frequently used in clinical activity scores to determine disease activity, even though abdominal pain is prevalent in both IBD patients in remission and with active disease. Many clinical trials, including registration trials, use clinical activity scores to select patients for inclusion and/or use clinical activity scores as an outcome, often as the only long‐term outcome measure 43 . As abdominal pain, but also other gastrointestinal symptoms, are frequent in patients with IBD without ongoing inflammation, this may lead to misclassification of patients and eventually misinterpretation of these trials.…”