Selecting End Points for Disease-Modification Trials in Inflammatory Bowel Disease: the SPIRIT Consensus From the IOIBD

Abstract

BACKGROUND AND AIMS: Inflammatory bowel diseases (IBD), encompassing Crohn's disease (CD) and ulcerative colitis (UC), are chronic and disabling disorders. Prospective diseasemodification trials to prevent disease progression are eagerly awaited. However, disease progression is not clearly defined. The objective of the Selecting End PoInts foR Disease-ModIfication Trials (SPIRIT) initiative was to achieve international expert consensus on the endpoints to be used in future IBD-disease modification trials. METHODS: This initiative under the auspices of the International Organization for the Study of Inflammatory Bowel Diseases (IOIBD) began with a systematic literature search to evaluate the current evidence on the definition of disease progression in IBD. On October 22, 2019, a consensus meeting took place during the United European Gastroenterology Week (UEGW) Congress in Barcelona, during which predefined proposed statements were discussed in a plenary session and voted on anonymously. Agreement was defined as at least 75% of participants voting for any one statement. RESULTS: The group agreed that the ultimate therapeutic goal in both CD and UC is to prevent disease impact on patient's life (health-related quality of life, disability, fecal incontinence), midterm complications (encompass bowel damage in CD, IBD-related surgery and hospitalizations, disease extension in UC, extraintestinal manifestations, permanent stoma, short bowel syndrome), and long-term complications (gastrointestinal and extraintestinal dysplasia or cancer, mortality). CONCLUSIONS: Recommendations on which goals to achieve in disease-modification trials for preventing disease progression in patients with IBD are proposed by the SPIRIT consensus. However, these recommendations will require validation in actual clinical studies before implementation in disease-modification trials.

Gut Microbiota Is a Potential Biomarker in Inflammatory Bowel Disease

“…During the last years, the therapeutic targets for IBD are focusing on "target" to treat that target the stage of mucosal healing in intestinal inflammation especially histological healing (88,89). A significant proportion of patients with IBD are resistant to treatment using standard drugs.…”

Biomarker discovery for personalized therapy selection in inflammatory bowel diseases: Challenges and promises

“…Predictive biomarker studies recruiting IBD patients with heterogeneous phenotypes, are more likely to benefit from a holistic and multi-dimensional assessment of therapy effect, which can be a composite of several unidimensional outcome measures. The recent SPIRIT initiative to establish a consensus on outcome measures for disease modification could serve as a starting point towards an objective, holistic and patient-centered outcome assessment that can be further adapted to serve the aims of biomarker studies ( Le Berre and Peyrin-Biroulet, 2021 ). Alternatively, objectivity in assessing the reliability of discovered biomarkers can be ensured by designing studies including subgroups of patients with homogenous phenotypes, where therapy effect can be adequately assessed in each subgroup using the most suitable simple outcome measure.…”

Cutting-Edge Research Trends in Colorectal Disease

“…A continuous upgrade of colorectal disease diagnosis and treatment is mandatory if we want to improve patients' outcomes, and this is not only true when treating a patient with malignant disease but also benign disease. When facing an inflammatory bowel disease (IBD) diagnosis, we can still provide little assurance to patients' questions about their future [3]. How will this affect my life?…”