Development and Validation of a Patient-reported Score to Screen for Mucosal Inflammation in Inflammatory Bowel Disease.
BACKGROUND AND AIMS
Patient-reported outcome measures [PROMs] assessing inflammatory bowel disease [IBD] activity are of interest for monitoring in clinical practice, telemedicine systems, or trials. Different PROMs for follow-up of disease activity are available; however, none was developed with endoscopy as gold standard. The objective of this study was to develop and validate a PROM to predict endoscopic disease activity, following the recommendations of the Food and Drug Administration.


METHODS
During development, 178 IBD patients undergoing a colonoscopy were asked to fill out 13 clinical questions derived from the literature. During endoscopy, inflammation was assessed with the simplified endoscopic score for Crohn's disease [CD] and the Mayo endoscopic subscore for ulcerative colitis [UC]. Based on correlation with endoscopic inflammation, questions were reduced to a total of six for CD and five for UC. The newly developed Monitor IBD At Home questionnaire [MIAH] was validated in an independent cohort of 135 CD and 131 UC patients. Additionally, diagnostic accuracy of the MIAH combined with a calprotectin home test [CHT] was assessed.


RESULTS
The MIAH-CD includes questions on rectal bleeding, mucus, stool frequency, urgency, fatigue, and patient-reported disease activity. The MIAH-UC contains items on rectal bleeding, stool frequency, urgency, abdominal pain, and patient-reported disease activity. Both questionnaires showed to be valid, reliable, and responsive to changes. The MIAH and CHT combined had a sensitivity, specificity, negative predictive value [NPV], and positive predicitive value [PPV] of 96.7%, 66.7%, 94.7%, and 76.3% for CD and of 88.2%, 81.4%, 95.6%, and 60.0% for UC, respectively, compared with endoscopy.


CONCLUSIONS
The MIAH is the first PROM developed to predict endoscopic inflammation in IBD patients. A combination of this questionnaire and a CHT shows excellent diagnostic accuracy to screen for patients who need further assessment of disease activity, and can be used in daily practice, telemedicine systems, and trials.
['de Jong|Marin J|MJ|', 'Roosen|Danielle|D|', 'Degens|Juliette H R J|JHRJ|', 'van den Heuvel|Tim R A|TRA|', 'Romberg-Camps|Marielle|M|', 'Hameeteman|W|W|', 'Bodelier|Alexander G L|AGL|', 'Romanko|Igor|I|', 'Lukas|Milan|M|', 'Winkens|Bjorn|B|', 'Markus|Tineke|T|', 'Masclee|Ad A M|AAM|', 'van Tubergen|Astrid|A|', 'Jonkers|Daisy M A E|DMAE|', 'Pierik|Marie J|MJ|']
D000328:Adult D003092:Colitis D003093:Colitis, Ulcerative D003113:Colonoscopy D003424:Crohn Disease D005243:Feces D005260:Female D006801:Humans D015212:Inflammatory Bowel Diseases D007413:Intestinal Mucosa D039841:Leukocyte L1 Antigen Complex D008297:Male D008875:Middle Aged D000071066:Patient Reported Outcome Measures D011446:Prospective Studies D015203:Reproducibility of Results D011795:Surveys and Questionnaires
mucosal inflammation PROM IBD