TY - JOUR
T1 - Patient-Reported Outcome Measures for Use in Clinical Trials and Clinical Practice in Inflammatory Bowel Diseases
T2 - A Systematic Review
AU - de Jong, Marin J.
AU - Huibregtse, Roxanne
AU - Masclee, Ad A. M.
AU - Jonkers, Daisy M. A. E.
AU - Pierik, Marie J.
PY - 2018/5
Y1 - 2018/5
N2 - BACKGROUND & AIMS: Mucosal inflammation must be carefully monitored to improve the long-term outcomes of patients with inflammatory bowel diseases (IBD). Patient-reported outcome measures (PROMs) are used increasingly to monitor disease activity in clinical practice and as endpoints in clinical trials. We performed a systematic review to provide an overview of the available PROMs on IBD activity and to evaluate their diagnostic value.METHODS: A systematic search of the PubMed, Medline, Cochrane library, and Embase databases using defined keywords, identified 973 articles. These were screened by 2 independent reviewers, and 37 articles on development or validation ofPROMsto assess IBDactivity were identified for further analysis. Based on the recommendations of the Food and Drug Administration (FDA), the following measurement properties were evaluated: content, construct, and criterionvalidity; reliability; and responsiveness to change. In addition, data on ease of use in clinical practice were collected.RESULTS: Seventeen articles presenting 20 different PROMs were included the final analysis, although none met all the FDA-recommended criteria. Only 2 PROMs (patient-reported Harvey Bradshaw Index and Simple Clinical Colitis Activity Index scores) reported patient involvement during its development. Only 6 PROMs (patient-reported global assessment, patient assessment of disease activity, mobile health index for Crohn's disease, mobile health index for ulcerative colitis, patient-reported outcome derived from the Mayo score, and the 6-point Mayo score) were validated as markers of IBD activity, using findings from endoscopy as the reference standard; these PROMs identified patients with mucosal inflammation with area under the curve values of 0.63-0.82. The mobile health index for CD and UC scores had the best measurement properties for use in clinical practice and in clinical trials.CONCLUSIONS: In a systematic review, we identified more than 20 PROMS that have been developed and tested for their ability to determine IBD activity. Further studies are needed to determine their accuracy and whether they can be used effectively in routine practice, clinical trials, telemedicine systems, and value-based healthcare programs.
AB - BACKGROUND & AIMS: Mucosal inflammation must be carefully monitored to improve the long-term outcomes of patients with inflammatory bowel diseases (IBD). Patient-reported outcome measures (PROMs) are used increasingly to monitor disease activity in clinical practice and as endpoints in clinical trials. We performed a systematic review to provide an overview of the available PROMs on IBD activity and to evaluate their diagnostic value.METHODS: A systematic search of the PubMed, Medline, Cochrane library, and Embase databases using defined keywords, identified 973 articles. These were screened by 2 independent reviewers, and 37 articles on development or validation ofPROMsto assess IBDactivity were identified for further analysis. Based on the recommendations of the Food and Drug Administration (FDA), the following measurement properties were evaluated: content, construct, and criterionvalidity; reliability; and responsiveness to change. In addition, data on ease of use in clinical practice were collected.RESULTS: Seventeen articles presenting 20 different PROMs were included the final analysis, although none met all the FDA-recommended criteria. Only 2 PROMs (patient-reported Harvey Bradshaw Index and Simple Clinical Colitis Activity Index scores) reported patient involvement during its development. Only 6 PROMs (patient-reported global assessment, patient assessment of disease activity, mobile health index for Crohn's disease, mobile health index for ulcerative colitis, patient-reported outcome derived from the Mayo score, and the 6-point Mayo score) were validated as markers of IBD activity, using findings from endoscopy as the reference standard; these PROMs identified patients with mucosal inflammation with area under the curve values of 0.63-0.82. The mobile health index for CD and UC scores had the best measurement properties for use in clinical practice and in clinical trials.CONCLUSIONS: In a systematic review, we identified more than 20 PROMS that have been developed and tested for their ability to determine IBD activity. Further studies are needed to determine their accuracy and whether they can be used effectively in routine practice, clinical trials, telemedicine systems, and value-based healthcare programs.
KW - IBD
KW - HBI
KW - SCCAI
KW - PRO2
KW - Marker
KW - COLITIS ACTIVITY INDEX
KW - ULCERATIVE-COLITIS
KW - CROHNS-DISEASE
KW - MUCOSAL INFLAMMATION
KW - HEALTH-STATUS
KW - MAYO SCORE
KW - TECHNOLOGIES
KW - VALIDATION
KW - REMISSION
KW - CARE
U2 - 10.1016/j.cgh.2017.10.019
DO - 10.1016/j.cgh.2017.10.019
M3 - (Systematic) Review article
C2 - 29074448
SN - 1542-3565
VL - 16
SP - 648
EP - 663
JO - Clinical gastroenterology and hepatology
JF - Clinical gastroenterology and hepatology
IS - 5
ER -