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Patient-Reported Outcomes as Primary End Points in Clinical Trials of Inflammatory Bowel Disease
Abstract
The Food and Drug Administration (FDA) is moving from the Crohn's Disease Activity Index to patient-reported outcomes (PROs) and objective measures of disease, such as findings from endoscopy. PROs will become an important aspect of assessing activity of inflammatory bowel disease (IBD) and for labeling specific drugs for this disease. PROs always have been considered in the management of patients with rheumatoid arthritis or multiple sclerosis, and have included measurements of quality of life, disability, or fatigue. Several disease-specific scales have been developed to assess these PROs and commonly are used in clinical trials. Outcomes reported by patients in clinical trials of IBD initially focused on quality of life, measured by the Short-Form 36 questionnaire or disease-specific scales such as the Inflammatory Bowel Disease Questionnaire or its shorter version. Recently considered factors include fatigue, depression and anxiety, and work productivity, as measured by the Functional Assessment Chronic Illness Therapy-Fatigue, the Hospital Anxiety Depression, and the Work Productivity Activity Impairment Questionnaire, respectively. However, few data are available on how treatment affects these factors in patients with IBD. Although disability generally is recognized in patients with IBD, it is not measured. The international IBD disability index currently is being validated. None of the PROs currently used in IBD were developed according to FDA guidance for PRO development. PROs will be a major primary end point of future trials. FDA guidance is needed to develop additional PROs for IBD that can be incorporated into trials, to better compare patients' experience with different therapies.
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“Given such convincing evidence of a psychological component and impact in and of IBD, psychological support is now considered an integral component of modern IBD management [16,17].”
Citations
Cites
The Hospital Anxiety and Depression Scale
Zigmond, A. S., & Snaith, R. P. (1983) · Acta Psychiatrica Scandinavica · doi:10.1111/j.1600-0447.1983.tb09716.x · Cited by 47,139
An inventory for measuring depression
BECK, A. T. (1961) · Archives of General Psychiatry · doi:10.1001/archpsyc.1961.01710120031004 · Cited by 38,032
(1992) · PubMed · Cited by 33,716
The MOS 36-ltem Short-Form Health Survey (SF-36): I. Conceptual Framework and Item Selection
Ware, J. E., & Sherbourne, C. D. (1992) · Medical Care · doi:10.1097/00005650-199206000-00002 · Cited by 30,033
(1970) · Cited by 24,983
Rating neurologic impairment in multiple sclerosis: An expanded disability status scale (EDSS)
Kurtzke, J. F. (1983) · Neurology · doi:10.1212/wnl.33.11.1444 · Cited by 15,011
Krupp, L. B. (1989) · Archives of Neurology · doi:10.1001/archneur.1989.00520460115022 · Cited by 6,172
Measurement of patient outcome in arthritis
Fries, J. F., Spitz, P. W., Kraines, R. G., & Holman, H. R. (1980) · Arthritis & Rheumatism · doi:10.1002/art.1780230202 · Cited by 4,404
Smets, E. M. A., Garssen, B., Bonke, B., & de Haes, J. C. J. M. (1995) · Journal of Psychosomatic Research · doi:10.1016/0022-3999(94)00125-o · Cited by 3,622
Development and validation of the State Hope Scale.
Snyder, C. R. R., Sympson, S. C., Ybasco, F. C., Borders, T. F., Babyak, M. A., & Higgins, R. L. (1996) · Journal of Personality and Social Psychology · doi:10.1037/0022-3514.70.2.321 · Cited by 3,584
Flagged first, then by citations
Research works printed but not linked: 2
- Piper, 1990, Piper fatigue scale available for clinical testing, Oncol Nurs Forum, 17, 661
- Masachs, 2007, [Spanish translation, adaptation, and validation of the 32-item questionnaire on quality of life for inflammatory bowel disease(IBDQ-32)], Rev Esp Enferm Dig, 99, 511
Cited by
Sorted by citations
Peyrin‐Biroulet, L., Sandborn, W. J., Sands, B. E., Reinisch, W., Bemelman, W. A., Bryant, R. V., D’Haens, G., Dotan, I., Dubinsky, M. C., Feagan, B. G., et al. (2015) · The American Journal of Gastroenterology · doi:10.1038/ajg.2015.233 · Cited by 1,902
ACG Clinical Guideline: Management of Crohn's Disease in Adults
Lichtenstein, G. R., Loftus, E. V., Isaacs, K. L., Regueiro, M. D., Gerson, L. B., & Sands, B. E. (2018) · The American Journal of Gastroenterology · doi:10.1038/ajg.2018.27 · Cited by 1,424
Quality of life in inflammatory bowel disease: a systematic review and meta-analyses—part I
Knowles, S. R., Graff, L. A., Wilding, H., Hewitt, C., Keefer, L., & Mikocka-Walus, A. (2018) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izx100 · Cited by 503
Sturm, A., Maaser, C., Calabrese, E., Annese, V., Fiorino, G., Kucharzik, T. F., Vavricka, S. R., Verstockt, B., van Rheenen, P., Tolan, D. J. M., et al. (2018) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjy114 · Cited by 462
Defining Disease Severity in Inflammatory Bowel Diseases: Current and Future Directions
Peyrin‐Biroulet, L., Panés, J., Sandborn, W. J., Vermeire, S., Danese, S., Feagan, B. G., Colombel, J., Hanauer, S. B., & Rycroft, B. (2015) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2015.06.001 · Cited by 455
Quality of life in inflammatory bowel disease: a systematic review and meta-analyses—part II
Knowles, S. R., Keefer, L., Wilding, H., Hewitt, C., Graff, L. A., & Mikocka-Walus, A. (2018) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izy015 · Cited by 416
Systematic review with meta-analysis: The efficacy of probiotics in inflammatory bowel disease
Derwa, Y., Gracie, D. J., Hamlin, P. J., & Ford, A. C. (2017) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.14203 · Cited by 393
“These previous systematic reviews also pooled data from RCTs that did not meet our inclusion criteria, including studies that examined the effect of probiotics in children with IBD, and the effect of synbiotics in IBD.28,29 Studies investigating the effect of manipulation of the micro- biome on disease activity in IBD…”
BackgroundInfluentialWilliams, J. G., Alam, M. F., Alrubaiy, L., Arnott, I. D., Clement, C., Cohen, D. R., Gordon, J. N., Hawthorne, A. B., Hilton, M., Hutchings, H. A., et al. (2016) · The Lancet. Gastroenterology & hepatology · doi:10.1016/s2468-1253(16)30003-6 · Cited by 271
Colombel, J., Reinisch, W., Mantzaris, G. J., Kornbluth, A. A., Rutgeerts, P., Tang, K. L., Oortwijn, A. F., Bevelander, G. S., Cornillie, F. J., & Sandborn, W. J. (2015) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.13139 · Cited by 198
“As such, the Food and Drug Administration is moving away from the CDAI score requiring a co-primary endpoint comprised of a pure patient-reported outcome measure in combination with an endoscopic measure of disease activity.(25)”
BackgroundWynne, B., McHugh, L., Gao, W., Keegan, D., Byrne, K., Rowan, C., Hartery, K., Kirschbaum, C., Doherty, G., Cullen, G., et al. (2019) · Gastroenterology · doi:10.1053/j.gastro.2018.11.030 · Cited by 188
“have stressed the value of mental health indices as patient reported outcomes in clinical trials (13), while psychological services are now considered an essential component of contemporary IBD therapy (14,15).”
BackgroundGracie, D. J., Williams, C. J. M., Sood, R., Mumtaz, S., Bholah, H. M., Hamlin, J. P., & Ford, A. C. (2016) · American Journal of Gastroenterology · doi:10.1038/ajg.2016.59 · Cited by 173
Doherty, M. K., Ding, T., Koumpouras, C. C., Telesco, S., Monast, C. S., Das, A., Brodmerkel, C. M., & Schloss, P. D. (2018) · mBio · doi:10.1128/mbio.02120-17 · Cited by 168
“While the field appears to be moving away from CDAI and toward patient-reported outcomes and more objectively quantifiable measures such as endoscopic verification of mucosal healing (21, 46), research is ongoing to discover less invasive and more quantifiable biomarkers (24, 25, 36).”
Patient-reported Outcomes in a French Nationwide Survey of Inflammatory Bowel Disease Patients
Williet, N., Sarter, H., Gower‐Rousseau, C., Adrianjafy, C., Olympie, A., Buisson, A., Beaugerie, L., & Peyrin‐Biroulet, L. (2016) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjw145 · Cited by 162
Validation of the Inflammatory Bowel Disease Disability Index in a population-based cohort
Gower‐Rousseau, C., Sarter, H., Savoye, G., Tavernier, N., Fumery, M., Sandborn, W. J., Feagan, B. G., Duhamel, A., Guillon-Dellac, N., Colombel, J., et al. (2015) · Gut · doi:10.1136/gutjnl-2015-310151 · Cited by 157
Ghosh, S. K., Louis, E. J., Beaugerie, L., Bossuyt, P. J., Bouguen, G., Bourreille, A., Ferrante, M., Franchimont, D. P., Frost, K., Hébuterne, X., et al. (2017) · Inflammatory Bowel Diseases · doi:10.1097/mib.0000000000001033 · Cited by 142
“Body image: problem with the way your body or body parts look (11) Body image: not liking the way body or body parts look Body image: not liking the way body or body parts look”
Efficacy of Medical Therapies for Fistulizing Crohn’s Disease: Systematic Review and Meta-analysis
Lee, M. J., Parker, C. E., Taylor, S. R., Guizzetti, L., Feagan, B. G., Lobo, A., & Jairath, V. (2018) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2018.01.030 · Cited by 138
EHealth Technologies in Inflammatory Bowel Disease: A Systematic Review.
Jackson, B. D., Gray, K., Knowles, S. R., & De Cruz, P. (2016) · Journal of Crohn's and Colitis · doi:10.1093/ecco-jcc/jjw059 · Cited by 126
de Jong, M. J., Huibregtse, R., Masclee, A., Jonkers, D. M. A. E., & Pierik, M. J. (2017) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2017.10.019 · Cited by 132
“ability, and ability to detect change should be evaluated to finalize the instrument.(7,8)”
BackgroundChoi, K., Chun, J., Han, K., Park, S., Soh, H., Kim, J., Lee, J., Lee, J., Im, J., & Im, J. P. (2019) · Journal of Clinical Medicine · doi:10.3390/jcm8050654 · Cited by 136
“Anxiety and depression are the most common psychological disorders in patients with IBD and patient-reported outcome measures (PROMs) for anxiety and depression are considered to be critical to IBD patient care [11,12].”
BackgroundEarly intervention in Crohn’s disease: towards disease modification trials
Danese, S., Fiorino, G., & Peyrin‐Biroulet, L. (2017) · Gut · doi:10.1136/gutjnl-2017-314519 · Cited by 113
Rapid Response to Vedolizumab Therapy in Biologic-Naive Patients With Inflammatory Bowel Diseases
Feagan, B. G., Lasch, K. L., Lissoos, T. W., Cao, C., Wojtowicz, A. M., Khalid, J. M., & Colombel, J. (2018) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2018.05.026 · Cited by 108
Ulcerative Colitis Care Pathway
Dassopoulos, T., Cohen, R. D., Scherl, E., Schwartz, R. M., Kosinski, L. R., & Regueiro, M. D. (2015) · Gastroenterology · doi:10.1053/j.gastro.2015.05.036 · Cited by 106
Panés, J., Vermeire, S., Lindsay, J. O., Sands, B. E., Su, C. G., Friedman, G. S., Zhang, H., Yarlas, A. S., Bayliss, M. S., Maher, S. M., et al. (2017) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjx133 · Cited by 100
Iacucci, M., Daperno, M., Lazarev, M., Arsenascu, R., Tontini, G. E., Akinola, O., Gui, X. S., Villanacci, V., Goetz, M., Lowerison, M., et al. (2017) · Gastrointestinal Endoscopy · doi:10.1016/j.gie.2017.03.012 · Cited by 92
Parra, R. S., Chebli, J. M., Amarante, H. M., Flores, C., Parente, J. M., Ramos, O., Fernandes, M., Rocha, J. J., Feitosa, M. R., Feres, O., et al. (2019) · World Journal of Gastroenterology · doi:10.3748/wjg.v25.i38.5862 · Cited by 92
Mucosal Healing in Ulcerative Colitis – When Zero is Better
Carvalho, P. B., de Castro, F. D., Rosa, B. J. F., Moreira, M. J., & Cotter, J. B. (2015) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjv180 · Cited by 92
Sandborn, W. J., Vermeire, S., Tyrrell, H. E. J., Hassanali, A., Lacey, S., Tole, S., & Tatro, A. R. (2020) · Advances in Therapy · doi:10.1007/s12325-020-01366-2 · Cited by 92
Assessment of mucosal healing in inflammatory bowel disease: review
Dulai, P. S., Levesque, B. G., Feagan, B. G., D’Haens, G., & Sandborn, W. J. (2015) · Gastrointestinal Endoscopy · doi:10.1016/j.gie.2015.03.1974 · Cited by 89
Impact of Drug Therapy and Surgery on Quality of Life in Crohnʼs Disease
Wright, E. K., & Kamm, M. A. (2014) · Inflammatory Bowel Diseases · doi:10.1097/mib.0000000000000271 · Cited by 83
“Results from the validation of the disability index is expected in 2014.(1) Another disability questionnaire had a significant inverse correlation with QoL measures and a significant correlation with the Crohn’s Disease Activity Index.”
BackgroundMethodRodríguez‐Almagro, J., García‐Manzanares, Á., Lucendo, A. J., & Hernández‐Martínez, A. (2018) · Journal of Clinical Nursing · doi:10.1111/jocn.14624 · Cited by 80
van Deen, W. K., Jong, A. E. V. D. M., Parekh, N. K., Kane, E., Zand, A., DiNicola, C. A., Hall, L., Inserra, E. K., Choi, J. M., Ha, C. Y., et al. (2015) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2015.10.035 · Cited by 74
“14 Furthermore, the use of PROs as primary outcome measures for evaluating effectiveness of IBD interventions is progressively supported by the Food and Drug Administration.”
Atreya, R. N., Bloom, S. L., Scaldaferri, F., Gerardi, V., Admyre, C., Karlsson, A. E., Knittel, T. E., Kowalski, J. H., Lukáš, M., Löfberg, R., et al. (2016) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjw103 · Cited by 71
Patient preferences for inflammatory bowel disease treatment objectives
Casellas, F., Guise, C. H.-D., Robles, V., Navarro-Correal, E., & Borruel, N. (2016) · Digestive and Liver Disease · doi:10.1016/j.dld.2016.09.009 · Cited by 69
“009 linical trials, assessing symptoms such as abdominal pain, bowel ovements, or bloody stools, and the repercussion perceived by atients expressed as their quality of life [6,7].”
BackgroundMa, C., Panaccione, R., Fedorak, R. N., Parker, C. E., Nguyen, T. M., Khanna, R., Siegel, C. A., Peyrin‐Biroulet, L., D’Haens, G., Sandborn, W. J., et al. (2017) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2017.08.025 · Cited by 69
“As PROs become an increasingly central component of assessing treatment response, standardization of definitions and units of meaningful change will be needed.(3) Endoscopic endpoints are nearly universally reported as an outcome domain in contemporary RCTs.”
BackgroundHorst, S., Chao, A., Rosen, M., Nohl, A., Duley, C., Wagnon, J. H., Beaulieu, D. B., Taylor, W., Gaines, L., & Schwartz, D. A. (2015) · Digestive Diseases and Sciences · doi:10.1007/s10620-014-3375-0 · Cited by 75
“Measuring disease activity using the Harvey Bradshaw Index (HBI) has limitations including capturing functional gastrointestinal symptoms that can be associated with depression, and there is literature regarding lack of precision of this tool as a measure for active inflammatory bowel disease [31].”
MethodVarni, J. W., Bendo, C. B., Shulman, R. J., Self, M. M., Nurko, S., Franciosi, J. P., Saps, M., Saeed, S. A., Zacur, G. M., Dark, C. V., et al. (2015) · Journal of Pediatric Psychology · doi:10.1093/jpepsy/jsv005 · Cited by 65
“This paradigm shift toward PROs in clinical trials has provided the opportunity to include the perspective of pediatric patients using PROs as efficacy outcomes (Mohammad et al., 2014; Williet et al., 2014).”
BackgroundGray, M. A., Chao, C.-Y., Staudacher, H. M., Kolosky, N. A., Talley, N. J., & Holtmann, G. (2018) · PLOS ONE · doi:10.1371/journal.pone.0193542 · Cited by 63
Patient Reported Outcome Measures (PROMs) in Inflammatory Bowel Disease: New Data
Bojic, D., Bodger, K., & Travis, S. P. L. (2016) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjw187 · Cited by 60
Lo, B. Z. S., Prosberg, M. V., Gluud, L. L., Chan, W. P. W., Leong, R. W. L., van der List, E., van der Have, M., Sarter, H., Gower‐Rousseau, C., Peyrin‐Biroulet, L., et al. (2017) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.14373 · Cited by 60
“However, the United States food and drug administration (FDA) is beginning to shift from disease-related outcomes as its primary endpoints towards accepting patient-reported outcomes and clinician-reported outcomes.(3,4) Much effort has been put into investigating quality of life, while less work has been carried out…”
BackgroundMethodReal-World Evidence: Bridging Gaps in Evidence to Guide Payer Decisions
Roberts, M. H., & Ferguson, G. T. (2020) · PharmacoEconomics - Open · doi:10.1007/s41669-020-00221-y · Cited by 59
“Of note, while disease impact on work productivity costs (to include absenteeism and/or presenteeism) may be included in economic evaluations, and may be particularly meaningful for a condition such as irritable bowel disease that impacts a much younger population [68], to date productivity costs have been minimally i…”
BackgroundSystematic Review: Disease Activity Indices in Eosinophilic Esophagitis
Warners, M. J., Hindryckx, P., Levesque, B. G., Parker, C. E., Shackelton, L. M., Khanna, R., Sandborn, W. J., D’Haens, G., Feagan, B. G., Bredenoord, A. J., et al. (2017) · The American Journal of Gastroenterology · doi:10.1038/ajg.2017.363 · Cited by 58
Assche, G. V., Peyrin‐Biroulet, L., Sturm, A., Gisbert, J. P., Gaya, D. R., Bokemeyer, B., Mantzaris, G. J., Armuzzi, A., Sebastian, S., Lara, N., et al. (2016) · Digestive and Liver Disease · doi:10.1016/j.dld.2016.01.011 · Cited by 56
Development of the symptoms and impacts questionnaire for Crohn's disease and ulcerative colitis
Dulai, P. S., Jairath, V., Khanna, R., Ma, C., McCarrier, K. P., Martin, M. L., Parker, C. E., Morris, J. C., Feagan, B. G., & Sandborn, W. J. (2020) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.15726 · Cited by 56
Clinical Trials in Ulcerative Colitis: A Historical Perspective
Hindryckx, P., Baert, F., Hart, A. L., Magro, F., Armuzzi, A., & Peyrin‐Biroulet, L. (2015) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjv074 · Cited by 55
Artom, M., Czuber‐Dochan, W., Sturt, J. A., Murrells, T., & Norton, C. S. (2016) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.13870 · Cited by 58
“0 (20–83) Education Up to 16 2 (3) 19 (16) 0 (0) 21 (12) Up to 18 8 (13) 22 (19) 1 (17) 31 (17) Higher education 50 (83) 75 (65) 5 (83) 130 (71) Marital status Married/living with partner 35 (58) 76 (66) 3 (50) 115 (63) Widowed/divorced 2 (4) 7 (6) 0 (0) 9 (5) Single/single parent/other 23 (38) 33 (28) 3 (50) 58 (32)…”
Yarlas, A. S., Maher, S. M., Bayliss, M. S., Lovley, A., Cappelleri, J. C., Bushmakin, A. G., & DiBonaventura, M. (2020) · Journal of patient-centered research and reviews · doi:10.17294/2330-0698.1722 · Cited by 56
Marín‐Jiménez, I., Nos, P., Domènech, E., Riestra, S., Gisbert, J. P., Calvet, X., Cortés, X., Iglesias, E. L., Huguet, J. M., Taxonera, C., et al. (2016) · The American Journal of Gastroenterology · doi:10.1038/ajg.2015.403 · Cited by 54
Ma, C., Hussein, I. M., Al-Abbar, Y. J., Panaccione, R., Fedorak, R. N., Parker, C. E., Nguyen, T. M., Khanna, R., Siegel, C. A., Peyrin‐Biroulet, L., et al. (2018) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2018.02.051 · Cited by 53
“First, the formal construction and validation of a CD-specific PRO appropriate for use in randomized controlled trials (RCTs) could take years.(4) Second, current endoscopic indices, including the Crohn’s Disease Endoscopic Index of Severity (CDEIS)(5) and Simple Endoscopic Score for Crohn’s Disease (SES-CD)(6) are to…”
MethodHindryckx, P., Levesque, B. G., Holvoet, T., Durand, S., Tang, C., Parker, C. E., Khanna, R., Shackelton, L. M., D’Haens, G., Sandborn, W. J., et al. (2016) · Gut · doi:10.1136/gutjnl-2016-312762 · Cited by 53
Sarid, O., Slonim‐Nevo, V., Pereg, A., Friger, M. D., Sergienko, R., Schwartz, D., Greenberg, D., Shahar, I., Chernin, E., Vardi, H., et al. (2017) · PLoS ONE · doi:10.1371/journal.pone.0172779 · Cited by 50
Sexton, K. A., Walker, J. R., Targownik, L. E., Graff, L. A., Haviva, C., Beatie, B. E., Petty, S. K., Bernstein, M. T., Singh, H., Miller, N., et al. (2019) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izz038 · Cited by 51
Impact of Obesity on Inflammatory Bowel Disease
Khakoo, N. S., Ioannou, S., Khakoo, N. S., Vedantam, S., & Pearlman, M. Y. (2022) · Current Gastroenterology Reports · doi:10.1007/s11894-022-00840-x · Cited by 51
“In IBD, HRQoL is generally assessed by patient-reported outcomes obtained via the Short-Form 36 questionnaire or disease-specific scales, such as the Inflammatory Bowel Disease Questionnaire (IBDQ) [53].”
Zittan, E., Kabakchiev, B., Kelly, O., Milgrom, R., Nguyen, G. C., Croitoru, K., Steinhart, A. H., & Silverberg, M. S. (2016) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjw200 · Cited by 48
Review article: moving towards common therapeutic goals in Crohn's disease and rheumatoid arthritis
Allen, P. B., Olivera, P. A., Emery, P., Moulin, D., Jouzeau, J., Netter, P., Danese, S., Feagan, B. G., Sandborn, W. J., & Peyrin‐Biroulet, L. (2017) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.13995 · Cited by 48
“was not developed and validated according to FDA guidance for the development of patient-reported outcomes (PROs).(45)”
MethodVarni, J. W., Franciosi, J. P., Shulman, R. J., Saeed, S. A., Nurko, S., Neigut, D. A., Bendo, C. B., Patel, A., Self, M. M., Saps, M., et al. (2015) · Inflammatory Bowel Diseases · doi:10.1097/mib.0000000000000351 · Cited by 47
“Future research with these new scales will also benefit from direct comparisons with the IMPACT-III in the validation process, as was done in the validation of the PedsQL 4.0 Generic Core Scales and the PedsQL Multidimensional Fatigue Scale in pediatric patients with IBD.18 The PedsQL Gastrointestinal Symptoms Scales…”
InfluentialFecal calprotectin: current and future perspectives for inflammatory bowel disease treatment
Bertani, L., Mumolo, M. G., Tapete, G., Albano, E., Svizzero, G. B., Zanzi, F., Ceccarelli, L., Bellini, M., Marchi, S., & Costa, F. (2020) · European Journal of Gastroenterology & Hepatology · doi:10.1097/meg.0000000000001731 · Cited by 44
Patient-Reported Outcome Measures in Inflammatory Bowel Disease
El‐Matary, W. (2014) · Canadian Journal of Gastroenterology and Hepatology · doi:10.1155/2014/792386 · Cited by 42
Ma, C., Panaccione, R., Fedorak, R. N., Parker, C. E., Khanna, R., Levesque, B. G., Sandborn, W. J., Feagan, B. G., & Jairath, V. (2017) · BMJ Open · doi:10.1136/bmjopen-2017-016146 · Cited by 39
“Furthermore, recognising the need to accurately measure the patient experience with IBD, the US Food and Drug Administration (FDA) has advocated for measurement of patient-reported outcomes (PROs) in clinical trials.(20) The utilisation of PROs as a treatment end point in IBD trials poses unique challenges: importantl…”
BackgroundPreventing disability in inflammatory bowel disease
Allen, P. B., Gower‐Rousseau, C., Danese, S., & Peyrin‐Biroulet, L. (2017) · Therapeutic Advances in Gastroenterology · doi:10.1177/1756283x17732720 · Cited by 40
Feagan, B. G., Sandborn, W. J., Rutgeerts, P. J., Levesque, B. G., Khanna, R., Huang, B., Zhou, Q., Maa, J., Wallace, K. L., Lacerda, A. P., et al. (2018) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izx082 · Cited by 40
The state of the art on treatment of Crohn’s disease
Shi, H. Y., & Ng, S. C. (2018) · Journal of Gastroenterology · doi:10.1007/s00535-018-1479-6 · Cited by 39
Impact of Inflammatory Bowel Disease on Disability
Büsch, K., Sonnenberg, A., & Bansback, N. J. (2014) · Current Gastroenterology Reports · doi:10.1007/s11894-014-0414-0 · Cited by 39
Review article: treating‐to‐target for inflammatory bowel disease‐associated anaemia
Peyrin‐Biroulet, L., Lopez, A., Cummings, F. J. R., Dignass, A. U., Detlie, T. E., & Danese, S. (2018) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.14922 · Cited by 38
“PROs are increasingly replacing conventional endpoints such as the Crohn's disease Activity Index (CDAI) for the assessment of disease activity.(39) The primary outcome of most interventional studies PEYRIN‐BIROULET ET AL.”
Christiansen, L. K., Lo, B. Z. S., Bendtsen, F., Vind, I., Vester-Andersen, M. K., & Burisch, J. M. (2019) · United European Gastroenterology Journal · doi:10.1177/2050640619852532 · Cited by 37
Theede, K., Kiszka‐Kanowitz, M., Nordgaard-Lassen, I., & Nielsen, A. M. (2015) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjv081 · Cited by 36
“5] Smoking • Never 48% [53] • Former 36% [40] • Current 15% [17] Actual disease extent [%, n] • Inactive 29% [32] • Proctitis 20% [22] • Left-sided colitis 33% [36] • Extensive colitis 18% [20] Clinical disease activity [median, IQR] • SCCAI 3 [1-7] Mayo Endoscopic Score [%, n] • 0 29% [32] • 1 26% [29] • 2 31% [34] •…”
Development of a Microscopic Colitis Disease Activity Index: a prospective cohort study
Cotter, T. G., Binder, M., Loftus, E. V., Abboud, R., McNally, M. A., Smyrk, T. C., Tremaine, W. J., Sandborn, W. J., & Pardi, D. S. (2016) · Gut · doi:10.1136/gutjnl-2016-313051 · Cited by 36
Tumor Necrosis Factor’s Pathway in Crohn’s Disease: Potential for Intervention
Pagnini, C., & Cominelli, F. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms221910273 · Cited by 34
Review article: Measuring disease severity in inflammatory bowel disease – Beyond treat to target
Swaminathan, A., Day, A. S., Sparrow, M., Peyrin‐Biroulet, L., Siegel, C. A., & Gearry, R. B. (2024) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.18231 · Cited by 35
Tadbiri, S., Nachury, M., Bouhnik, Y., Serrero, M., Hébuterne, X., Roblin, X., Kirchgesner, J., Bouguen, G., Franchimont, D. P., Savoye, G., et al. (2020) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjaa244 · Cited by 35
Poor Sleep in Inflammatory Bowel Disease Is Reflective of Distinct Sleep Disorders
Salwen-Deremer, J. K., Smith, M. T., Haskell, H. G., Schreyer, C., & Siegel, C. A. (2022) · Digestive Diseases and Sciences · doi:10.1007/s10620-021-07176-y · Cited by 35
Rodríguez‐Almagro, J., Hernández‐Martínez, A., Lucendo, A. J., Casellas, F., Ruiz, M. C. S., & González, J. S. (2016) · Revista Española de Enfermedades Digestivas · doi:10.17235/reed.2016.4094/2015 · Cited by 34
“Changes in HRQL are now considered as an integral part in the assessment of therapeutic interventions for chronic or prolonged pathologies (23,24).”
BackgroundCarpio, D., López–Sanromán, A., Calvet, X., Romero, C., Cea‐Calvo, L., Juliá, B., & Argüelles‐Arias, F. (2016) · European Journal of Gastroenterology & Hepatology · doi:10.1097/meg.0000000000000658 · Cited by 34
Azzam, N., Aljebreen, A., Almuhareb, A., & Almadi, M. A. (2020) · Saudi Journal of Gastroenterology · doi:10.4103/sjg.sjg_175_20 · Cited by 34
“[6,7] Our current targets in the care for patients effected by IBD is to alleviate disease symptoms, decrease the incidence of flares, prevention of long term complications, as well as to 'improve patients HRQoL.[8,9] Numerous studies have focused on HRQoL assessment to measure and provide insight on the disability st…”
BackgroundEcharri, A., Vera, I., Ollero, V., Aràjol, C., Riestra, S., Robledo, P., Calvo, M., Gallego, F., CEBALLOS, D. S., Castro, B., et al. (2019) · Telemedicine Journal and e-Health · doi:10.1089/tmj.2018.0264 · Cited by 32
Patient-Reported Outcomes Measurement Information System in Children with Crohn's Disease
Arvanitis, M., DeWalt, D. A., Martin, C. F., Long, M. D., Chen, W., Jaeger, B., Sandler, R. S., & Kappelman, M. D. (2016) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.03.069 · Cited by 32
Treatment algorithms in Crohn's – Up, down or something else?
Antunès, O., Filippi, J., Hébuterne, X., & Peyrin‐Biroulet, L. (2014) · Best Practice & Research Clinical Gastroenterology · doi:10.1016/j.bpg.2014.05.001 · Cited by 31
“there is a growing interest for PROs in IBD [67], none of those currently used in IBD were developed according to FDA guidance.”
BackgroundMethodVarbobitis, I. C., Kokkotis, G., Gizis, M., Perlepe, N., Laoudi, E., Bletsa, M., Bekiari, D., Koutsounas, I. S., Kounadis, G., Xourafas, V., et al. (2020) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izaa201 · Cited by 31
Quality of care in inflammatory bowel diseases: What is the best way to better outcomes?
Strohl, M., Gönczi, L., Kurt, Z., Bessissow, T., & Lakatos, P. L. (2018) · World Journal of Gastroenterology · doi:10.3748/wjg.v24.i22.2363 · Cited by 31
Twenty‐five years of biologicals in IBD: What´s all the hype about?
Herrlinger, K. R., & Stange, E. F. (2021) · Journal of Internal Medicine · doi:10.1111/joim.13345 · Cited by 30
Hanžel, J., Sever, N., Ferkolj, I., Štabuc, B., Smrekar, N., Kurent, T., Koželj, M., Novak, G., Compernolle, G., Tops, S., et al. (2019) · United European Gastroenterology Journal · doi:10.1177/2050640619840211 · Cited by 29
Peyrin‐Biroulet, L., Ghosh, S. K., Lee, S. D., Lee, W., Griffith, J. M., Wallace, K. L., Berg, S., Liao, X., Panés, J., Loftus, E. V., et al. (2022) · Alimentary Pharmacology & Therapeutics · doi:10.1111/apt.17242 · Cited by 28
Luo, D., Zhou, M., Sun, L., Lin, Z., Bian, Q., Liu, M., & Ren, S.-R. (2021) · Frontiers in Psychiatry · doi:10.3389/fpsyt.2021.709295 · Cited by 28
“The quality of life (QOL) among patients with IBD is of growing significance since the patient-reported outcomes are recognized as primary endpoints by regulatory agencies (6).”
BackgroundPanés, J., Domènech, E., Peris, M. A., Nos, P., Riestra, S., Paramo, B. J. D., Cea‐Calvo, L., Romero, C., & Marín‐Jiménez, I. (2017) · Journal of Gastroenterology and Hepatology · doi:10.1111/jgh.13795 · Cited by 27
Salwen‐Deremer, J. K., Smith, M. T., Aschbrenner, K. A., Haskell, H. G., Speed, B. C., & Siegel, C. A. (2021) · BMJ Open Gastroenterology · doi:10.1136/bmjgast-2021-000805 · Cited by 26
“Examples from participants for whom the sleep/IBD relationship was a novel idea include: It was an unexpected avenue to take but one of the best things I have done in a long time, I know sleep in general is important, but I don’t think I would have thought of it initially as a treatment to help with IBD, and It met a…”
InfluentialLavelle, J., Storan, D., Eswara Murthy, V., De Dominicis, N., Mulcahy, H. E., & McHugh, L. (2022) · Journal of Clinical Medicine · doi:10.3390/jcm11102757 · Cited by 23
“Given such convincing evidence of a psychological component and impact in and of IBD, psychological support is now considered an integral component of modern IBD management [16,17].”
BackgroundNarula, N., Wong, E. C. L., Dulai, P. S., Marshall, J. K., Colombel, J., & Reinisch, W. (2020) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjaa189 · Cited by 25
Hardy, P., Fikri, J., Libbrecht, D., Louis, E. J., & Joris, J. L. (2022) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjac051 · Cited by 25
Derwa, Y. H., Williams, C. J. M., Sood, R., Mumtaz, S., Bholah, M. H., Selinger, C. P., Hamlin, P. J., Ford, A. C., & Gracie, D. J. (2018) · Therapeutic Advances in Gastroenterology · doi:10.1177/1756283x17744739 · Cited by 25
Atreya, R. N., Reinisch, W., Peyrin‐Biroulet, L., Scaldaferri, F., Admyre, C., Knittel, T. E., Kowalski, J. H., Neurath, M. F., & Hawkey, C. J. (2018) · Digestive and Liver Disease · doi:10.1016/j.dld.2018.06.010 · Cited by 25
Taleban, S., Stewart, K. O., Li, D. K., Singh, P., Pardi, D. S., Sturgeon, H. C., Yajnik, V., Xavier, R. J., Ananthakrishnan, A. N., & Khalili, H. (2016) · Digestive Diseases and Sciences · doi:10.1007/s10620-016-4180-8 · Cited by 24
“Recently, the FDA has mandated that clinical trial endpoints move away from using CAIs and toward endoscopic disease assessment and patient-reported outcome measures [38].”
Dulai, P. S., Singh, S., Casteele, N. V., Boland, B. S., & Sandborn, W. J. (2016) · Inflammatory Bowel Diseases · doi:10.1097/mib.0000000000000661 · Cited by 23
Morris, M. W., Stewart, S. A., Heisler, C., Sandborn, W. J., Loftus, E. V., Zello, G. A., Fowler, S. A., & Jones, J. (2018) · Inflammatory Bowel Diseases · doi:10.1093/ibd/izx018 · Cited by 23
Estevinho, M. M., Afonso, J., Rosa, I., Lago, P. M., Trindade, E., Correia, L., Dias, C. C., Magro, F., & GEDII [Portuguese IBD Group] (2018) · Journal of Crohn s and Colitis · doi:10.1093/ecco-jcc/jjy100 · Cited by 23
Toyonaga, T., Kobayashi, T., Nakano, M., Saito, E., Umeda, S., Okabayashi, S., Ozaki, R., & Hibi∥, T. (2017) · PLoS ONE · doi:10.1371/journal.pone.0185131 · Cited by 23
Evaluation of fatigue in inflammatory bowel disease - a useful tool in daily practice
Saraiva, S., Cortez-Pinto, J., Barosa, R., Castela, J., Moleiro, J., Rosa, I., da Siva, J. P., & Dias Pereira, A. (2019) · Scandinavian Journal of Gastroenterology · doi:10.1080/00365521.2019.1602669 · Cited by 22
“More recently, Williet et al. [9], developed a nationwide cohort study in order to test all the dimensions of PROs in the context of IBD.”
BackgroundInfluentialArtom, M., Czuber‐Dochan, W., Sturt, J. A., & Norton, C. S. (2017) · Trials · doi:10.1186/s13063-017-1926-3 · Cited by 24
“In line with the international expert consensus of the recent Therapeutic Targets in Inflammatory Bowel Disease (STRIDE) initiative [14] on treatment targets for IBD, it is therefore important to shift from management of IBD aimed solely at achieving endoscopic remission to also evaluating patient reported outcomes (P…”
ResultGhosh, S. K., Sandborn, W. J., Colombel, J., Feagan, B. G., Panaccione, R., Hanauer, S. B., Schreiber, S., Peyrin‐Biroulet, L., Vermeire, S., Eichner, S. F., et al. (2016) · Inflammatory Bowel Diseases · doi:10.1097/mib.0000000000000909 · Cited by 22
“Ideally, future trials will include PROs and endoscopic measures of disease activity as co-primary endpoints.(44) Evidence from UC populations has shown that there is still a subjective interpretation of endoscopic and cross-sectional images,(46,47) which may be aided by using a central reader.”
BackgroundLo, B. Z. S., Julsgaard, M., Vester-Andersen, M. K., Vind, I., & Burisch, J. M. (2018) · European Journal of Gastroenterology & Hepatology · doi:10.1097/meg.0000000000001199 · Cited by 22
Moon, J. R., Lee, C. K., Hong, S. N., Im, J. P., Ye, B. D., Cha, J. M., Jung, S.-A., Lee, K., Park, D. I., Jeen, Y. T., et al. (2019) · Gut and Liver · doi:10.5009/gnl19107 · Cited by 22
Bamias, G., Kokkotis, G., Gizis, M., Kapizioni, C., Κarmiris, K., Koureta, E., Kyriakos, N., Leonidakis, G., Makris, K., Markopoulos, P., et al. (2021) · Digestive Diseases and Sciences · doi:10.1007/s10620-021-06907-5 · Cited by 21
Metadata
- Authors
- Williet, N., Sandborn, W. J., & Peyrin–Biroulet, L.
- Affiliations
- Inserm (FR)
- University of California San Diego (US)
- Venue
- Clinical Gastroenterology and Hepatology
- Publisher
- Elsevier BV
- Published
- 2014-08
- Volume
- 12
- Issue
- 8
- Pages
- 1246-1256.e6
- ISSN
- 1542-3565 (ISSN-L)
- OpenAlex
- W2033081617
- Cited by
- 260
- References
- 150
- Citations by year
- 2026: 9 · 2025: 19 · 2024: 16 · 2023: 14 · 2022: 21 · 2021: 19 · 2020: 26 · 2019: 18 · 2018: 30 · 2017: 24 · 2016: 33 · 2015: 22 · 2014: 8
- FWCI
- 3.83
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Clinical Gastroenterology and Hepatology (publishedVersion)
- PubMed (publishedVersion)
- HAL (submittedVersion)
- Funders
- Optimer Pharmaceuticals
- Boehringer Ingelheim
- Shire
- Bristol-Myers Squibb
- Pfizer
- Orexigen Therapeutics
- UCB Pharma
- Novartis
- Roche
- Gilead Sciences
- Genentech
- Merck
- Topics
- Health Sciences › Medicine › Gastroenterology › Inflammatory Bowel Disease
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Keywords
- patient-reported outcomes, inflammatory bowel disease, Crohn's disease, primary endpoint, clinical trial endpoints, CDAI, quality of life, fatigue, anxiety and depression, labor productivity, disability, IBDQ, Short Form-36
- MeSH terms
- Humans, Surveys and Questionnaires, Inflammatory Bowel Diseases, Treatment Outcome, Drug Monitoring, Patient Satisfaction
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