Perspectives in clinical gastroenterology and hepatologyPatient-Reported Outcomes as Primary End Points in Clinical Trials of Inflammatory Bowel Disease
Section snippets
Methods
See the Supplementary Materials and Methods section for more detail.3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23
Food and Drug Administration Guidance for Developing Patient-Reported Outcomes
Recently, the FDA proposed guidance for the development of PROs with the ultimate goal of supporting labeling claims.24 An overview of this guidance is shown in Figure 1. Importantly, PROs should not be confused with patient-based outcomes, which are developed by and/or collected by practitioners; PROs should be obtained from validated self-administered questionnaire(s) using items that are generated solely by patients.24 A PRO is any report that comes directly from a patient about a health
Experience From Other Chronic Conditions
See the Supplementary Materials and Methods section.25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 48, 49
Quality of Life in Inflammatory Bowel Disease
The definition and epidemiology4, 50, 51, 52, 53, 54, 55 and available tools to assess quality of life in IBD (Table 1) can be found in the Supplementary Materials and Methods section.3, 4, 5, 13, 14, 15, 56, 57, 58, 59, 60, 61, 62, 63, 64, 65, 66, 67, 68, 69, 70, 71, 72, 73, 74, 75, 76, 77, 78, 79, 80, 81, 82
Work Productivity in Inflammatory Bowel Disease
The definition and epidemiology106, 107 and available tools to assess work productivity in IBD (Table 2) can be found in the Supplementary Materials and Methods section.7, 8, 70, 71, 92, 108, 109, 110, 111, 112
Disability in Inflammatory Bowel Disease
The definition and epidemiology12, 107, 116, 117, 118, 119 and available tools to assess disability in IBD (Table 3) can be found in the Supplementary Materials and Methods section.12, 120, 121, 122, 123, 124, 125
Fatigue in Inflammatory Bowel Disease
The definition and epidemiology126, 127, 128 and available tools to assess fatigue in IBD (Table 4) can be found in the Supplementary Materials and Methods section.6, 20, 21, 22, 127, 129, 130, 131, 132, 133
Depression and Anxiety in Inflammatory Bowel Disease
The definition and epidemiology135, 136, 137, 138, 139, 140 and available tools to assess depression and anxiety in IBD (Table 5) can be found in the Supplementary Materials and Methods section.9, 10, 11, 23, 136
Relationships Between Patient-Reported Outcomes and Disease Activity in Inflammatory Bowel Disease
See the Supplementary Materials and Methods section for more detail.67, 71, 74, 76, 77, 78, 83, 85, 86, 88, 89, 91, 94, 99, 103, 142, 143
Conclusions and Perspectives
There is a growing interest for PROs in IBD. In the future, physicians managing patients with IBD may evaluate a variety of PROs that assess fatigue, disability, quality of life, work productivity, anxiety, and depression, with the final aim of improving patients’ lives. However, none of the existing IBD PROs were developed according to FDA guidance, and thus cannot lead to PRO labeling. Available questionnaires such as the IBDQ assessing quality of life in IBD and the IBD Disability Index do
References (143)
- et al.
A new measure of health status for clinical trials in inflammatory bowel disease
Gastroenterology
(1989) - et al.
Measuring fatigue and other anemia-related symptoms with the Functional Assessment of Cancer Therapy (FACT) measurement system
J Pain Symptom Manage
(1997) - et al.
Validity, reliability, and responsiveness of the work productivity and activity impairment questionnaire in Crohn's disease
Clin Ther
(2008) - et al.
Prospective assessment of Cleveland Global Quality of Life (CGQL) as a novel marker of quality of life and disease activity in Crohn's disease
Am J Gastroenterol
(2003) - et al.
The Multidimensional Fatigue Inventory (MFI) psychometric qualities of an instrument to assess fatigue
J Psychosom Res
(1995) - et al.
Disability outcome measures in multiple sclerosis clinical trials: current status and future prospects
Lancet Neurol
(2012) - et al.
Randomised trial of oral and intravenous methylprednisolone in acute relapses of multiple sclerosis
Lancet
(1997) - et al.
Progressive burden of myelofibrosis in untreated patients: assessment of patient-reported outcomes in patients randomized to placebo in the COMFORT-I study
Leuk Res
(2013) - et al.
Quality of life after surgery for Crohn's disease: a psychosocial survey
Gastroenterology
(1980) - et al.
Health-related quality of life in Crohn's disease: a prospective longitudinal study in 231 patients
Am J Gastroenterol
(2001)
Determinants of health-related quality of life in Crohn's disease: a systematic review and meta-analysis
J Crohns Colitis
Instruments for quality of life assessment in patients with inflammatory bowel disease
Dig Liver Dis
Quality of life: a valid and reliable measure of therapeutic efficacy in the treatment of inflammatory bowel disease. Canadian Crohn's Relapse Prevention Trial Study Group
Gastroenterology
The UK IBDQ-a British version of the inflammatory bowel disease questionnaire. Development and validation
J Clin Epidemiol
Validation of the Italian translation of the Inflammatory Bowel Disease Questionnaire
Dig Liver Dis
Is patient reported outcome (PRO) affected by different follow-up regimens in inflammatory bowel disease (IBD)? A one year prospective, longitudinal comparison of nurse-led versus conventional follow-up
J Crohns Colitis
The effects of infliximab maintenance therapy on health-related quality of life
Am J Gastroenterol
Long-term treatment of rectovaginal fistulas in Crohn's disease: response to infliximab in the ACCENT II Study
Clin Gastroenterol Hepatol
Adalimumab for the induction and maintenance of clinical remission in Japanese patients with Crohn's disease
J Crohns Colitis
An engineered human antibody to TNF (CDP571) for active Crohn's disease: a randomized double-blind placebo-controlled trial
Gastroenterology
A randomized, placebo-controlled trial of certolizumab pegol (CDP870) for treatment of Crohn's disease
Gastroenterology
Etanercept for active Crohn's disease: a randomized, double-blind, placebo-controlled trial
Gastroenterology
Efficacy and safety of retreatment with anti-tumor necrosis factor antibody (infliximab) to maintain remission in Crohn's disease
Gastroenterology
FDA and EMA end points: which outcome end points should we use in clinical trials in patients with irritable bowel syndrome?
Neurogastroenterol Motil
Measuring irritable bowel syndrome patient-reported outcomes with an abdominal pain numeric rating scale
Aliment Pharmacol Ther
Quality of life in inflammatory bowel disease
J Clin Gastroenterol
The Short Inflammatory Bowel Disease Questionnaire: a quality of life instrument for community physicians managing inflammatory bowel disease. CCRPT Investigators. Canadian Crohn's Relapse Prevention Trial
Am J Gastroenterol
The validity and reproducibility of a work productivity and activity impairment instrument
Pharmacoeconomics
The Depression Status Inventory: an adjunct to the self-rating depression scale
J Clin Psychol
The hospital anxiety and depression scale
Acta Psychiatr Scand
An inventory for measuring depression
Arch Gen Psychiatry
Development of the first disability index for inflammatory bowel disease based on the international classification of functioning, disability and health
Gut
The rating form of IBD patient concerns: a new measure of health status
Psychosom Med
The MOS 36-item Short-form Health Survey (SF-36). I. Conceptual framework and item selection
Med Care
Evaluation of index and profile measures of health status in a randomized controlled trial. Comparison of the Medical Outcomes Study 36-Item Short Form Health Survey, EuroQol, and disease specific measures
Med Care
Comparison of conditional quality of life terminology and visual analogue scale measurements
Qual Life Res
The IMPACT questionnaire: a valid measure of health-related quality of life in pediatric inflammatory bowel disease
J Pediatr Gastroenterol Nutr
The PedsQL: measurement model for the pediatric quality of life inventory
Med Care
Piper fatigue scale available for clinical testing
Oncol Nurs Forum
Measuring the functional impact of fatigue: initial validation of the fatigue impact scale
Clin Infect Dis
STAI manual for the State–Trait Anxiety Inventory
Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance
Health Qual Life Outcomes
Fatigue, normal and pathological, with special consideration of myasthenia gravis and multiple sclerosis
South Med Surg
Mortality and disability in multiple sclerosis; a statistical estimate of prognosis
JAMA
[Medicosocial consequences of inflammatory rheumatism]
Soins
The prevalence and meaning of fatigue in rheumatic disease
J Rheumatol
Factors associated with absenteeism, presenteeism and activity impairment in patients in the first years of RA
Rheumatology (Oxford)
Reporting of patient-reported outcomes in recent trials in rheumatoid arthritis: a systematic literature review
Ann Rheum Dis
Measurement of patient outcome in arthritis
Arthritis Rheum
Induction of response with etanercept-methotrexate therapy in patients with moderately active rheumatoid arthritis in Central and Eastern Europe in the PRESERVE study
Clin Rheumatol
Cited by (196)
Patient-reported Outcome Measures in Ileoanal Pouch Surgery: a Systematic Review
2024, Journal of Crohn's and ColitisBurden of Anxiety, Depression and Perceived Stress in Patients with Inflammatory Bowel Disease: A Cohort Study from North India
2024, Digestive Diseases and SciencesBaseline Patient-reported Symptoms Less Predictive Than MM-SES-CD for Endoscopic Remission in Crohn's Disease
2023, Journal of Clinical Gastroenterology
Conflicts of interest These authors disclose the following: Laurent Peyrin–Biroulet has received consulting and/or lecture fees from Merck, Abbott, Janssen, Genentech, Mitsubishi, Ferring, Norgine, Tillots, Vifor, Shire, Therakos, Pharmacosmos, Pilège, BMS, UCB-pharma, Hospira, and Takeda. William Sandborn has received consulting fees from ActoGeniX NV, AGI Therapeutics, Inc, Alba Therapeutics Corporation, Albireo, Alfa Wasserman, Amgen, AM-Pharma BV, Anaphore, Astellas Pharma, Athersys, Inc, Atlantic Healthcare Limited, Axcan Pharma (now Aptalis), BioBalance Corporation, Boehringer-Ingelheim, Inc, Bristol Meyers Squibb, Celgene, Celek Pharmaceuticals, Cellerix SL, Cerimon Pharmaceuticals, ChemoCentryx, CoMentis, Cosmo Technologies, Coronado Biosciences, Cytokine Pharmasciences, Eagle Pharmaceuticals, Eisai Medical Research, Inc, Elan Pharmaceuticals, EnGene, Inc, Eli Lilly, Enteromedics, Exagen Diagnostics, Inc, Ferring Pharmaceuticals, Flexion Therapeutics, Inc, Funxional Therapeutics Limited, Genzyme Corporation, Genentech (now Roche), Gilead Sciences, Given Imaging, Glaxo Smith Kline, Human Genome Sciences, Ironwood Pharmaceuticals (previously Microbia, Inc), Janssen (previously Centocor), KaloBios Pharmaceuticals, Inc, Lexicon Pharmaceuticals, Lycera Corporation, Meda Pharmaceuticals (previously Alaven Pharmaceuticals), Merck Research Laboratories, MerckSerono, Millennium Pharmaceuticals (subsequently merged with Takeda), Nisshin Kyorin Pharmaceuticals Co, Ltd, Novo Nordisk A/S, NPS Pharmaceuticals, Optimer Pharmaceuticals, Orexigen Therapeutics, Inc, PDL Biopharma, Pfizer, Procter and Gamble, Prometheus Laboratories, ProtAb Limited, Purgenesis Technologies, Inc, Receptos, Relypsa, Inc, Salient Pharmaceuticals, Salix Pharmaceuticals, Inc, Santarus, Schering Plough Corporation (acquired by Merck), Shire Pharmaceuticals, Sigmoid Pharma Limited, Sirtris Pharmaceuticals, Inc (a GSK company), S.L.A. Pharma (UK) Limited, Targacept, Teva Pharmaceuticals, Therakos, Tillotts Pharma AG (acquired by Zeria Pharmaceutical Co, Ltd), TxCell SA, UCB Pharma, Viamet Pharmaceuticals, Vascular Biogenics Limited, Warner Chilcott UK Limited, and Wyeth (now Pfizer); has received lecture fees from Bristol Meyers Squibb and Janssen (previously Centocor); and has received research support from Bristol Meyers Squibb, Genentech, Glaxo Smith Kline, Janssen (previously Centocor), Millennium Pharmaceuticals (now Takeda), Novartis, Pfizer, Procter and Gamble Pharmaceuticals, Shire Pharmaceuticals, and UCB Pharma. The remaining author discloses no conflicts.