Introduction
For patients living with inflammatory bowel diseases (IBD), the ultimate treatment goal is to live a “normal life.” However, no validated patient-reported outcome measurement (PROM) exists yet to measure this. Moreover, most PROMs were developed by western healthcare professionals, lacking a global, patient-centred approach.
Aims & Methods
Through a world-wide patient-centred approach, we aimed to develop and validate a PROM that identifies and measures normality of life for patients with IBD, the “IBD LIFE.”
IBD patients without an ostomy, pouch or significant other comorbidities could participate in the project. The development phase included 6-10 patients with Crohn’s disease (CD) and 6-10 patients with ulcerative colitis (UC) from each of the participating countries (Argentina, Australia, India, the Netherlands, South-Korea and the USA).
The development phase consisted of (i) semi-structured qualitative interviews about living a normal life with IBD, (ii) an online Delphi round to rate item importance, (iii) a focus group to clarify wording, categorisation and scales, (iv) pilot-testing and (v) cross-cultural translation.
For the validation phase, 200 adult IBD patients are recruited per language (100 CD, 100 UC). At baseline (BL), we assessed clinical disease activity (using the Harvey Bradshaw Index (HBI) or Simple Clinical Colitis Activity Index (SCCAI)) and asked patients to complete a PROM set including the IBD LIFE, a general quality of life (QoL) PROM and an IBD-specific QoL PROM (Short-From-36 (SF-36) and IBD Questionnaire (IBDQ)). In addition, 30 consecutive patients per language were asked to complete a second PROM set 2 weeks after baseline (W2), comprising of the IBD LIFE, IBDQ and question 2 from the IBD-control.
Internal consistency was assessed using Cronbach’s α. Test-retest reliability was assessed through the intraclass correlation coefficient (ICC) by comparing IBD LIFE scores at BL and W2 in patients with stable QoL (defined as <10% difference in the IBDQ score and the IBD control question 2 answered “no change”). Construct validity was assessed at BL through Spearman’s correlation coefficients between the IBD LIFE, the comparator QoL PROMs and disease activity indices.
Results
The IBD LIFE consists of 23 questions divided into 5 categories: physical items, activities, social items, psychological items and circumstances.
For the validation phase, we report on the first 364 included patients (200 Dutch, 100 Spanish and 64 English speaking patients, 187 UC and 177 CD, median disease duration 10 years [5-18], see table 1). In CD, median HBI was 2 [1-5] and in UC, median SCCAI was 1 [0-2]. PROM response percentage was 85%. 38 patients completed W2 visit, of whom 71% had stable QoL.
The IBD LIFE showed excellent internal consistency (Cronbach’s α = 0.95). Test-retest reliability was good (ICC = 0.94 [95% CI 0.88-0.98]). The IBD LIFE exhibited good construct validity, depicted by a strong correlation with IBD-specific QoL (IBDQ r = 0.90), and general QoL (SF-36 r = 0.84) and a moderate correlation with clinical disease activity (HBI r = 0.58; SCCAI r = 0.54).
Disease characteristics
Diagnosis (CD), n (%)
Median disease duration (year), median [IQR]
Naive to advanced therapies, n (%)
Crohn’s disease
Underwent CD-related surgery, n (%)
HBI, median [IQR]
Ulcerative colitis
Underwent UC-related surgery, n (%)
SCCAI, median [IQR]
|
177 (49%)
10 [5-18]
215 (59%)
186 (51%)
2 [1-5]
51 (14%)
1 [0-2]
|
| Table 1. Baseline characteristics | n = 364 |
Demographics
Gender (male), n (%)
Age (year), median [IQR]
Employment status (employed), n (%)
|
160 (41%)
41 [30-54]
328 (90%)
|
Conclusion
The IBD LIFE is a novel, valid and reliable PROM that has been developed through a rigorous process with maximal patient involvement. Further validation in English, Spanish, Hindi, French and Korean is ongoing, as well as responsiveness analyses and cross-cultural validation.
Disclosure
This project was supported by an unrestricted grant from Eli Lilly and Company.
Joep van Oostrom has received speaker’s fees from Tillotts
Neda Karimi has received grant support from Janssen and conference attendance support from Ferring, and Pfizer.
Juan Lasa has received consulting fees and honoraria from Abbvie; honoraria from Janssen; honoraria from Takeda Pharmaceuticals; consulting fees from Glaxo Smith Kline.
Pablo A. Olivera has received consulting fees from AbbVie, Takeda, and Janssen; honoraria from Takeda and Janssen; and financial support for attending meetings, travel, or both from AbbVie, Takeda, Janssen, and Ferring.
Byong Duk Ye has served on advisory boards for AbbVie Korea, Celltrion, Daewoong Pharma, Ferring Korea, Janssen Korea, Pfizer Korea, and Takeda Korea; has received research grants from Celltrion and Pfizer Korea; has received consulting fees from Chong Kun Dang Pharm., CJ Red BIO, Cornerstones Health, Daewoong Pharma, IQVIA, Kangstem Biotech, Korea United Pharm. Inc., Medtronic Korea, NanoEntek, and Takeda; and has received speaking fees from AbbVie Korea, Celltrion, Ferring Korea, IQVIA, Janssen Korea, Pfizer Korea, Takeda, and Takeda Korea.
Rupa Banerjee has served on advisory boards of Jansen, Takeda, Pfeizer India, Cipla, Menarini, Sun pharma, Abbott , Ferring pharma, MSN labs , Dr. Reddys , Zydus and Cadila She has received research support from DRL, Microlabs, Takeda, J &J at the IBDENC. She has grants from The Leona M and Harry B Helmsley Charitable Trust.
Susan J Connor has received honoraria for Advisory Board participation, speaker fees, educational support and/or research support from: Abbvie, Amgen, BMS, Celltrion, Chiesi, DrFalk, Eli-Lilly, Ferring, Fresenius Kabi, GSK, Janssen, MSD, Novartis, Organon, Pfizer, Sandoz, Takeda, Agency for Clinical Innovation, Gastroenterological Society of Australia, Medical Research Future Fund, South Western Sydney Local Health District, Sydney Partnership for Health, Research and Enterprise (SPHERE) and The Leona M and Harry B Helmsley Charitable Trust..
Corey A Siegel has received grant support from Abbvie, Janssen, Pfizer and Takeda; has served as a consultant for Abbvie, BMS, Lilly, Janssen, Napo Pharmaceuticals, Pfizer, ProciseDx, Prometheus Biosciences, Takeda, and Trellus Health; has served as a speaker for CME activities for Abbvie, Janssen, Pfizer and Takeda; has received licensing fees from Takeda for intellectual property created by MiTest Health, LLC; and is a co-founder of MiTest Health, LLC.
Laurent Peyrin-Biroulet reports personal fees from Galapagos, AbbVie, Janssen Pharmaceuticals, Genentech, Alimentiv, Ferring Pharmaceuticals, Tillots Pharma, Celltrion, Takeda, Pfizer, Index Pharmaceuticals, Sandoz, Celgene, Biogen, Samsung Bioepis, Inotrem, Allergan, MSD, Roche, Arena Pharmaceuticals, Gilead Sciences, Amgen, BMS, Vifor Pharma, Norgine, Mylan, Eli Lilly, Fresenius Kabi, OSE Immunotherapeutics, Enthera, Theravance, Pandion Therapeutics, Gossamer Bio, Viatris, and Thermo Fisher; grants from AbbVie, MSD, Takeda, and Fresenius Kabi; and stock options from Clinical Trials Mobile Application.
Krisztina Gecse has received grants from Pfizer Inc, Celltrion and Galapagos; consultancy fees from AbbVie, Arena Pharmaceuticals, Galapagos, Gilead, Immunic Therapeutics, Janssen Pharmaceuticals, Novartis, Pfizer Inc., Samsung Bioepis and Takeda and speaker’s honoraria from Abbvie, Celltrion, Ferring, Janssen Pharmaceuticals, Novartis, Pfizer Inc, Samsung Bioepis, Takeda and Tillotts.
Geert D’Haens has served as a consultant and/or received speaker’s fees from AbbVie, Alimentiv, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Celltrion Healthcare, Cytoki Pharma, Eli Lilly, Ferring, Galapagos, Glaxo SmithKline, Gossamer Bio, Immunic, InDex Pharmaceuticals, Janssen, Johnson & Johnson, Kaleido, Pfizer, Progenity, Prometheus Biosciences, Prometheus Laboratories, Protagonist Therapeutics, Takeda, Tillotts, Ventyx Biosciences.
Suzanne Anjie, Marlou Braad, Jamie Horrigan, Bhargavi Adi, Girish Ganesh, Suk-Kyun Yang, Christian Broër, Anja de Kruif and Rupa Banerjee report no conflicts of interest
