Development of the celiac disease symptom diary version 2.1
CDSD
Celiac disease
Coeliac disease
Patient-reported outcome
Symptoms
Journal
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
ISSN: 1573-2649
Titre abrégé: Qual Life Res
Pays: Netherlands
ID NLM: 9210257
Informations de publication
Date de publication:
26 Oct 2024
26 Oct 2024
Historique:
accepted:
27
09
2024
medline:
27
10
2024
pubmed:
27
10
2024
entrez:
27
10
2024
Statut:
aheadofprint
Résumé
For patients with celiac disease (CeD), the only current management option is adherence to a strict gluten-free diet (GFD); however, many patients on a GFD continue to experience symptoms with a significant impact on quality of life. Potential new treatments for CeD are under development and a validated patient-reported outcome measure is required to evaluate their utility in clinical trials. The purpose of this article is to provide a history of the development of the Celiac Disease Symptom Diary (CDSD) 2.1 Qualitative and quantitative studies were conducted from 2010 to 2021, including concept elicitation and cognitive debriefing interviews with adult and adolescent participants with CeD (N = 93) diagnosed via biopsy and/or serology and input from eight interviews with CeD clinical experts. During these studies, different iterations of the CDSD were presented to the US Food and Drug Administration and the European Medicines Agency, and modifications were made in line with their feedback. These studies ultimately led to the development of CDSD 2.1 CDSD 2.1
Identifiants
pubmed: 39461929
doi: 10.1007/s11136-024-03799-6
pii: 10.1007/s11136-024-03799-6
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024. The Author(s).
Références
Green, P. H., Lebwohl, B., & Greywoode, R. (2015). Celiac disease. The Journal of Allergy and Clinical Immunology, 135, 1099–1106.
doi: 10.1016/j.jaci.2015.01.044
pubmed: 25956012
Singh, P., Arora, A., Strand, T. A., et al. (2018). Global prevalence of celiac disease: Systematic review and meta-analysis. Clinical Gastroenterology and Hepatology, 16, 823-836e2.
doi: 10.1016/j.cgh.2017.06.037
pubmed: 29551598
Makharia, G. K., Chauhan, A., Singh, P., & Ahuja, V. (2022). Review article: Epidemiology of coeliac disease. Alimentary Pharmacology & Therapeutics, 56, S3–S17.
doi: 10.1111/apt.16787
Rubio-Tapia, A., Hill, I. D., Kelly, C. P., Calderwood, A. H., & Murray, J. A. (2013). ACG clinical guidelines: diagnosis and management of celiac disease. American Journal Gastroenterology, 108, 656–676.
doi: 10.1038/ajg.2013.79
Leffler, D. A., Acaster, S., Gallop, K., et al. (2017). A novel patient-derived conceptual model of the impact of celiac disease in adults: Implications for patient-reported outcome and health-related quality-of-life instrument development. Value Health, 20, 637–643.
doi: 10.1016/j.jval.2016.12.016
pubmed: 28408006
Möller, S. P., Apputhurai, P., Tye-Din, J. A., & Knowles, S. R. (2021). Quality of life in coeliac disease: Relationship between psychosocial processes and quality of life in a sample of 1697 adults living with coeliac disease. Journal of Psychosomatic Research, 151, 110652.
doi: 10.1016/j.jpsychores.2021.110652
pubmed: 34739942
Wilson, I. B., & Cleary, P. D. (1995). Linking clinical variables with health-related quality of life. A conceptual model of patient outcomes. Jama, 273, 59–65.
doi: 10.1001/jama.1995.03520250075037
pubmed: 7996652
Clifford, S., Taylor, A. J., Gerber, M., et al. (2020). Concepts and instruments for patient-reported outcome assessment in celiac disease: Literature review and experts’ perspectives. Value Health, 23, 104–113.
doi: 10.1016/j.jval.2019.07.018
pubmed: 31952665
Kivelä, L., Caminero, A., Leffler, D. A., et al. (2021). Current and emerging therapies for coeliac disease. Nature Reviews Gastroenterology & Hepatology, 18, 181–195.
doi: 10.1038/s41575-020-00378-1
Food and Drug Adminisation draft guidance document. Patient-focused drug development: selecting, developing, or modifying fit-for-purpose clinical outcome assessments. June 2022. https://www.fda.gov/media/159500/download . Last accessed January 2023.
Food and Drug Administration guidance for industry. Patient-reported outcome measures: use in medical product development to support labelling claims. December 2009. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-support-labeling-claims . (Accessed January 2024).
European Medicines Agency scientific guideline. January 2006. https://www.ema.europa.eu/en/regulatory-guidance-use-health-related-quality-life-hrql-measures-evaluation-medicinal-products . Last accessed March 2023.
Hindryckx, P., Levesque, B. G., Holvoet, T., et al. (2018). Disease activity indices in coeliac disease: Systematic review and recommendations for clinical trials. Gut, 67, 61–69.
doi: 10.1136/gutjnl-2016-312762
pubmed: 27799282
Canestaro, W. J., Edwards, T. C., & Patrick, D. L. (2016). Systematic review: Patient-reported outcome measures in coeliac disease for regulatory submissions. Alimentary Pharmacology & Therapeutics, 44, 313–331.
doi: 10.1111/apt.13703
Ludvigsson, J. F., Ciacci, C., Green, P. H., et al. (2018). Outcome measures in coeliac disease trials: The Tampere recommendations. Gut, 67, 1410–1424.
doi: 10.1136/gutjnl-2017-314853
pubmed: 29440464
Gottlieb, K., Dawson, J., Hussain, F., & Murray, J. A. (2015). Development of drugs for celiac disease: Review of endpoints for Phase 2 and 3 trials. Gastroenterology Report, 3, 91–102.
doi: 10.1093/gastro/gov006
pubmed: 25725041
pmcid: 4423465
Leffler, D., Kupfer, S. S., Lebwohl, B., et al. (2016). Development of celiac disease therapeutics: Report of the third gastroenterology regulatory endpoints and advancement of therapeutics workshop. Gastroenterology, 151, 407–411.
doi: 10.1053/j.gastro.2016.07.025
pubmed: 27456385
Food and Drug Administration. Level 1 guidance, draft April 2022. Celiac disease: developing drugs for adjunctive treatment to a gluten-free diet. April 2022. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/celiac-disease-developing-drugs-adjunctive-treatment-gluten-free-diet . Last accessed January 2023.
Patrick, D. L., Burke, L. B., Gwaltney, C. J., et al. (2011). Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 1–eliciting concepts for a new PRO instrument. Value Health, 14, 967–977.
doi: 10.1016/j.jval.2011.06.014
pubmed: 22152165
Patrick, D. L., Burke, L. B., Gwaltney, C. J., et al. (2011). Content validity–establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 2–assessing respondent understanding. Value Health, 14, 978–988.
doi: 10.1016/j.jval.2011.06.013
pubmed: 22152166
Lewis, S. J., & Heaton, K. W. (1997). Stool form scale as a useful guide to intestinal transit time. Scandinavian Journal of Gastroenterology, 32, 920–924.
doi: 10.3109/00365529709011203
pubmed: 9299672
Food and Drug Administration guidance document. Patient-focused drug development: methods to identify what is important to patients. February 2022. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-methods-identify-what-important-patients . Accessed January 2024.
Zbrozek, A., Hebert, J., Gogates, G., et al. (2013). Validation of electronic systems to collect patient-reported outcome (PRO) data-recommendations for clinical trial teams: Report of the ISPOR ePRO systems validation good research practices task force. Value Health, 16, 480–489.
doi: 10.1016/j.jval.2013.04.002
pubmed: 23796281
Mardini, H. E., Westgate, P., & Grigorian, A. Y. (2015). Racial differences in the prevalence of celiac disease in the US population: National health and nutrition examination survey (NHANES) 2009–2012. Digestive Diseases and Sciences, 60, 1738–1742.
doi: 10.1007/s10620-014-3514-7
pubmed: 25577269
Meckley, L. M., Marden, J. R., Sethi, S., et al. (2023). Tu1377 the virtual celiac symptoms study: symptom and gluten-free diet perceptions at baseline. Gastroenterology. https://doi.org/10.1016/S0016-5085(23)03410-8
doi: 10.1016/S0016-5085(23)03410-8