Skip to main content
Top

Open Access 26-10-2024 | Celiac Disease

Development of the celiac disease symptom diary version 2.1© (CDSD 2.1©) patient-reported outcome measure

Authors: Kellee Howard, Daniel Adelman, Sonal Ghura, Sarah Acaster, Sarah Clifford, Ciaran P. Kelly, Susan A. Martin, Lisa M. Meckley, Daniel A. Leffler

Published in: Quality of Life Research

Login to get access

Abstract

Purpose

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© for use in clinical trials.

Methods

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.

Results

These studies ultimately led to the development of CDSD 2.1©, a daily diary which focuses on key symptoms of CeD (abdominal pain, bloating, diarrhea, nausea and tiredness). This patient-reported outcome measure was readily understood by adult and adolescent participants with CeD and content validity was demonstrated in both populations.

Conclusion

CDSD 2.1© is a content-valid patient-reported outcome measure developed in accordance with best practices and regulatory guidance. A thorough exploration of the psychometric properties of CDSD 2.1© for both adult and adolescent participants with CeD is ongoing to support utilization in clinical trials.
Appendix
Available only for authorised users
Literature
1.
go back to reference Green, P. H., Lebwohl, B., & Greywoode, R. (2015). Celiac disease. The Journal of Allergy and Clinical Immunology, 135, 1099–1106.CrossRefPubMed Green, P. H., Lebwohl, B., & Greywoode, R. (2015). Celiac disease. The Journal of Allergy and Clinical Immunology, 135, 1099–1106.CrossRefPubMed
2.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
3.
go back to reference Makharia, G. K., Chauhan, A., Singh, P., & Ahuja, V. (2022). Review article: Epidemiology of coeliac disease. Alimentary Pharmacology & Therapeutics, 56, S3–S17.CrossRef Makharia, G. K., Chauhan, A., Singh, P., & Ahuja, V. (2022). Review article: Epidemiology of coeliac disease. Alimentary Pharmacology & Therapeutics, 56, S3–S17.CrossRef
4.
go back to reference 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.CrossRef 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.CrossRef
5.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
6.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
7.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
8.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
9.
go back to reference Kivelä, L., Caminero, A., Leffler, D. A., et al. (2021). Current and emerging therapies for coeliac disease. Nature Reviews Gastroenterology & Hepatology, 18, 181–195.CrossRef Kivelä, L., Caminero, A., Leffler, D. A., et al. (2021). Current and emerging therapies for coeliac disease. Nature Reviews Gastroenterology & Hepatology, 18, 181–195.CrossRef
13.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
14.
go back to reference 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.CrossRef 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.CrossRef
15.
go back to reference Ludvigsson, J. F., Ciacci, C., Green, P. H., et al. (2018). Outcome measures in coeliac disease trials: The Tampere recommendations. Gut, 67, 1410–1424.CrossRefPubMed Ludvigsson, J. F., Ciacci, C., Green, P. H., et al. (2018). Outcome measures in coeliac disease trials: The Tampere recommendations. Gut, 67, 1410–1424.CrossRefPubMed
16.
go back to reference 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.CrossRefPubMedPubMedCentral 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.CrossRefPubMedPubMedCentral
17.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
19.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
20.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
21.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
23.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
24.
go back to reference 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.CrossRefPubMed 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.CrossRefPubMed
Metadata
Title
Development of the celiac disease symptom diary version 2.1© (CDSD 2.1©) patient-reported outcome measure
Authors
Kellee Howard
Daniel Adelman
Sonal Ghura
Sarah Acaster
Sarah Clifford
Ciaran P. Kelly
Susan A. Martin
Lisa M. Meckley
Daniel A. Leffler
Publication date
26-10-2024
Publisher
Springer International Publishing
Published in
Quality of Life Research
Print ISSN: 0962-9343
Electronic ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-024-03799-6