Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2017;1(1):3.
doi: 10.1186/s41687-017-0006-5. Epub 2017 Sep 12.

Psychometric validation of the Dysphagia Symptom Questionnaire in patients with eosinophilic esophagitis treated with budesonide oral suspension

Stacie Hudgens  1 , Christopher Evans  2 , Elaine Phillips  3 , Malcolm Hill  3
Affiliations

Psychometric validation of the Dysphagia Symptom Questionnaire in patients with eosinophilic esophagitis treated with budesonide oral suspension

Stacie Hudgens et al. J Patient Rep Outcomes. 2017.

Abstract

Background: Eosinophilic esophagitis (EoE) is characterized by high levels of eosinophils in the esophageal mucosa. Patients with the disease present with a range of symptoms, including dysphagia (difficulty swallowing). The aim of this analysis was to assess the psychometric properties of the Dysphagia Symptom Questionnaire (DSQ), a patient-reported outcome (PRO) measure of dysphagia associated with EoE. Psychometric properties of the DSQ were assessed using data collected from a 12-week, phase 2, multicenter, randomized, double-blind, placebo-controlled trial of budesonide oral suspension in adolescents and adults (11-40 years old) with EoE.

Results: The study population comprised 93 patients with EoE; 94.6% of whom were white, 68.8% were male and the mean age (standard deviation) was 21.6 (7.7) years. Patients had been diagnosed with EoE for a mean of 37.6 months before study initiation. The DSQ was feasible to implement with few item-level data missing at baseline. Item discrimination was high, with floor and ceiling effects below the predefined threshold (≤9%). Higher DSQ scores corresponded with presence and increased severity of dysphagia, indicative of strong item discrimination among patients at baseline (threshold >50%). The DSQ was able to detect changes in symptoms over time and produced similar outcomes to those from physician- and other patient-rated measures, supportive of construct validity. The DSQ had strong test-retest reliability (intraclass correlation coefficient, r = 0.82); and was also responsive to disease-level changes, with higher DSQ scores corresponding to increased esophageal eosinophilic burden. Lastly, the percentage changes in the minimal clinically important difference and clinically important difference in DSQ score were estimated at -27.4% and -55.4%, respectively.

Conclusions: These analyses support the DSQ as a valid and reliable measure of dysphagia in patients with EoE. Changes in DSQ scores suggest a level of agreement between clinician, patient and histologic response. The DSQ should therefore be considered a viable PRO measure of dysphagia for use in future therapeutic studies of EoE.

Keywords: Budesonide oral suspension; Dysphagia; Eosinophilic esophagitis; Minimal clinically important difference; Patient-reported outcome; Psychometric properties; Responsiveness; Validity.

PubMed Disclaimer

Conflict of interest statement

SH is a Managing Partner and Strategic Lead at Clinical Outcome Solutions. CE is the Chief Executive Officer and Senior Research Leader at Endpoint Outcomes and Adjunct faculty at Tufts University School of Medicine, Boston, Massachusetts, USA. EP is the former President and Chief Executive Officer of Meritage Pharma, Inc. MH was an employee of Meritage Pharma, Inc. at the time of this study.The analysis presented here used data collected from a 12-week, phase 2 trial of budesonide oral suspension in patients with eosinophilic esophagitis, registered at ClinicalTrials.gov (identifier: NCT01642212). The study was approved by the Institutional Review Board at each center and was carried out in accordance with the International Conference on Harmonisation of Good Clinical Practice guidelines and the Declaration of Helsinki.Not applicable.SH was funded by Endpoint Outcomes to conduct the analysis. CE received consulting fees from Meritage Pharma, Inc., now part of the Shire group of companies, to complete this work. EP and MH were employees of Meritage Pharma Inc. at the time the study was conducted.Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

References

    1. Hirano, I. (2016). 2015 David Y. Graham Lecture: The first two decades of eosinophilic esophagitis – from acid reflux to food allergy. The American Journal of Gastroenterology, 111, 770–776. - PMC - PubMed
    1. Furuta GT, Liacouras CA, Collins MH, Gupta SK, Justinich C, Putnam PE, Bonis P, Hassall E, Straumann A, Rothenberg ME. Eosinophilic esophagitis in children and adults: A systematic review and consensus recommendations for diagnosis and treatment. Gastroenterology. 2007;133:1342–1363. doi: 10.1053/j.gastro.2007.08.017. - DOI - PubMed
    1. DeBrosse CW, Franciosi JP, King EC, Buckmeier Butz BK, Greenberg AB, Collins MH, Abonia JP, Assa'ad A, Putnam PE, Rothenberg ME. Long-term outcomes in pediatric-onset esophageal eosinophilia. The Journal of Allergy and Clinical Immunology. 2011;128:132–138. doi: 10.1016/j.jaci.2011.05.006. - DOI - PMC - PubMed
    1. Schoepfer A, Safroneeva E, Bussmann C, Kuchen T, Portmann S, Simon HU, Straumann A. Delay in diagnosis of eosinophilic esophagitis increases risk for stricture formation in a time-dependent manner. Gastroenterology. 2013;145:1230–1236. doi: 10.1053/j.gastro.2013.08.015. - DOI - PubMed
    1. Dellon ES, Jensen ET, Martin CF, Shaheen NJ, Kappelman MD. Prevalence of eosinophilic esophagitis in the United States. Clinical Gastroenterology and Hepatology. 2014;12:589–596. doi: 10.1016/j.cgh.2013.09.008. - DOI - PMC - PubMed