Validation of the novel Eosinophilic Esophagitis Impact Questionnaire.


Journal

Journal of patient-reported outcomes
ISSN: 2509-8020
Titre abrégé: J Patient Rep Outcomes
Pays: Germany
ID NLM: 101722688

Informations de publication

Date de publication:
27 Nov 2023
Historique:
received: 03 07 2023
accepted: 02 11 2023
medline: 29 11 2023
pubmed: 27 11 2023
entrez: 27 11 2023
Statut: epublish

Résumé

Eosinophilic esophagitis (EoE) has a detrimental effect on health-related quality of life (HRQOL). The Eosinophilic Esophagitis Impact Questionnaire (EoE-IQ) is a novel patient-reported outcome (PRO) measure assessing the impact of EoE on HRQOL. To assess suitability of the EoE-IQ, its measurement properties were evaluated. Using baseline and week 24 data from the pivotal, randomized, placebo-controlled, multinational phase 3 R668-EE-1774 trial (NCT03633617) of dupilumab, we evaluated EoE-IQ's measurement properties (including reliability, construct and known-groups validity, and ability to detect change) and established the threshold for change in scores that can be considered clinically meaningful. The analysis population comprised 239 adults and adolescents with EoE. Mean age was 28.1 (standard deviation, 13.14) years; 63.6% were male, and 90.4% were White. Reliability estimates for the EoE-IQ average score exceeded acceptable thresholds for patients who were stable as indicated by ratings of Patient Global Impression of Severity (PGIS) and Change (PGIC) (intraclass correlation coefficients, 0.75 and 0.81). Construct validity correlations with other EoE-specific PRO scores were moderate at baseline (|r|= 0.44-0.60) and moderate to strong at week 24 (|r|= 0.61-0.72). In known-groups analysis, EoE-IQ average score discriminated among groups of patients at varying EoE severity levels defined by PGIS scores. A ≥ 0.6-point reduction in EoE-IQ average score (where scores range from 1 to 5, with higher scores indicating worse HRQOL) from baseline to week 24 can be considered clinically meaningful. The EoE-IQ's measurement properties are acceptable, making it a valid, reliable measure of the HRQOL impacts of EoE among adults and adolescents. ClinicalTrials.gov, NCT03633617. Registered August 14, 2018, https://clinicaltrials.gov/study/NCT03633617 .

Sections du résumé

BACKGROUND BACKGROUND
Eosinophilic esophagitis (EoE) has a detrimental effect on health-related quality of life (HRQOL). The Eosinophilic Esophagitis Impact Questionnaire (EoE-IQ) is a novel patient-reported outcome (PRO) measure assessing the impact of EoE on HRQOL. To assess suitability of the EoE-IQ, its measurement properties were evaluated.
METHODS METHODS
Using baseline and week 24 data from the pivotal, randomized, placebo-controlled, multinational phase 3 R668-EE-1774 trial (NCT03633617) of dupilumab, we evaluated EoE-IQ's measurement properties (including reliability, construct and known-groups validity, and ability to detect change) and established the threshold for change in scores that can be considered clinically meaningful.
RESULTS RESULTS
The analysis population comprised 239 adults and adolescents with EoE. Mean age was 28.1 (standard deviation, 13.14) years; 63.6% were male, and 90.4% were White. Reliability estimates for the EoE-IQ average score exceeded acceptable thresholds for patients who were stable as indicated by ratings of Patient Global Impression of Severity (PGIS) and Change (PGIC) (intraclass correlation coefficients, 0.75 and 0.81). Construct validity correlations with other EoE-specific PRO scores were moderate at baseline (|r|= 0.44-0.60) and moderate to strong at week 24 (|r|= 0.61-0.72). In known-groups analysis, EoE-IQ average score discriminated among groups of patients at varying EoE severity levels defined by PGIS scores. A ≥ 0.6-point reduction in EoE-IQ average score (where scores range from 1 to 5, with higher scores indicating worse HRQOL) from baseline to week 24 can be considered clinically meaningful.
CONCLUSIONS CONCLUSIONS
The EoE-IQ's measurement properties are acceptable, making it a valid, reliable measure of the HRQOL impacts of EoE among adults and adolescents.
TRIAL REGISTRATION BACKGROUND
ClinicalTrials.gov, NCT03633617. Registered August 14, 2018, https://clinicaltrials.gov/study/NCT03633617 .

Identifiants

pubmed: 38010430
doi: 10.1186/s41687-023-00654-z
pii: 10.1186/s41687-023-00654-z
pmc: PMC10682344
doi:

Banques de données

ClinicalTrials.gov
['NCT03633617']

Types de publication

Clinical Trial Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

120

Informations de copyright

© 2023. The Author(s).

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Auteurs

Eilish McCann (E)

Regeneron Pharmaceuticals, Tarrytown, NY, USA. eilish.mccann@regeneron.com.

Mirna Chehade (M)

Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, NY, USA.

Jonathan M Spergel (JM)

Division of Allergy and Immunology, Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine at University of Pennsylvania School of Medicine, Philadelphia, PA, USA.

Andrew Yaworsky (A)

Adelphi Values, Boston, MA, USA.

Tara Symonds (T)

Clinical Outcomes Solutions, Folkestone, Kent, CT19 4RH, UK.

Jonathan Stokes (J)

Adelphi Values, Boston, MA, USA.

Sarette T Tilton (ST)

Sanofi Global, Bridgewater, NJ, USA.

Xian Sun (X)

Regeneron Pharmaceuticals, Tarrytown, NY, USA.

Siddhesh Kamat (S)

Regeneron Pharmaceuticals, Tarrytown, NY, USA.

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Classifications MeSH