ArticleThe journal of allergy and clinical immunology. In practice2025
Development of a Patient-Reported Outcome Measure for Hypereosinophilic Syndrome.
Article in The journal of allergy and clinical immunology. In practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
1 citing paper in PubMed.
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Authors and funding
8 authors.
Funding
Abstract
backgroundAlthough a phase 3 double-blind, placebo-controlled registration trial of mepolizumab for hypereosinophilic syndromes (HES) used a patient-reported severity assessment, no patient reported outcome (PRO) measures have been formally developed for HES. Prior attempts to develop a PRO have been unsuccessful.
objectiveThe purpose of this study was to develop an outcome measure using accepted approaches for qualitative methods, conceptual models, and analyses to detect symptom severity and patient experience of disease activity.
methodsThe study was performed using U.S. Food and Drug Administration guidance for development of symptom scales for outcome metrics using psychometric analysis and patient-administered questionnaires. Sixty-three heterogeneous patients with different HES subtypes enrolled on a natural history study of eosinophilia were invited to participate. All participants provided informed consent. At baseline, patients filled out an HES Symptom Inventory (HES-SI) and indicated up to 4 of their most bothersome symptoms (HES-MBS). Symptom fluctuation was recorded over the course of 3 months, and clinical metrics (treatment changes) were assessed during the same period.
resultsAcross all timepoints, strong statistically significant correlations were observed for the HES-SI total score (r = 0.69-0.80; P < .0001) and HES-MBS score (r = 0.71-0.81; P < .0001) with the patients' perception of disease status (Patient Global Assessment). The HES-SI and HES-MBS showed sensitivity to change and demonstrated test-retest reliability.
conclusionsAnalyses of the HES-SI and HES-MBS scores provided strong preliminary evidence supporting the reliability and validity of these measures for use in future HES clinical trials. The ability of these scales to accurately measure changes in HES symptoms with successful treatment requires further study.
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