Evidence map›Paper›PMID 42694028›Full record

ArticlePatient related outcome measures2026

Validation of a respiratory symptom score from the Quality of Life-Bronchiectasis Questionnaire in adults with

Daniel Serrano, Mariam Hassan, Marie-Laure Nevoret, Dayton W Yuen, Shauna McManus, Lauren Podger, Sam Parsons, Bryant Barnes, Charles L Daley, Kevin C Mange

2 registry-linked trialsAbstract read
In one paragraph

Article in Patient related outcome measures, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04677543 phase3completednot on this map

A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Validate Patient-Reported Outcome Instruments in Adult Subjects With Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)

TypeinterventionalSponsorInsmed IncorporatedRan2020 to 2023Enrolled99ConditionsMycobacterium Infections, NontuberculousArmsALIS, Azithromycin, Ethambutol, ELC
NCT04677569 phase3completednot on this map

ENCORE - A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Evaluate the Efficacy and Safety of an Amikacin Liposome Inhalation Suspension (ALIS)-Based Regimen in Adult Subjects With Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)

TypeinterventionalSponsorInsmed IncorporatedRan2021 to 2026Enrolled425ConditionsMycobacterium Infections, NontuberculousArmsALIS, Azithromycin, Ethambutol, ELC (matching placebo for ALIS)
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Daniel SerranoThe Psychometrics Team, London, UK.
Mariam HassanHealth Economics and Outcomes Research, Insmed Incorporated, Bridgewater, NJ, USA.
Marie-Laure NevoretClinical Development, Insmed Incorporated, Bridgewater, NJ, USA.
Dayton W YuenClinical Development, Insmed Incorporated, Bridgewater, NJ, USA.
Shauna McManusPatient Centered Outcomes, OPEN Health, Bethesda, MD, USA.
Lauren PodgerThe Psychometrics Team, London, UK.
Sam ParsonsPatient Centered Outcomes, OPEN Health, London, UK.
Bryant BarnesPatient Centered Outcomes, OPEN Health, Bethesda, MD, USA.
Charles L DaleyDepartment of Medicine, National Jewish Health and the University of Colorado School of Medicine, Denver, CO, USA.
Kevin C MangeClinical Development, Insmed Incorporated, Bridgewater, NJ, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Objective: To assess the psychometric properties of a respiratory symptom score from the Quality of Life-Bronchiectasis Questionnaire in adults with Methods: Structural validity, internal consistency, test-retest reliability, and known-groups validity were assessed using data from the ARISE (NCT04677543) and ENCORE (NCT04677569) phase 3 trials. Convergent validity was assessed using Exacerbations of Chronic Pulmonary Disease Tool (EXACT), EXACT Respiratory Symptoms (E-RS), St. George Respiratory Questionnaire (SGRQ), and Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale. Anchor-based meaningful within-patient change (MWPC) thresholds were estimated using the Patient Global Impression of Severity Respiratory scale. Results: In total, 362 patients were included for cross-sectional (ARISE: 97, ENCORE: 265) and longitudinal (ARISE: 99) validation analyses. Modern psychometric methods supported item relevance and an essentially unidimensional mean score. The respiratory symptom score demonstrated adequate internal consistency (McDonald's omega: 0.86, Cronbach's alpha: 0.81), test-retest reliability (intraclass correlation coefficient: 0.73), convergent validity (EXACT: -0.72, E-RS: -0.72, SGRQ: -0.77, FACIT-Fatigue: 0.51), and known-groups validity. The estimated MWPC threshold was a 16.67-point median change from baseline (95% CI: 8.33-16.83 points). Conclusions: The respiratory symptom score is a robust, sensitive, and responsive measure of respiratory symptoms in adults with a new or recurrent MAC lung disease diagnosis.

Indexed as

lung diseaseMycobacterium avium complexpatient-reported outcomepsychometric validationRespiratory symptoms

Identifiers

PMID42694028
PMCPMC13536775

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.