SynthesisBMJ open respiratory research2025
Patient-reported outcome measures for use in patients with alpha-1-antitrypsin deficiency: results of a systematic selection process.
Synthesis in BMJ open respiratory research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled 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.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed, 1 synthesis or guideline pooled it.
- Patient-reported outcome measures for use in patients with alpha-1-antitrypsin deficiency: results of a systematic selection process.BMJ open respiratory research · 2025Pooled it
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveAlpha-1-antitrypsin deficiency (AATD) is a hereditary condition associated with the risk of developing chronic lung and liver disease. We aimed for a patient-reported outcome measure (PROM) that addresses generic, lung-specific and liver-specific aspects of quality of life (QoL) in individuals with AATD. Rather than developing a new PROM, we evaluated existing PROMs.
methodAn extensive literature search and eligibility assessment, accompanied by standardised group processes including patient representatives, were conducted in accordance with the guidelines in this research area (COnsensus-based Standards for the selection of health Measurement INstruments, COSMIN; Preferred Reporting Items for Systematic Reviews and Meta-Analyses, Prisma; Peer Review of Electronic Search Strategies, PRESS).
resultsOver 1200 records were identified and screened. In total, 427 PROMs were obtained and assessed for eligibility. 15 of the 247 PROMs fulfilled the predefined eligibility criteria. The final selection of the three PROMs-EuroQoL 5 Dimension 5 Level (generic), COPD Assessment Test (lung-specific) and Chronic Liver Disease Quality of Life (liver-specific)-was guided by factors such as brevity, ease of use, interpretability, strong content validity, availability of minimal clinically important differences, sensitivity to change, ability to differentiate disease severity and availability of reference data. A questionnaire was assembled that incorporated the three selected PROMs, as well as additional QoL issues not covered by them, to ensure a comprehensive assessment of patient-reported outcomes.
conclusionThe PROM named Assessment of Lung, Liver and Patient Health in Alpha-1 (ALPHA) was designed to address generic, lung-specific as well as liver-specific QoL issues in patients with AATD. As a next step, the validity and feasibility in clinical practice of the designed questionnaire will be evaluated. PROSPERO REGISTRATION NUMBER: CRD42021265360.
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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.