ArticleRespiratory research2018
Health status decline in α-1 antitrypsin deficiency: a feasible outcome for disease modifying therapies?
Article in Respiratory research, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 3 of them syntheses that pooled it.
What it found
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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
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
16 citing papers in PubMed, 3 syntheses or guidelines pooled it, 20 citations in OpenAlex.
- The Relationship Between Gas Transfer Measurements, Lung Volumes, and Spirometric Indices in Alpha-1 Antitrypsin Deficiency and Non-AATD COPD: A Systematic Review.International journal of chronic obstructive pulmonary disease · 2026Pooled it
- Pulmonary Function Decline in Alpha-1 Antitrypsin Deficiency: A Systematic Review and Meta-Analysis.International journal of chronic obstructive pulmonary disease · 2026Pooled it
- Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews.European respiratory review : an official journal of the European Respiratory Society · 2022Pooled it
- The Biological Effects of Double-Dose Alpha-1 Antitrypsin Augmentation Therapy. A Pilot Clinical Trial.American journal of respiratory and critical care medicine · 2019Trial
- Trial
- Phenotypic Expression of Respiratory Diseases and Tailored Treatment in Patients with Intermediate Alpha-1 Antitrypsin Deficiency: Evidence from a Retrospective Analysis of a Selected Cohort of Patients.Medicina (Kaunas, Lithuania) · 2025Article
- Is It Time Alpha-1 Antitrypsin Deficiency Had a Specific Patient Reported Outcome Measure? A Review.Patient related outcome measures · 2025Review
- Antitrypsin deficiency: still more to learn about the lung after 60 years.ERJ open research · 2024Review
- Assessing the efficacy of AlphaHeliyon · 2024Review
- Quality of Life and Mortality Outcomes for Augmentation Naïve and Augmented Patients with Severe Alpha-1 Antitrypsin Deficiency.Chronic obstructive pulmonary diseases (Miami, Fla.) · 2023Article
- Designing Clinical Trials in "Regular" COPD Versus Alpha-1 Antitrypsin Deficiency-Associated COPD: "More Alike Than Unalike?"Chronic obstructive pulmonary diseases (Miami, Fla.) · 2022Article
- Alpha 1 Antitrypsin Therapy in Patients with Alpha 1 Antitrypsin Deficiency: Perspectives from a Registry Study and Practical Considerations for Self-Administration During the COVID-19 Pandemic.International journal of chronic obstructive pulmonary disease · 2021Review
- Article
- Alpha-1 antitrypsin deficiency: an update on clinical aspects of diagnosis and management.Faculty reviews · 2020Review
- Advancing the understanding and treatment of lung pathologies associated with alpha 1 antitrypsin deficiency.Therapeutic advances in respiratory diseaseReview
- The importance of effective registries in pulmonary diseases and how to optimize their output.Chronic respiratory diseaseReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundTrials of disease modifying therapies in Chronic Obstructive Pulmonary Disease (COPD) provide challenges for detecting physiological and patient centred outcomes. The purpose of the current study was to monitor decline in health status in Alpha-1 antitrypsin deficiency (AATD) and determine its' relationship to conventional physiology.
methodsPatients recruited to the UK-AATD database with a median follow up of 7 years (IQR 5-10) were studied to determine annual change in St George's Respiratory Questionnaire (SGRQ), FEV
resultsAnnual decline in SGRQ had a wide range, was greater for patients with established COPD and correlated with decline in FEV
conclusionDespite AATD being a rapidly declining form of COPD, deterioration in SGRQ was slow consistent with ageing and the chronic nature of disease progression. Power calculations indicate the numbers needed to detect a difference with disease modifying therapies would be prohibitive especially in this rare cause of COPD. These data have important implications for future study design of disease modifying therapies even in COPD not associated with AATD.
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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.