ArticleEuropean respiratory review : an official journal of the European Respiratory Society2023
Nine controversial questions about augmentation therapy for alpha-1 antitrypsin deficiency: a viewpoint.
Article in European respiratory review : an official journal of the European Respiratory Society, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 19 citations in OpenAlex.
- Alpha-1 Antitrypsin Deficiency Beyond COPD and Emphysema: A Narrative Review.Medical sciences (Basel, Switzerland) · 2026Review
- Characteristics of bronchiectasis in patients with different genotypes of severe αERJ open research · 2026Article
- Lung disease in never-smokers with severe αERJ open research · 2025Article
- 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
- Alpha-1 Antitrypsin Deficiency and Bronchial Asthma: Current Challenges.Biomolecules · 2025Review
- Is Disease Stability an Attainable Chronic Obstructive Pulmonary Disease Treatment Goal?American journal of respiratory and critical care medicine · 2025Review
- Response to: risk of lung disease with the Pi*SS genotype of alpha-1 antitrypsin: the evidence in context.Respiratory research · 2025Article
- Prevalence of SERPINA1 mutations in a bronchiectasis cohort: implications of extended screening for alpha-1 antitrypsin deficiency.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2025Article
- Assessment and monitoring of lung disease in patients with severe alpha 1 antitrypsin deficiency: a european delphi consensus of the EARCO group.Respiratory research · 2024Article
- Antitrypsin deficiency: still more to learn about the lung after 60 years.ERJ open research · 2024Review
- Risk of lung disease in the PI*SS genotype of alpha-1 antitrypsin: an EARCO research project.Respiratory research · 2024Article
- Airborne indoor allergen serine proteases and their contribution to sensitisation and activation of innate immunity in allergic airway disease.European respiratory review : an official journal of the European Respiratory Society · 2024Review
- Recommendations for the diagnosis and treatment of alpha-1 antitrypsin deficiency.Jornal brasileiro de pneumologia : publicacao oficial da Sociedade Brasileira de Pneumologia e Tisilogia · 2024Article
- Delphi Consensus on the Management of Patients With Advanced COPD: COPD-Avanz Working Group.Open respiratory archivesArticle
- Personalised indication of augmentation therapy for emphysema associated with severe alpha-1 antitrypsin deficiency: a case series.Therapeutic advances in respiratory diseaseArticle
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Augmentation therapy with intravenous alpha-1 antitrypsin is the only specific treatment for alpha-1 antitrypsin deficiency (AATD)-associated emphysema. This treatment has been available and remained basically unchanged for more than 35 years, but many questions persist regarding its indications, regimen of administration and efficacy. Because AATD is a rare disease, it has not been possible to conduct randomised, placebo-controlled trials that are adequately powered for the usual outcomes analysed in non-AATD-related COPD, such as lung function decline, exacerbations, symptoms or quality of life. New outcomes such as lung densitometry measured by computed tomography are more sensitive for identifying emphysema progression but are not widely accepted by regulatory agencies. In addition, clinical manifestations, severity and the natural history of lung disease associated with AATD are very heterogeneous, which means that individual prediction of prognosis is challenging. Therefore, the indication for augmentation is sometimes a dilemma between initiating treatment in individuals who may not develop significant lung disease or in whom disease will not progress and delaying it in patients who will otherwise rapidly and irreversibly progress.Other areas of debate are the possible indication for augmentation in patients with severe AATD and respiratory diseases other than emphysema, such as bronchiectasis or asthma, and the use of therapy after lung transplant in AATD patients. All these uncertainties imply that the indication for treatment must be personalised in expert reference centres after in-depth discussion of the pros and cons of augmentation with the patient.
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