ArticleInternational journal of chronic obstructive pulmonary disease
Opinions and Attitudes of Pulmonologists About Augmentation Therapy in Patients with Alpha-1 Antitrypsin Deficiency. A Survey of the EARCO Group.
Article in International journal of chronic obstructive pulmonary disease. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 12 citations in OpenAlex.
- Bronchiectasis in 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
- Clinical characteristics of AATD-related COPD patients vary with age at diagnosis: data from the EARCO international registry.BMC pulmonary medicine · 2025Article
- Densitometric and Functional Progression in Patients with Alpha-1 Antitrypsin Deficiency Genotype SZ.Journal of clinical medicine · 2025Article
- Article
- 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
- Nine controversial questions about augmentation therapy for alpha-1 antitrypsin deficiency: a viewpoint.European respiratory review : an official journal of the European Respiratory Society · 2023Article
- Augmentation Therapy for Alpha-1 Antitrypsin Deficiency: Patient Experiences With Self-Infusion, Home Providers, and Clinics.Chronic obstructive pulmonary diseases (Miami, Fla.) · 2023Article
- Demographic and clinical characteristics of patients with αERJ open research · 2022Article
- Personalised indication of augmentation therapy for emphysema associated with severe alpha-1 antitrypsin deficiency: a case series.Therapeutic advances in respiratory diseaseArticle
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Authors and funding
11 authors at 5 institutions in 4 countries.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundAugmentation therapy (AT) is the only specific treatment licensed for patients with alpha-1 antitrypsin deficiency (AATD) associated lung disease. Since patients with severe AATD may have a very different prognosis and AT requires intravenous infusions for life, the decision to initiate AT may be challenging.
methodsThis survey was conducted on 63 experts in AATD from 13 European countries about their opinions and attitudes regarding AT. Participants were asked to rank the importance of 11 identified factors related with the prescription of AT. In addition, each participant was asked to respond to the indication of AT for 30 out of 500 hypothetical cases developed with the combinations of the 11 factors. Each case was evaluated by 3 experts to check the concordance.
resultsThe variables that scored higher on preferences for initiating AT were AAT genotype (score 8.6 from a Likert scale 0-10 (SD: 1.7)), AATD serum level (8.2 (SD:2.4)) and FEV1 (%) decline (7.9 (SD:2.4)). Among the 500 different cases, there was an agreement in indication of AT among the 3 experts in 291 (58.2%). Regarding the variables associated with AT, it was indicated to 81.9% of Pi*ZZ, 52.4% of Pi*SZ and 9.8% of Pi*MZ (p < 0.0001). For Pi*ZZ patients, multivariate analysis identified younger age, reduced FEV1 (%), higher FEV1 decline and worse emphysema as significantly associated with prescription (AUC = 0.8114); for Pi*SZ variables were younger age, worse FEV1 (%) and worse emphysema (AUC = 0.7414); and for Pi*MZ younger age, worse DLCO (%), higher DLCO decline and dyspnea (AUC = 0.8387).
conclusionThere is a high variability in the criteria for prescription of AT among European experts. Most cases were recommended AT according to guidelines, but a significant number of patients with genotype Pi*SZ and almost 10% Pi*MZ were recommended to initiate AT despite the lack of evidence of efficacy in these genotypes.
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