Alpha-1 antitrypsin (A1-PI) treatment slows emphysema progression independent of baseline FEV1

Ficker, Joachim H.; Turner, Alice; Sandhaus, Robert A.; Wencker, Marion; Vit, Oliver; Fries, Michael; McElvaney, Noel G.

doi:10.1183/1393003.congress-2017.oa3416

Cited by 8 publications

(7 citation statements)

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“…According to the US Alpha-1 Foundation [31], intravenous augmentation therapy in those with genetically confirmed AATD is recommended for individuals with an FEV 1 ⩽65% pred and for those with necrotising panniculitis. For patients with lung disease related to AATD and FEV 1 >65%, there is need for a discussion with each individual regarding the potential benefits of reducing lung function decline with consideration of the cost of therapy and lack of evidence for such benefit, although a subanalysis of the RAPID-RCT trial suggests that AAT therapy is effective at all levels of FEV 1 impairment, and that treatment is therefore beneficial in both early-and late-stage disease [44]. In contrast, intravenous augmentation therapy is not recommended for individuals with the MZ genotype of AATD, those with lung disease due to AATD who continue to smoke and individuals with AATD and emphysema or bronchiectasis who do not have airflow obstruction.…”

Section: Best Practice In Diagnosis Of Pulmonary Diseases In Aatdmentioning

confidence: 99%

α₁-Antitrypsin deficiency and chronic respiratory disorders

et al. 2020

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α1-antitrypsin deficiency (AATD) is a hereditary disorder associated with a risk of developing liver disease and pulmonary emphysema, and other chronic respiratory disorders (mainly asthma and bronchiectasis); Z variant is the commonest deficient variant of AAT. Determining AAT concentration in serum or plasma and identifying allelic variants by phenotyping or genotyping are fundamental in the diagnosis of AATD. Initial evaluation and annual follow-up measurement of lung function, including post-bronchodilator forced expiratory volume in 1 s and gas transfer inform on disease progression. Lung densitometry is the most sensitive measure of emphysema progression, but must not be use in the follow-up of patients in routine clinical practice. The exogenous administration of purified human serum-derived AAT is the only approved specific treatment for AATD in PiZZ. AAT augmentation therapy is not recommended in PiSZ, PiMZ or current smokers of any protein phenotype, or in patients with hepatic disease. Lung volume reduction and endoscopic bronchial valve placement are useful in selected patients, whereas the survival benefit of lung transplant is unclear. There are several new lines of research in AATD to improve the diagnosis and evaluation of the response to therapy and to develop genetic and regenerative therapies and other treatments.

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Section: Best Practice In Diagnosis Of Pulmonary Diseases In Aatdmentioning

confidence: 99%

α₁-Antitrypsin deficiency and chronic respiratory disorders

et al. 2020

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“…However, some respondents would consider providing AAT in early-stage disease. Recent evidence provides a strong rationale for early intervention in AATD: data from the RAPID clinical trial programme suggest improved outcomes with early AAT therapy, with a discernible effect on lung density decline observed regardless of baseline FEV 1 [12, 32, 33].…”

Section: Discussionmentioning

confidence: 99%

Diagnosis and management of α₁-antitrypsin deficiency in Europe: an expert survey

et al. 2019

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Despite recent improvements, α1-antitrypsin deficiency (AATD) remains a rarely diagnosed and treated condition. To assess the variability of AATD diagnosis/treatment in Europe, and to evaluate clinicians’ views on methods to optimise management, specialist AATD clinicians were invited to complete a web-based survey.Surveys were completed by 15 physicians from 14 centres in 13 European countries. All respondents perceived the AATD diagnosis rate to be low in their country; 77% of physicians believed that ∼15% of cases were diagnosed. Low awareness was perceived as the greatest barrier to diagnosis. Spirometry was considered more practical than quantitative computed tomography (QCT) for monitoring AATD patients in clinical practice; QCT was considered more useful in trials. AAT therapy provision was reported to be highly variable: France and Germany were reported to treat the highest proportion (∼60%) of diagnosed patients, in contrast to the UK and Hungary, where virtually no patients receive AAT therapy. Most clinicians supported self-administration and extended dosing intervals to improve convenience of AAT therapy.This survey indicates that AATD diagnosis and management are highly heterogeneous in Europe; European cooperation is essential to generate data to support access to AAT therapy. Improving convenience of AAT therapy is an ongoing objective.

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“…In addition, response to treatment in the RAPID trial did not seem to vary by starting FEV 1 . 22 Therefore, there is no clinical rationale to limit screening and treatment of these patients by the severity of illness as defined by FEV 1 . 13 Using computerized tomography densitometry, a 2-year, double-blind, randomized, placebo-controlled trial (RAPID) and its open-label extension phase (RAPID-OLE), were the first to demonstrate the disease-modifying effect of AAT therapy on emphysema progression.…”

Section: The Impact Of Aat Therapy On Mortality and Qol Mortalitymentioning

confidence: 99%

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

Herth¹,

Sandhaus²,

Turner³

et al. 2021

COPD

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Alpha-1 antitrypsin (A1-PI) treatment slows emphysema progression independent of baseline FEV1

Cited by 8 publications

References 0 publications

α₁-Antitrypsin deficiency and chronic respiratory disorders

α₁-Antitrypsin deficiency and chronic respiratory disorders

Diagnosis and management of α₁-antitrypsin deficiency in Europe: an expert survey

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

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Alpha-1 antitrypsin (A1-PI) treatment slows emphysema progression independent of baseline FEV1

Cited by 8 publications

References 0 publications

α1-Antitrypsin deficiency and chronic respiratory disorders

α1-Antitrypsin deficiency and chronic respiratory disorders

Diagnosis and management of α1-antitrypsin deficiency in Europe: an expert survey

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

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Product

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α₁-Antitrypsin deficiency and chronic respiratory disorders

α₁-Antitrypsin deficiency and chronic respiratory disorders

Diagnosis and management of α₁-antitrypsin deficiency in Europe: an expert survey