Alpha-1 Antitrypsin Deficiency: a Rare Disease?

Cortes-Lopez, Roxana

doi:10.1007/s11882-020-00942-4

Cited by 4 publications

(2 citation statements)

References 65 publications

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“…For patients with AATD that do not meet the criteria for augmentation therapy, there are many new therapeutic technologies being investigated, which are discussed in more detail within the chapter on research and emerging treatment strategies. 111 , 112…”

Section: Specific Pharmacological Treatment Of Aatd-associated Lung Disease: Augmentation Therapymentioning

confidence: 99%

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Campos

2021

Therapeutic Advances in Chronic Disease

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Management of lung disease in patients with alpha-1 antitrypsin deficiency (AATD) includes both non-pharmacological and pharmacological approaches. Lifestyle changes with avoidance of environmental pollutants, including tobacco smoke, improving exercise levels and nutritional status, all encompassed under a disease management program, are crucial pillars of AATD management. Non-pharmacological therapies follow conventional treatment guidelines for chronic obstructive pulmonary disease. Specific pharmacological treatment consists of administering exogenous alpha-1 antitrypsin (AAT) protein intravenously (augmentation therapy). This intervention raises AAT levels in serum and lung epithelial lining fluid, increases anti-elastase capacity, and decreases several inflammatory mediators in the lung. Radiologically, augmentation therapy reduces lung density loss over time, thus delaying disease progression. The effect of augmentation therapy on other lung-related outcomes, such as exacerbation frequency/length, quality of life, lung function decline, and mortality, are less clear and questions regarding dose optimization or route of administration are still debatable. This review discusses the rationale and available evidence for these interventions in AATD.

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Section: Specific Pharmacological Treatment Of Aatd-associated Lung Disease: Augmentation Therapymentioning

confidence: 99%

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Campos

2021

Therapeutic Advances in Chronic Disease

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“…Inherited deficiency in AAT (AATD) is associated with various diseases such as lung emphysema and other respiratory diseases, liver diseases, panniculitis, kidney disease, heart disease, rheumatoid arthritis, and systemic vasculitis. [1][2][3][4][5][6][7][8][9][10] In the last decade, associations between AATD and vascular disease, such as Abdominal Aortic Aneurysm (AAA), have been theorized. 11 However, further investigations are needed before this association can be confirmed.…”

Section: Introductionmentioning

confidence: 99%

Investigating the Link between Alpha-1 Antitrypsin Deficiency and Abdominal Aortic Aneurysms

Pini

Peroni

Zanotti

et al. 2021

Annals of Vascular Surgery

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Alpha-1 antitrypsin deficiency and PiS and PiZ SERPINA1 variants are associated with asthma exacerbations

Martín-González,

Hernández-Pérez,

Pérez

et al. 2023

Pulmonology

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Alpha-1 Antitrypsin Deficiency: a Rare Disease?

Cited by 4 publications

References 65 publications

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Investigating the Link between Alpha-1 Antitrypsin Deficiency and Abdominal Aortic Aneurysms

Alpha-1 antitrypsin deficiency and PiS and PiZ SERPINA1 variants are associated with asthma exacerbations

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Alpha-1 Antitrypsin Deficiency: a Rare Disease?

Cited by 4 publications

References 65 publications

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Management of lung disease in alpha-1 antitrypsin deficiency: what we do and what we do not know

Investigating the Link between Alpha-1 Antitrypsin Deficiency and Abdominal Aortic Aneurysms

Alpha-1 antitrypsin deficiency and Pi*S and Pi*Z SERPINA1 variants are associated with asthma exacerbations

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Product

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About

Alpha-1 antitrypsin deficiency and PiS and PiZ SERPINA1 variants are associated with asthma exacerbations