Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes (Q35063726)

6 August 2017

1 reference

title

Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes (English)

1 reference

alpha-1-antitrypsin deficiency

6 August 2017

1 reference

Sanjay H Chotirmall

1

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author name string

Mazen Al-Alawi

series ordinal

2

1 reference

PubMed ID

25673994

6 August 2017

Thomas McEnery

series ordinal

3

1 reference

PubMed ID

25673994

6 August 2017

Noel G McElvaney

series ordinal

4

1 reference

6 August 2017

29 January 2015

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Therapeutics and Clinical Risk Management

6 August 2017

1 reference

6 August 2017

11

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6 August 2017

143-151

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Creative Commons Attribution-NonCommercial 3.0 Unported

6 August 2017

start time

1 January 2015

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

0 references

cites work

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Intrapleural administration of an AAVrh.10 vector coding for human α1-antitrypsin for the treatment of α1-antitrypsin deficiency

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

8 July 2018

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Therapeutic efficacy of α-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometry

8 July 2018

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Pharmacokinetic comparability of Prolastin®-C to Prolastin® in alpha₁-antitrypsin deficiency: a randomized study

8 July 2018

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Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method

8 July 2018

1 reference

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Alpha-1-antitrypsin augmentation therapy in deficient individuals enrolled in the Alpha-1 Foundation DNA and Tissue Bank

8 July 2018

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Societal implications of the pharmacoeconomics of alpha1-antitrypsin deficiency.

8 July 2018

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

8 July 2018

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alpha1-Antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary note.

8 July 2018

1 reference

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Elafin, an elastase-specific inhibitor, is cleaved by its cognate enzyme neutrophil elastase in sputum from individuals with cystic fibrosis

8 July 2018

1 reference

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Cleavage of CXCR1 on neutrophils disables bacterial killing in cystic fibrosis lung disease

8 July 2018

1 reference

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Alpha-1-antitrypsin deficiency: optimal therapeutic regimen based on population pharmacokinetics

8 July 2018

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Alpha1-antitrypsin deficiency

8 July 2018

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Worldwide racial and ethnic distribution of alpha1-antitrypsin deficiency: summary of an analysis of published genetic epidemiologic surveys

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Augmentation therapy reduces frequency of lung infections in antitrypsin deficiency: a new hypothesis with supporting data

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Commercialization of proteins produced in the mammary gland

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

8 July 2018

1 reference

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Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Lung transplantation for alpha 1-antitrypsin deficiency emphysema

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Alpha 1-antitrypsin deficiency: evaluation of bronchiectasis with CT.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Mutation of antitrypsin to antithrombin. alpha 1-antitrypsin Pittsburgh (358 Met leads to Arg), a fatal bleeding disorder

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Proteins of the cystic fibrosis respiratory tract. Fragmented immunoglobulin G opsonic antibody causing defective opsonophagocytosis

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Reduction of ciliary beat frequency in vitro by sputum from patients with bronchiectasis: a serine proteinase effect

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Cirrhosis associated with alpha-1-antitrypsin deficiency: a previously unrecognized inherited disorder

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Late manifestation of chronic liver disease in adults with alpha-1-antitrypsin deficiency

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Clinical features and history of the destructive lung disease associated with alpha-1-antitrypsin deficiency of adults with pulmonary symptoms.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Smoking, lung function, and alpha 1-antitrypsin deficiency.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Recombinant DNA-produced alpha 1-antitrypsin administered by aerosol augments lower respiratory tract antineutrophil elastase defenses in individuals with alpha 1-antitrypsin deficiency

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Neutrophil elastase cleaves C3bi on opsonized pseudomonas as well as CR1 on neutrophils to create a functionally important opsonin receptor mismatch

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

The mechanism of Z alpha 1-antitrypsin accumulation in the liver

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Natural History and Life Expectancy in Severe Alpha1-Antitrypsin Deficiency, Pi Z

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Clarification of the risk of chronic obstructive pulmonary disease in α1-antitrypsin deficiency PiMZ heterozygotes.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

α1-Proteinase inhibitor (human) in the treatment of hereditary emphysema secondary to α1-antitrypsin deficiency: number and costs of years of life gained.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Tauroursodeoxycholic acid inhibits apoptosis induced by Z alpha-1 antitrypsin via inhibition of Bad.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Z alpha1-antitrypsin polymerizes in the lung and acts as a neutrophil chemoattractant.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

PULMONARY EMPHYSEMA AND ALPHA1-ANTITRYPSIN DEFICIENCY.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Major components of the direct medical costs of alpha1-antitrypsin deficiency.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Cost-effectiveness analysis of augmentation therapy for severe alpha1-antitrypsin deficiency.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Cathepsin B, L, and S cleave and inactivate secretory leucoprotease inhibitor

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

The direct medical costs of alpha(1)-antitrypsin deficiency.

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4321641

High resolution computed tomography (HRCT) in emphysema associated with alpha-1-antitrypsin deficiency

27 September 2018

1 reference

12 December 2020

Serum alpha 1-antitrypsin deficiency associated with the common S-type (Glu264----Val) mutation results from intracellular degradation of alpha 1-antitrypsin prior to secretion

1 reference

12 December 2020

Giant intracranial aneurysm and fibromuscular dysplasia in an adolescent with alpha 1-antitrypsin deficiency

1 reference

12 December 2020

Alpha 1-antitrypsin deficiency: the radiological features of pulmonary emphysema in subjects of Pi type Z and Pi type SZ: a survey by the British Thoracic Association

1 reference

12 December 2020

The liver in adolescents with alpha 1-antitrypsin deficiency

1 reference

12 December 2020

Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group

1 reference

12 December 2020

Baseline characteristics of enrollees in the National Heart, Lung and Blood Institute Registry of alpha 1-antitrypsin deficiency. Alpha 1-Antitrypsin Deficiency Registry Study Group

1 reference

12 December 2020

Alpha 1 antitrypsin deficiency: the clinical and physiological features of pulmonary emphysema in subjects homozygous for Pi type Z. A survey by the British Thoracic Association

1 reference

12 December 2020

Pharmacokinetic study of alpha1-antitrypsin infusion in alpha1-antitrypsin deficiency

1 reference

12 December 2020

Alpha 1-antitrypsin deficiency and anti-proteinase 3 antibodies in anti-neutrophil cytoplasmic antibody (ANCA)-associated systemic vasculitis

1 reference

12 December 2020

The pathologic spectrum of the nephropathy associated with alpha 1-antitrypsin deficiency

1 reference

12 December 2020

The effect of augmentation therapy on bronchial inflammation in alpha1-antitrypsin deficiency

1 reference

12 December 2020

Liver Disease in Alpha1-Antitrypsin Deficiency Detected by Screening of 200,000 Infants

1 reference

12 December 2020

Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency

1 reference

12 December 2020

Augmentation therapy for alpha1 antitrypsin deficiency: a meta-analysis

1 reference

12 December 2020

Atopy, asthma, and emphysema in patients with severe alpha-1-antitrypysin deficiency

1 reference

12 December 2020

Augmentation therapy with alpha1-antitrypsin: patterns of use and adverse events

1 reference

12 December 2020

Short-term supplementation therapy does not affect elastin degradation in severe alpha(1)-antitrypsin deficiency. The American-Italian AATD Study Group

1 reference

12 December 2020

Alpha 1-antitrypsin deficiency in Europe: geographical distribution of Pi types S and Z

1 reference

12 December 2020

Survival in relation to lung function and smoking cessation in patients with severe hereditary alpha 1-antitrypsin deficiency

1 reference

12 December 2020

Preliminary evidence that augmentation therapy diminishes degradation of cross-linked elastin in alpha-1-antitrypsin-deficient humans

1 reference

12 December 2020

Longitudinal follow-up of patients with alpha(1)-protease inhibitor deficiency before and during therapy with IV alpha(1)-protease inhibitor

1 reference

12 December 2020

Clinical features of individuals with PI*SZ phenotype of alpha 1-antitrypsin deficiency. alpha 1-Antitrypsin Deficiency Registry Study Group

1 reference

12 December 2020

Detection of circulating and endothelial cell polymers of Z and wild type alpha 1-antitrypsin by a monoclonal antibody

1 reference

12 December 2020

Augmentation of lung antineutrophil elastase capacity with recombinant human alpha-1-antitrypsin

1 reference

12 December 2020

High level expression of active human alpha-1-antitrypsin in the milk of transgenic sheep

1 reference

12 December 2020

Alpha 1-antitrypsin deficiency and liver cirrhosis in adults. An analysis of 35 Swedish autopsied cases

1 reference

12 December 2020

Acute allergic reaction and demonstration of specific IgE antibodies against alpha-1-protease inhibitor

1 reference

12 December 2020

The proteinase-antiproteinase balance in alpha-1-proteinase inhibitor-deficient lung transplant recipients

1 reference

12 December 2020

Failure to Achieve Adequate Serum Levels With Monthly Replacement Therapy in α1-Antitrypsin Deficiency

1 reference

12 December 2020

Glomerulonephritis with end-stage liver disease in childhood

1 reference

12 December 2020

Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group

1 reference

12 December 2020

Identifiers

DOI

10.2147/TCRM.S51474

1 reference

6 August 2017

1 reference

6 August 2017

1 reference

PubMed ID

25673994