alpha 1-Antitrypsin Deficiency/therapy
Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency
... Abstract: Alpha-1-antitrypsin deficiency (AATD), also known as alpha1-proteinase inhibitor deficiency, is an autosomal co-dominant ...dysfunctional ...
12
Augmentation therapy for alpha-1 antitrypsin deficiency: towards a personalised approach
... augmentation therapy could be established at a given point between both ends, always recommended or never recommended, based on the existing evidence of benefits of therapy, baseline presenting ...
9
The effects of weekly augmentation therapy in patients with PiZZ α1-antitrypsin deficiency
... Prolastin therapy provides protective levels of circulating AAT protein and it is a well-tolerated and safe ...within 1 week of augmentation therapy, fluctuations of certain cytokines were observed, ...
10
Alpha 1 antitrypsin deficiency, emphysema, and liver disease Genetic basis and strategies for therapy
... Whereas all are associated with a risk for emphysema to some degree, only mutations causing intracellular accumulation of the newly synthesized alAT are associated with a risk for liver [r] ...
11
Alpha 1 antitrypsin to treat lung disease in alpha 1 antitrypsin deficiency: recent developments and clinical implications
... Abstract: Alpha 1 antitrypsin deficiency is a hereditary condition characterized by low alpha 1 proteinase inhibitor (also known as alpha 1 antitrypsin ...
14
Gene targeted therapeutics for liver disease in alpha-1 antitrypsin deficiency
... PNAs could constitute highly effi cient compounds for gene therapy as the oligomers have good stability and a strong binding affi nity for DNA and RNA. They are also nontoxic, even at relatively high concentrations. ...
14
Alpha-1 Antitrypsin Deficiency Presenting with MPO-ANCA Associated Vasculitis and Aortic Dissection
... Complex elective endovascular treatment of aortic pathology in patients with suspected connective tissue disorders is controversial because of high reintervention and complication rates [6]. Together with the patient, we ...
5
Alpha -1-antitrypsin deficiency, associated diseases and treatments: a review
... AATD individuals who have or show signs of developing significant emphysema can be treated with Prolastin, a pooled, purified, human plasma protein concentrate replacement for the missing enzyme that has been screened ...
7
Cardiovascular risk in patients with alpha-1-antitrypsin deficiency
... AAT [30, 31] These activities might include i) loss of vascu- lar elastic recoil and decreased resistance due to excess activ- ity of elastase; ii) upregulation and release of angiopoietin- like protein 4 (Angptl4) by ...
11
The TNFalpha gene relates to clinical phenotype in alpha-1-antitrypsin deficiency
... Anti-TNF therapy has been disappointing in COPD[36], but was not assessed specifically in chronic bronchitis despite the importance of TNFα in the patho- physiology of the ...
8
<p>New Patient-Centric Approaches to the Management of Alpha-1 Antitrypsin Deficiency</p>
... The fi rst project of EARCO is the foundation of a new pan-European AATD registry. 40 Since the discovery of AATD, national and international registries have been critical to increasing knowledge and understanding of the ...
11
The impact of alpha-1 antitrypsin augmentation therapy on neutrophil-driven respiratory disease in deficient individuals
... Abstract: Alpha-1 antitrypsin (AAT) is the most abundant serine protease inhibitor circulating in the ...AAT deficiency (AATD) is an autosomal codominant condition affecting an estimated ...
12
Hereditary alpha-1-antitrypsin deficiency and its clinical consequences
... Alpha-1-antitrypsin deficiency (AATD) is a genetic disorder that manifests as pulmonary emphysema, liver cirrhosis and, rarely, as the skin disease panniculitis, and is characterized by low ...
9
Alpha-1 antitrypsin deficiency: outstanding questions and future directions
... augmentation therapy or placebo and followed for two years by CT ...augmentation therapy and those who received placebo (− ...augmentation therapy. These results showed that augmen- tation ...
15
Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes
... augmentation therapy for a period of seven ...mentation therapy less frequently than ...α 1 -antiprotease batches in 1989 and 1991, which were associated with a clustering of adverse ...
9
Endobronchial coil treatment in severe emphysema patients with alpha-1 antitrypsin deficiency
... augmentation therapy. However, the impact of AAT augmentation therapy on our results should not be con- sidered as a confounding factor because AAT augmentation ...
5
Regional lung densities in alpha-1 antitrypsin deficiency compared to predicted values
... dicted all cause and respiratory mortality. However, CT densitometry consistently proved to be the best independ- ent predictor of mortality [15]. Some years later, the European Medicines Agency (EMA) approved phase II ...
10
Treatment of lung disease in alpha-1 antitrypsin deficiency: a systematic review
... augmentation therapy (as opposed to sometimes or always) when FEV 1 ,50% predicted ...augmentation therapy groups. FEV 1 decline was calculated using a slope equation in 927 patients with n = ...
14
Why has it been so difficult to prove the efficacy of alpha-1-antitrypsin replacement therapy? Insights from the study of disease pathogenesis
... Study of the consequences of abnormally folded pro- tein accumulation in hepatocytes has helped shed light on the mechanism of this gain-of-function toxicity that causes α 1 -antitrypsin deficiency ...
15
Alpha<sub>1</sub>-antitrypsin deficiency: a clinical-genetic overview
... determined by the SERPINA1 gene which expresses codominant alleles. The three most common alleles are the normal M, the S with plasma levels of 60% of normal, and the severely deficient Z with levels of about 15% of ...
11