NP Medical News about alpha 1-Antitrypsin Deficiency

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Alpha-1 antitrypsin (AAT) deficiency - what are the treatment options?
Expert Opinion in Pharmacotherapy, 11/05/09

Modrykamien M et al. – In addition to the usual treatments for emphysema, infusion of purified AAT from pooled human plasma represents a specific therapy for AAT deficiency and raises serum and epithelial lining fluid levels above the protective threshold. Substantial evidence supports the biochemical efficacy of this approach, particularly for the weekly infusion regimen. Definitive evidence of clinical efficacy is still needed, as the two available randomized controlled trials showed non–significant trends towards slowing rates of loss of lung density on lung computerized axial tomography. However, concordant results of prospective cohort studies suggest that augmentation therapy has efficacy in slowing the rate of decline of lung function in patients with moderate airflow obstruction and severe deficiency of AAT. Overall, augmentation therapy is well–tolerated and, despite its failure to satisfy criteria for cost–effectiveness, is recommended because it is the only currently available specific therapy for AAT deficiency.

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The liver in 30-year-old individuals with alpha 1 -antitrypsin deficiency
Scandinavian Journal of Gastroenterology, 11/06/09 Relevance Score: 79%

Effect of Feeding, Exercise, and Genotype on Plasma 3-Hydroxyacylcarnitines in Children With LCHAD Deficiency
Topics in Clinical Nutrition, 11/17/09 Relevance Score: 67%

Anti-apoptotic effects of Z alpha-1 antitrypsin in human bronchial epithelial cells
European Respiratory Journal, 10/20/09 Relevance Score: 66%

Z and Mmalton-1-antitrypsin deficiency-associated hepatocellular carcinoma: a genetic study
Liver International, 10/08/09 Relevance Score: 65%

C1q inhibits immune complex-induced interferon-[alpha] production in plasmacytoid dendritic cells: A novel link between C1q deficiency and systemic lupus erythematosus pathogenesis
Arthritis & Rheumatism, 10/05/09 Relevance Score: 65%