Abstract:
:Alpha-1 antitrypsin (AAT) deficiency is an under-recognized genetic condition that predisposes to liver disease and early-onset emphysema. Although AAT is mainly produced in the liver, its main function is to protect the lung against proteolytic damage from neutrophil elastase. The most common mutation responsible for severe AAT deficiency, the so-called Z variant, reduces serum levels by promoting polymerization of the molecule within the hepatocyte, thereby reducing secretion. Serum levels below the putative protective threshold level of 11 micromolar (mumol/L) increase the risk of emphysema. 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.
journal_name
Expert Opin Pharmacotherjournal_title
Expert opinion on pharmacotherapyauthors
Modrykamien A,Stoller JKdoi
10.1517/14656560903300111subject
Has Abstractpub_date
2009-11-01 00:00:00pages
2653-61issue
16eissn
1465-6566issn
1744-7666journal_volume
10pub_type
杂志文章,评审abstract::An increasing need for economic evaluations of non-Hodgkin's lymphoma (NHL) treatments exists. We performed a literature review on the currently available NHL economic evaluations, using PubMed and the Cochrane database. English and Dutch language papers on treatment in adults were selected. A total of 88 publications...
journal_title:Expert opinion on pharmacotherapy
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doi:10.1517/14656566.5.12.2529
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journal_title:Expert opinion on pharmacotherapy
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journal_title:Expert opinion on pharmacotherapy
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journal_title:Expert opinion on pharmacotherapy
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journal_title:Expert opinion on pharmacotherapy
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journal_title:Expert opinion on pharmacotherapy
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