Augmentation therapy in alpha-1 antitrypsin deficiency.
BACKGROUND
Alpha-1 antitrypsin deficiency is a genetic disorder that leads to early-onset emphysema. Recently, exogenous supplementation of the enzyme has become a therapeutic alternative.


OBJECTIVE
To review the role of so-called augmentation therapy with pooled human plasma alpha-1 antitrypsin as a specific treatment for emphysema caused by alpha-1 antitrypsin deficiency.


METHODS
The authors performed a Medline (1966 - 2007) search with the keywords 'alpha-1 antitrypsin deficiency' and 'therapy'. The authors focused on articles regarding biochemical and clinical efficacy.


CONCLUSIONS
Augmentation therapy has been shown to raise antiprotease serum and epithelial lining fluid levels above the 'protective threshold' value. Evidence suggests that this approach slows the decline in lung function, could reduce infection rates, might enhance survival, and is well tolerated. Questions about the cost-effectiveness of this therapy remain.
Heresi
Gustavo A
GA
Respiratory Institute, Cleveland Clinic, 9500 Euclid Avenue, Cleveland, Ohio 44195, USA.
Stoller
James K
JK
D006801:Humans; D007262:Infusions, Intravenous; D011480:Protease Inhibitors / Q000008:administration & dosage / Q000378:metabolism / Q000627:therapeutic use*; D011656:Pulmonary Emphysema / Q000188:drug therapy* / Q000201:enzymology / Q000209:etiology; D016896:Treatment Outcome; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage / Q000378:metabolism / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000150:complications / Q000188:drug therapy* / Q000201:enzymology
D011480:Protease Inhibitors; D000515:alpha 1-Antitrypsin