Alpha-1 antitrypsin: now available, but do we need it?
Severe alpha1-antitrypsin (AAT) deficiency is the best characterised genetic risk factor for the development of emphysema. AAT has a wide spectrum of antiprotease activity and its primary function is inhibition of neutrophil elastase in the lung. Smokers with this genetic defect develop severe impairment in their fifth to sixth decade of life. Intravenous administration of human AAT is well tolerated and has been shown to increase the levels of AAT in the alveolar lining fluid of individuals with this deficiency. In contrast to the proof of the biochemical effectiveness of augmentation treatment, the favourable clinical effect of AAT on pulmonary function, emphysema progression, morbidity and survival has not been persuasively demonstrated by prospective controlled clinical trials and remains controversial.
Russi
Erich W
EW
Pulmonary Division, Department of Internal Medicine, University Hospital, Zurich, Switzerland. erich.russi@usz.ch
D006801:Humans; D007262:Infusions, Intravenous; D019272:Leukocyte Elastase / Q000037:antagonists & inhibitors*; D029424:Pulmonary Disease, Chronic Obstructive / Q000503:physiopathology; D011656:Pulmonary Emphysema / Q000188:drug therapy / Q000503:physiopathology*; D012907:Smoking / Q000009:adverse effects; D014361:Trypsin Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000188:drug therapy*
D014361:Trypsin Inhibitors; D000515:alpha 1-Antitrypsin; D019272:Leukocyte Elastase