ALPHA(1)-ANTITRYPSIN - HOPE ON THE HORIZON FOR EMPHYSEMA SUFFERERS

alpha(1)-Antitrypsin (alpha(1)AT) deficiency is the most common geneti c cause of liver disease in children and emphysema in adults. Therapy for pulmonary disease attributable to alpha(1)AT deficiency includes a lpha(1)AT augmentation therapy along with supportive measures. The alp ha(1)AT preparation that is currently used for therapy is derived from fractionated plasma. The results of clinical trials suggest that augm entation therapy with alpha(1)AT slows the progression of emphysema an d causes few adverse events. Patients with plasma levels of alpha(1)AT that are <11 mu mol/L and who have airway obstruction should be consi dered for augmentation therapy. Novel approaches include the administr ation of aerosolised alpha(1)AT, recombinant alpha(1)AT, gene therapy and synthetic elastase inhibitors.