Augmentation therapy with human α1-protease inhibitor:: Whom to treat when?

Background: Patients with a congenital alpha(1)-protease inhibitor (alpha(1)-Pi) deficiency frequently develop a pulmonary emphysema early in life. The replacement of the missing glycoprotein can correct the protease-antiprotease imbalance. Studies: Clinical studies evaluating the course of the lung disease show a slowed progression of the emphysema in patients with moderately impaired lung function (forced expiratory volume in one second between 30 and 65% of predicted normal) as well as a reduced mortality. In this group of patients, weekly augmentation therapy with human alpha(1)-Pi seem to be efficacious. However, from these studies no final conclusion can be drawn regarding the augmentation therapy of patients with normal lung function without a rapid progression of the disease or patients with severely impaired lung function.