Augmentation therapy with human alpha 1-protease inhibitor: whom to treat when?

Abstract

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. 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.