Long-term augmentation therapy in twenty patients with severe alpha-1-antitrypsin deficiency--three-year follow-up

The purpose of this uncontrolled, prospective study was to evaluate the influence of long-term augmentation therapy with plasma-derived alpha 1-antitrypsin (AAT) on lung function parameters in patients with severe emphysema caused by AAT deficiency. Twenty patients (mean age 48 years) received AAT infusions once weekly for up to 36 months. No adverse effects were observed. At the beginning of the study, mean (+/- SEM) FEV1 was 1.35 +/- 0.12 liters and mean TLCO was 54 +/- 4% of predicted. After 36 months of treatment, mean FEV1 was 1.25 +/- 0.12 liters (p = n.s) and the TLCO was 52 +/- 4% predicted (p = n.s). Similar values were obtained before and after therapy for FVC (2.79 +/- 0.23 vs. 2.82 +/- 0.21 liters), MEF50 (0.72 +/- 0.09 vs. 0.68 +/- 0.08 liters/s), RV (4.60 +/0 0.44 vs 4.45 +/- 0.311) and TLC (7.72 +/- 0.49 vs. 7.38 +/- 0.42 l). The calculated annual loss of FEV1 (35.6 ml/year) was smaller than in historical untreated patients with AAT deficiency.