TY - JOUR T1 - Long-term effect of α<sub>1</sub>-antitrypsin augmentation therapy on the decline of FEV<sub>1</sub> in deficient patients: an analysis of the AIR database JF - ERJ Open Research JO - erjor DO - 10.1183/23120541.00194-2021 VL - 7 IS - 3 SP - 00194-2021 AU - Iris G.M. Schouten AU - Marise J. Kasteleyn AU - Roula Tsonaka AU - Robert Bals AU - Alice C. Turner AU - Ilaria Ferrarotti AU - Angelo G. Corsico AU - Beatriz Lara AU - Marc Miravitlles AU - Robert A. Stockley AU - Jan Stolk Y1 - 2021/07/01 UR - http://openres.ersjournals.com/content/7/3/00194-2021.abstract N2 - Background Patients with ZZ (Glu342Lys) α-1-antitrypsin deficiency (ZZ-AATD) who received augmentation therapy with α-1-antitrypsin (AAT) in randomised controlled trials over 2–3 years failed to show a significant reduction of the annual decline of forced expiratory volume in 1 s (FEV1).Methods To compare the trajectory of FEV1 change during 4 or more years in ZZ-AATD patients with emphysema receiving or not receiving intravenous augmentation therapy, a retrospective analysis of FEV1 values entered in the Alpha-1 International Registry (AIR) of ZZ-AATD patients from five different European countries (Germany, UK, Spain, Italy and the Netherlands) was performed. The post-bronchodilator FEV1 % predicted values for baseline and follow-up over time from patients were analysed using linear mixed effects models.Results Data of 374 patients were analysed: 246 untreated and 128 treated with intravenous AAT augmentation therapy. The mean±sd follow-up duration of the untreated group was 8.60±3.34 years and 8.59±2.62 years for the treated group. The mixed effects model analysis showed a mean FEV1 decline of −0.931% predicted per year (95% CI −1.144 to −0.718) in the untreated group and a decline of −1.016% predicted per year (95% CI −1.319 to −0.7145) in the treated group. The likelihood ratio test showed no difference between the two groups (p=0.71).Conclusion In our study population, we could not detect a significant difference in the annual decline of FEV1 by AAT augmentation treatment over a mean period of 8.6 years. Other approaches are needed to validate any benefit of augmentation therapy.This analysis of European real-world follow-up data of FEV1 % predicted in ZZ α1-ATD patients showed no difference in annual FEV1 decline between the i.v. α1-AT augmentation treated and untreated patients over a period of &gt;8 years https://bit.ly/35MglRc ER -