RT Journal Article
SR Electronic
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
FD European Respiratory Society
SP 00194-2021
DO 10.1183/23120541.00194-2021
VO 7
IS 3
A1 Iris G.M. Schouten
A1 Marise J. Kasteleyn
A1 Roula Tsonaka
A1 Robert Bals
A1 Alice C. Turner
A1 Ilaria Ferrarotti
A1 Angelo G. Corsico
A1 Beatriz Lara
A1 Marc Miravitlles
A1 Robert A. Stockley
A1 Jan Stolk
YR 2021
UL http://openres.ersjournals.com/content/7/3/00194-2021.abstract
AB 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