Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database. Issue 3 (23rd August 2021)

• Record Type: Journal Article
• Authors: Schouten, Iris G.M.; Kasteleyn, Marise J.; Tsonaka, Roula; Bals, Robert; Turner, Alice C.; Ferrarotti, Ilaria; Corsico, Angelo G.; Lara, Beatriz; Miravitlles, Marc; Stockley, Robert A.; Stolk, Jan
• Is Part Of: ERJ open research. Volume 7:Issue 3(2021)
• Publication Date: 2021-08-23
• Subjects: Respiratory organs -- Diseases -- Periodicals; Respiration -- Periodicals; Respiration; Respiratory organs -- Diseases; Respiratory organs -- Diseases -- Treatment; Respiratory Tract Diseases; Electronic journals; Fulltext; Internet Resources; Periodicals; Periodical; 616.2005
• DOI: 10.1183/23120541.00194-2021
• Languages: English
• ISSNs: 2312-0541

Abstract

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 >8 years https://bit.ly/35MglRc