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Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database Schouten, Iris G. M. Kasteleyn, Marise J. Tsonaka, Roula Bals, Robert Ferrarotti, Ilaria Miravitlles Fernández, Marc Turner, Alice Institut Català de la Salut [Schouten IGM, Kasteleyn MJ] Dept of Pulmonology, Leiden University Medical Center, Leiden, the Netherlands. [Tsonaka R] Dept of Advanced Data Management and Medical Statistics, Leiden University Medical Center, Leiden, the Netherlands. [Bals R] Dept of Pulmonology, Hospital of the University of the Saarland, Homburg, Germany. AATD Core Network of European Reference Network LUNG. [Turner AC] University of Birmingham, Institute of Applied Health Research, Edgbaston, UK. [Ferrarotti I] Fondazione IRCCS Policlinico San Matteo, Centro Diagnosi AATD – Clinica Malattie Apparato Respiratorio, Pavia, Italy. AATD Core Network of European Reference Network LUNG. [Miravitlles M] Servei de Pneumologia, Vall d'Hebron Hospital Universitari, Barcelona, Spain. Vall d'Hebron Institut de Recerca (VHIR), Barcelona, Spain Vall d'Hebron Barcelona Hospital Campus Pulmons - Malalties obstructives - Tractament Alfa 1-antitripsina - Ús terapèutic Espirometria DISEASES::Respiratory Tract Diseases::Lung Diseases::Lung Diseases, Obstructive::Pulmonary Disease, Chronic Obstructive Other subheadings::Other subheadings::Other subheadings::/drug therapy DISEASES::Respiratory Tract Diseases::Lung Diseases::alpha 1-Antitrypsin Deficiency PHENOMENA AND PROCESSES::Circulatory and Respiratory Physiological Phenomena::Respiratory Physiological Phenomena::Pulmonary Ventilation::Forced Expiratory Volume ENFERMEDADES::enfermedades respiratorias::enfermedades pulmonares::enfermedades pulmonares obstructivas::enfermedad pulmonar obstructiva crónica Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia ENFERMEDADES::enfermedades respiratorias::enfermedades pulmonares::deficiencia de alfa 1-antitripsina FENÓMENOS Y PROCESOS::fenómenos fisiológicos respiratorios y circulatorios::fenómenos fisiológicos respiratorios::ventilación pulmonar::volumen espiratorio forzado Lung structure and function; COPD and smoking Estructura y función pulmonar; EPOC y tabaquismo Estructura i funció pulmonar; MPOC i tabaquisme 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 study was supported by Stichting AIR. 2022-04-22T13:15:27Z 2022-04-22T13:15:27Z 2021-07-01 info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion Schouten IGM, Kasteleyn MJ, Tsonaka R, Bals R, Turner AC, Ferrarotti I, et al. Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database. ERJ Open Res. 2021 Jul;7(3):00194–2021. 2312-0541 https://hdl.handle.net/11351/7377 10.1183/23120541.00194-2021 34435030 000696250500014 http://hdl.handle.net/11351/7377 eng ERJ Open Research;7(3) https://doi.org/10.1183/23120541.00194-2021 Attribution-NonCommercial 4.0 International http://creativecommons.org/licenses/by-nc/4.0/ info:eu-repo/semantics/openAccess application/pdf application/pdf European Respiratory Society Scientia