Long-term effect of α 1 -antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients: an analysis of the AIR database

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 (FEV ). To compare the traject...

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Published in:ERJ open research 2021-07, Vol.7 (3), p.194
Main 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
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Language:English
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Summary: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 (FEV ). To compare the trajectory of FEV change during 4 or more years in ZZ-AATD patients with emphysema receiving or not receiving intravenous augmentation therapy, a retrospective analysis of FEV 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 FEV % predicted values for baseline and follow-up over time from patients were analysed using linear mixed effects models. 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 FEV 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). In our study population, we could not detect a significant difference in the annual decline of FEV by AAT augmentation treatment over a mean period of 8.6 years. Other approaches are needed to validate any benefit of augmentation therapy.
ISSN:2312-0541
2312-0541
DOI:10.1183/23120541.00194-2021