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Schouten, I.G.M.; Kasteleyn, M.J.; Tsonaka, R.; Bals, R.; Turner, A.C.; Ferrarotti, I.; ... ; Stolk, J. (2021)

Long-term effect of alpha(1)-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database

Article / Letter to editor
Background Patients with ZZ (Glu342Lys) alpha-1-antitrypsin deficiency (ZZ-AATD) who received augmentation therapy with alpha-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 postbronchodilator 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...Show moreBackground Patients with ZZ (Glu342Lys) alpha-1-antitrypsin deficiency (ZZ-AATD) who received augmentation therapy with alpha-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 postbronchodilator 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.Show less
All authors
Schouten, I.G.M.; Kasteleyn, M.J.; Tsonaka, R.; Bals, R.; Turner, A.C.; Ferrarotti, I.; Corsico, A.G.; Lara, B.; Miravitlles, M.; Stockley, R.A.; Stolk, J.
Date
2021-07-01
Journal
ERJ Open Research
Volume
7
Issue
3
DOI
doi:10.1183/23120541.00194-2021
Link
https://openres.ersjournals.com/content/erjor/7/3/00194-2021.full.pdf