Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients: an analysis of the AIR database

Research output: Contribution to journalArticlepeer-review

Standard

Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients : an analysis of the AIR database. / Schouten, Iris G.M. ; Kasteleyn, Marise J. ; Tsonaka, Roula ; Bals, Robert; Turner, Alice; Ferrarotti, Ilaria; Corsico, Angelo G. ; Lara, Beatriz ; Miravitlles, Marc; Stockley, Robert A; Stolk, Jan.

In: ERJ Open Research, Vol. 7, No. 3, 00194-2021, 23.08.2021.

Research output: Contribution to journalArticlepeer-review

Harvard

Schouten, IGM, Kasteleyn, MJ, Tsonaka, R, Bals, R, Turner, A, Ferrarotti, I, Corsico, AG, Lara, B, Miravitlles, M, Stockley, RA & Stolk, J 2021, 'Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients: an analysis of the AIR database', ERJ Open Research, vol. 7, no. 3, 00194-2021. https://doi.org/10.1183/23120541.00194-2021

APA

Schouten, I. G. M., Kasteleyn, M. J., Tsonaka, R., Bals, R., Turner, A., Ferrarotti, I., Corsico, A. G., Lara, B., Miravitlles, M., Stockley, R. A., & Stolk, J. (2021). Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients: an analysis of the AIR database. ERJ Open Research, 7(3), [00194-2021]. https://doi.org/10.1183/23120541.00194-2021

Vancouver

Author

Schouten, Iris G.M. ; Kasteleyn, Marise J. ; Tsonaka, Roula ; Bals, Robert ; Turner, Alice ; Ferrarotti, Ilaria ; Corsico, Angelo G. ; Lara, Beatriz ; Miravitlles, Marc ; Stockley, Robert A ; Stolk, Jan. / Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients : an analysis of the AIR database. In: ERJ Open Research. 2021 ; Vol. 7, No. 3.

Bibtex

@article{9655dcab91ca40458a4e923cd800f48f,
title = "Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients: an analysis of the AIR database",
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 (FEV 1). Methods: To compare the trajectory of FEV 1 change during 4 or more years in ZZ-AATD patients with emphysema receiving or not receiving intravenous augmentation therapy, a retrospective analysis of FEV 1 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 1 % 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 FEV 1 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 FEV 1 by AAT augmentation treatment over a mean period of 8.6 years. Other approaches are needed to validate any benefit of augmentation therapy. ",
author = "Schouten, {Iris G.M.} and Kasteleyn, {Marise J.} and Roula Tsonaka and Robert Bals and Alice Turner and Ilaria Ferrarotti and Corsico, {Angelo G.} and Beatriz Lara and Marc Miravitlles and Stockley, {Robert A} and Jan Stolk",
year = "2021",
month = aug,
day = "23",
doi = "10.1183/23120541.00194-2021",
language = "English",
volume = "7",
journal = "ERJ Open Research",
issn = "2312-0541",
publisher = "European Respiratory Society",
number = "3",

}

RIS

TY - JOUR

T1 - Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV 1 in deficient patients

T2 - an analysis of the AIR database

AU - Schouten, Iris G.M.

AU - Kasteleyn, Marise J.

AU - Tsonaka, Roula

AU - Bals, Robert

AU - Turner, Alice

AU - Ferrarotti, Ilaria

AU - Corsico, Angelo G.

AU - Lara, Beatriz

AU - Miravitlles, Marc

AU - Stockley, Robert A

AU - Stolk, Jan

PY - 2021/8/23

Y1 - 2021/8/23

N2 - 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 (FEV 1). Methods: To compare the trajectory of FEV 1 change during 4 or more years in ZZ-AATD patients with emphysema receiving or not receiving intravenous augmentation therapy, a retrospective analysis of FEV 1 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 1 % 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 FEV 1 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 FEV 1 by AAT augmentation treatment over a mean period of 8.6 years. Other approaches are needed to validate any benefit of augmentation therapy.

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 (FEV 1). Methods: To compare the trajectory of FEV 1 change during 4 or more years in ZZ-AATD patients with emphysema receiving or not receiving intravenous augmentation therapy, a retrospective analysis of FEV 1 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 1 % 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 FEV 1 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 FEV 1 by AAT augmentation treatment over a mean period of 8.6 years. Other approaches are needed to validate any benefit of augmentation therapy.

U2 - 10.1183/23120541.00194-2021

DO - 10.1183/23120541.00194-2021

M3 - Article

C2 - 34435030

VL - 7

JO - ERJ Open Research

JF - ERJ Open Research

SN - 2312-0541

IS - 3

M1 - 00194-2021

ER -