Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database
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...
Main Authors: | , , , , , , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
European Respiratory Society
2021-08-01
|
Series: | ERJ Open Research |
Online Access: | http://openres.ersjournals.com/content/7/3/00194-2021.full |
id |
doaj-c5c29e735f444343bcd757fbdb5cebf4 |
---|---|
record_format |
Article |
spelling |
doaj-c5c29e735f444343bcd757fbdb5cebf42021-10-04T13:41:20ZengEuropean Respiratory SocietyERJ Open Research2312-05412021-08-017310.1183/23120541.00194-202100194-2021Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR databaseIris G.M. Schouten0Marise J. Kasteleyn1Roula Tsonaka2Robert Bals3Alice C. Turner4Ilaria Ferrarotti5Angelo G. Corsico6Beatriz Lara7Marc Miravitlles8Robert A. Stockley9Jan Stolk10 Dept of Pulmonology, Leiden University Medical Center, Leiden, the Netherlands Dept of Pulmonology, Leiden University Medical Center, Leiden, the Netherlands Dept of Advanced Data Management and Medical Statistics, Leiden University Medical Center, Leiden, the Netherlands Dept of Pulmonology, Hospital of the University of the Saarland, Homburg, Germany University of Birmingham, Institute of Applied Health Research, Edgbaston, UK Fondazione IRCCS Policlinico San Matteo, Centro Diagnosi AATD – Clinica Malattie Apparato Respiratorio, Pavia, Italy Fondazione IRCCS Policlinico San Matteo, Centro Diagnosi AATD – Clinica Malattie Apparato Respiratorio, Pavia, Italy Dept of Respiratory Medicine, University Hospitals Coventry and Warwickshire NHS Trust, Coventry, UK Pneumology Dept, Hospital Universitari Vall d'Hebron, Vall d'Hebron Institut de Recerca, Barcelona, Spain Lung Investigation Unit, Queen Elizabeth Hospital, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK Dept of Pulmonology, Leiden University Medical Center, Leiden, the Netherlands 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.http://openres.ersjournals.com/content/7/3/00194-2021.full |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Iris G.M. Schouten Marise J. Kasteleyn Roula Tsonaka Robert Bals Alice C. Turner Ilaria Ferrarotti Angelo G. Corsico Beatriz Lara Marc Miravitlles Robert A. Stockley Jan Stolk |
spellingShingle |
Iris G.M. Schouten Marise J. Kasteleyn Roula Tsonaka Robert Bals Alice C. Turner Ilaria Ferrarotti Angelo G. Corsico Beatriz Lara Marc Miravitlles Robert A. Stockley Jan Stolk Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database ERJ Open Research |
author_facet |
Iris G.M. Schouten Marise J. Kasteleyn Roula Tsonaka Robert Bals Alice C. Turner Ilaria Ferrarotti Angelo G. Corsico Beatriz Lara Marc Miravitlles Robert A. Stockley Jan Stolk |
author_sort |
Iris G.M. Schouten |
title |
Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database |
title_short |
Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database |
title_full |
Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database |
title_fullStr |
Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database |
title_full_unstemmed |
Long-term effect of α1-antitrypsin augmentation therapy on the decline of FEV1 in deficient patients: an analysis of the AIR database |
title_sort |
long-term effect of α1-antitrypsin augmentation therapy on the decline of fev1 in deficient patients: an analysis of the air database |
publisher |
European Respiratory Society |
series |
ERJ Open Research |
issn |
2312-0541 |
publishDate |
2021-08-01 |
description |
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. |
url |
http://openres.ersjournals.com/content/7/3/00194-2021.full |
work_keys_str_mv |
AT irisgmschouten longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT marisejkasteleyn longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT roulatsonaka longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT robertbals longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT alicecturner longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT ilariaferrarotti longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT angelogcorsico longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT beatrizlara longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT marcmiravitlles longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT robertastockley longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase AT janstolk longtermeffectofa1antitrypsinaugmentationtherapyonthedeclineoffev1indeficientpatientsananalysisoftheairdatabase |
_version_ |
1716844082507022336 |