TY - JOUR
T1 - Protocol for the EARCO Registry
T2 - a pan-European observational study in patients with α1-antitrypsin deficiency
AU - Greulich, Timm
AU - Altraja, Alan
AU - Barrecheguren, Miriam
AU - Bals, Robert
AU - Chlumsky, Jan
AU - Chorostowska-Wynimko, Joanna
AU - Clarenbach, Christian
AU - Corda, Luciano
AU - Corsico, Angelo Guido
AU - Ferrarotti, Ilaria
AU - Esquinas, Cristina
AU - Gouder, Caroline
AU - Hećimović, Ana
AU - Ilic, Aleksandra
AU - Ivanov, Yavor
AU - Janciauskiene, Sabina
AU - Janssens, Wim
AU - Kohler, Malcolm
AU - Krams, Alvils
AU - Lara, Beatriz
AU - Mahadeva, Ravi
AU - McElvaney, Gerry
AU - Mornex, Jean-François
AU - O'Hara, Karen
AU - Parr, David
AU - Piitulainen, Eava
AU - Schmid-Scherzer, Karin
AU - Seersholm, Niels
AU - Stockley, Robert A
AU - Stolk, Jan
AU - Sucena, Maria
AU - Tanash, Hanan
AU - Turner, Alice
AU - Ulmeanu, Ruxandra
AU - Wilkens, Marion
AU - Yorgancioğlu, Arzu
AU - Zaharie, Ana
AU - Miravitlles, Marc
N1 - Copyright ©ERS 2020.
PY - 2020/3/2
Y1 - 2020/3/2
N2 - Rationale and objectives: Alpha-1 antitrypsin deficiency (AATD) is a genetic condition that leads to an increased risk of emphysema and liver disease. Despite extensive investigation, there remain unanswered questions concerning the natural history, pathophysiology, genetics and the prognosis of the lung disease in association with AATD. The European Alpha-1 Clinical Research Collaboration (EARCO) is designed to bring together researchers from European countries and to create a standardised database for the follow-up of patients with AATD.Study design and population: The EARCO Registry is a non-interventional, multicentre, pan-European, longitudinal observational cohort study enrolling patients with AATD. Data will be collected prospectively without interference/modification of patient's management by the study team. The major inclusion criterion is diagnosed severe AATD, defined by an AAT serum level <11 µM (50 mg·dL-1) and/or a proteinase inhibitor genotype ZZ, SZ or compound heterozygotes or homozygotes of other rare deficient variants. Assessments at baseline and during the yearly follow-up visits include lung function testing (spirometry, body plethysmography and diffusing capacity of the lung), exercise capacity, blood tests and questionnaires (symptoms, quality of life and physical activity). To ensure correct data collection, there will be designated investigator staff to document the data in the case report form. All data will be reviewed by the EARCO database manager.Summary: The EARCO Registry aims to understand the natural history and prognosis of AATD better with the goal to create and validate prognostic tools to support medical decision-making.
AB - Rationale and objectives: Alpha-1 antitrypsin deficiency (AATD) is a genetic condition that leads to an increased risk of emphysema and liver disease. Despite extensive investigation, there remain unanswered questions concerning the natural history, pathophysiology, genetics and the prognosis of the lung disease in association with AATD. The European Alpha-1 Clinical Research Collaboration (EARCO) is designed to bring together researchers from European countries and to create a standardised database for the follow-up of patients with AATD.Study design and population: The EARCO Registry is a non-interventional, multicentre, pan-European, longitudinal observational cohort study enrolling patients with AATD. Data will be collected prospectively without interference/modification of patient's management by the study team. The major inclusion criterion is diagnosed severe AATD, defined by an AAT serum level <11 µM (50 mg·dL-1) and/or a proteinase inhibitor genotype ZZ, SZ or compound heterozygotes or homozygotes of other rare deficient variants. Assessments at baseline and during the yearly follow-up visits include lung function testing (spirometry, body plethysmography and diffusing capacity of the lung), exercise capacity, blood tests and questionnaires (symptoms, quality of life and physical activity). To ensure correct data collection, there will be designated investigator staff to document the data in the case report form. All data will be reviewed by the EARCO database manager.Summary: The EARCO Registry aims to understand the natural history and prognosis of AATD better with the goal to create and validate prognostic tools to support medical decision-making.
U2 - 10.1183/23120541.00181-2019
DO - 10.1183/23120541.00181-2019
M3 - Journal article
C2 - 32154291
VL - 6
JO - BMJ Open
JF - BMJ Open
SN - 2044-6055
IS - 1
ER -