PMID- 32154291 OWN - NLM STAT- PubMed-not-MEDLINE LR - 20220413 IS - 2312-0541 (Print) IS - 2312-0541 (Electronic) IS - 2312-0541 (Linking) VI - 6 IP - 1 DP - 2020 Jan TI - Protocol for the EARCO Registry: a pan-European observational study in patients with alpha(1)-antitrypsin deficiency. LID - 00181-2019 [pii] LID - 10.1183/23120541.00181-2019 [doi] 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 microM (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. CI - Copyright (c)ERS 2020. FAU - Greulich, Timm AU - Greulich T AUID- ORCID: 0000-0002-2368-3014 AD - University Medical Centre Giessen and Marburg, Philipps-University, Dept of Medicine, Pulmonary and Critical Care Medicine, Member of the German Centre for Lung Research (DZL), Marburg, Germany. FAU - Altraja, Alan AU - Altraja A AUID- ORCID: 0000-0001-7798-9871 AD - Pneumology Dept, Tartu University, Tartu, Estonia. AD - Lung Clinic, Tartu University Hospital, Tartu, Estonia. FAU - Barrecheguren, Miriam AU - Barrecheguren M AD - Pneumology Dept, Hospital Universitari Vall d'Hebron/Vall d'Hebron Research Institute (VHIR), CIBER de Enfermedades Respiratorias (CIBERES), Barcelona, Spain. FAU - Bals, Robert AU - Bals R AUID- ORCID: 0000-0002-1472-9535 AD - Dept of Internal Medicine V - Pulmonology, Allergology, Intensive Care Medicine, Saarland University Hospital, Homburg, Germany. FAU - Chlumsky, Jan AU - Chlumsky J AD - Dept of Pneumology, Thomayer Hospital, First Faculty of Medicine, Charles University, Prague, Czech Republic. FAU - Chorostowska-Wynimko, Joanna AU - Chorostowska-Wynimko J AUID- ORCID: 0000-0003-1743-2961 AD - Dept of Genetics and Clinical Immunology, National Institute of Tuberculosis and Lung Diseases, Warsaw, Poland. FAU - Clarenbach, Christian AU - Clarenbach C AUID- ORCID: 0000-0003-2158-2321 AD - Division of Pulmonology, University Hospital Zurich, Zurich, Switzerland. FAU - Corda, Luciano AU - Corda L AD - Dept of Internal Medicine, Respiratory Disease Unit, Spedali Civili, Brescia, Italy. FAU - Corsico, Angelo Guido AU - Corsico AG AD - Dept of Internal Medicine and Therapeutics, Pneumology Unit IRCCS San Matteo Hospital Foundation, University of Pavia, Pavia, Italy. FAU - Ferrarotti, Ilaria AU - Ferrarotti I AUID- ORCID: 0000-0003-4892-4192 AD - Dept of Internal Medicine and Therapeutics, Pneumology Unit IRCCS San Matteo Hospital Foundation, University of Pavia, Pavia, Italy. FAU - Esquinas, Cristina AU - Esquinas C AD - Pneumology Dept, Hospital Universitari Vall d'Hebron/Vall d'Hebron Research Institute (VHIR), CIBER de Enfermedades Respiratorias (CIBERES), Barcelona, Spain. FAU - Gouder, Caroline AU - Gouder C AD - Dept of Respiratory Medicine, Mater Dei Hospital, Msida, Malta. FAU - Hecimovic, Ana AU - Hecimovic A AD - Dept of Respiratory Diseases "Jordanovac", University of Zagreb, School of Medicine, Zagreb, Croatia. FAU - Ilic, Aleksandra AU - Ilic A AD - Clinic for Pulmology, Clinical Center of Serbia, Belgrade, Serbia. AD - Faculty of Medicine, University of Belgrade, Belgrade, Serbia. FAU - Ivanov, Yavor AU - Ivanov Y AD - Pulmonary Clinic, University Hospital, Pleven, Bulgaria. FAU - Janciauskiene, Sabina AU - Janciauskiene S AD - Clinic for Pneumology, DZL, Medical University Hannover, Hannover, Germany. FAU - Janssens, Wim AU - Janssens W AUID- ORCID: 0000-0003-1830-2982 AD - Katholieke Universiteit Leuven, Laboratory of Respiratory Diseases, Dept of Chronic Disease, Metabolism and Ageing, Leuven, Belgium. AD - University Hospitals Leuven, Department of Respiratory Diseases, Leuven, Belgium. FAU - Kohler, Malcolm AU - Kohler M AD - Division of Pulmonology, University Hospital Zurich, Zurich, Switzerland. FAU - Krams, Alvils AU - Krams A AD - Faculty of Medicine, University of Latvia, Riga, Latvia. AD - Riga East University Hospital, Riga, Latvia. FAU - Lara, Beatriz AU - Lara B AUID- ORCID: 0000-0003-0090-8206 AD - Dept of Respiratory Medicine, University Hospitals of Coventry and Warwickshire, Coventry, UK. FAU - Mahadeva, Ravi AU - Mahadeva R AD - Respiratory Medicine, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK. FAU - McElvaney, Gerry AU - McElvaney G AD - Irish Centre for Rare Lung Diseases, Royal College of Surgeons in Ireland, Beaumont Hospital, Dublin, Ireland. FAU - Mornex, Jean-Francois AU - Mornex JF AD - Hospices Civils de Lyon, Service de Pneumologie, Hopital Louis Pradel, Lyon, France. AD - Universite de Lyon, Universite Lyon 1, UMR754, INRA, Lyon, France. FAU - O'Hara, Karen AU - O'Hara K AD - Alpha-1 UK Support Group, Droitwich, UK. AD - Alpha-1 Global, Miami, FL, USA. FAU - Parr, David AU - Parr D AD - Dept of Respiratory Medicine, University Hospitals of Coventry and Warwickshire, Coventry, UK. FAU - Piitulainen, Eava AU - Piitulainen E AD - Dept of Respiratory Medicine and Allergology, Skane University Hospital, Lund University, Malmo, Sweden. FAU - Schmid-Scherzer, Karin AU - Schmid-Scherzer K AD - Dept of Internal Medicine II and Pulmonology, Wilhelminenspital, Medical University of Vienna, Vienna, Austria. FAU - Seersholm, Niels AU - Seersholm N AD - Dept of Internal Medicine, Herlev and Gentofte University Hospital, Hellerup, Denmark. FAU - Stockley, Robert A AU - Stockley RA AD - Lung Investigation Unit Medicine, University Hospitals Birmingham NHS Foundation Trust Queen Elizabeth Hospital Birmingham, Birmingham, UK. FAU - Stolk, Jan AU - Stolk J AD - Dept of Pulmonology, Leiden University Medical Center, Leiden, The Netherlands. FAU - Sucena, Maria AU - Sucena M AD - Pulmonology Dept, Centro Hospitalar de Sao Joao, Porto, Portugal. FAU - Tanash, Hanan AU - Tanash H AD - Dept of Respiratory Medicine and Allergology, Skane University Hospital, Lund University, Malmo, Sweden. FAU - Turner, Alice AU - Turner A AUID- ORCID: 0000-0002-5947-3254 AD - Respiratory Medicine, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK. AD - Institute of Applied Health Research, University of Birmingham, Birmingham, UK. FAU - Ulmeanu, Ruxandra AU - Ulmeanu R AD - Marius Nasta Institute, Bucharest, Romania. AD - Faculty of Medicine, University of Medicine Oradea, Bucharest, Romania. FAU - Wilkens, Marion AU - Wilkens M AD - Patientenorganisation Alpha1 Deutschland e.V., Gernsheim, Germany. FAU - Yorgancioglu, Arzu AU - Yorgancioglu A AD - Dept of Pulmonary Diseases, Celal Bayar University, Faculty of Medicine (and the GARD Executive Committee), Manisa, Turkey. FAU - Zaharie, Ana AU - Zaharie A AD - Dept of Pneumophthisiology, University of Medicine and Pharmacy, "Marius Nasta" Institute of Pneumophthisiology, Bucharest, Romania. FAU - Miravitlles, Marc AU - Miravitlles M AUID- ORCID: 0000-0002-9850-9520 AD - Pneumology Dept, Hospital Universitari Vall d'Hebron/Vall d'Hebron Research Institute (VHIR), CIBER de Enfermedades Respiratorias (CIBERES), Barcelona, Spain. LA - eng PT - Journal Article DEP - 20200302 PL - England TA - ERJ Open Res JT - ERJ open research JID - 101671641 PMC - PMC7049712 COIS- Conflict of interest: T. Greulich reports grants from CSL-Behring, Grifols and Kamda during the conduct of the study; personal fees for lectures and advisory boards from AstraZeneca, Berlin-Chemie, Boehringer Ingelheim, Chiesi, CSL-Behring, GSK and Novartis, grants and personal fees for lectures and advisory boards from Grifols, and grants from the German Centre for Lung Research (DZL), outside the submitted work. Conflict of interest: A. Altraja reports travel support to attend the EARCO stakeholder board meeting on 17 June 2019 from EARCO during the conduct of the study; support for clinical trials and honoraria for advisory board meetings from CSL Behring, honoraria for lectures/chairmanships and advisory board meetings and travel support from Shire Pharmaceuticals, AstraZeneca, Boehringer Ingelheim, Chiesi (Norameda), GlaxoSmithKline, MSD, Novartis, Roche, Bayer and Actelion (Johnson & Johnson), research support from Pfizer, travel support from Teva, Sanofi and United Therapeutics (AOP Orphan), honoraria for lectures and travel support from Berlin-Chemie Menarini Group, and honoraria for lectures from Orion, outside the submitted work. Conflict of interest: M. Barrecheguren reports speaker fees from Menarini, Grifols, CSL Behring, Boehringer Ingelheim, Gebro Pharma and GlaxoSmithKline, and consulting fees from Novartis and GlaxoSmithKline, outside the submitted work. Conflict of interest: R. Bals reports grants and personal fees from AstraZeneca and Boehringer Ingelheim, personal fees from GlaxoSmithKline and Grifols, grants and personal fees from Novartis, personal fees from CSL Behring, grants from the German Federal Ministry of Education and Research (BMBF) Competence Network Asthma and COPD (ASCONET), Sander Stiftung, Schwiete Stiftung, Krebshilfe and Mukoviszidose e.V., outside the submitted work. Conflict of interest: J. Chlumsky reports personal fees for lectures and consultation from CSL Behring, and travel support from CSL Behring, outside the submitted work. Conflict of interest: J. Chorostowska-Wynimko reports grants, personal fees and nonfinancial support from Grifols, personal fees from Kamada, grants, personal fees and nonfinancial support from AstraZeneca, personal fees and nonfinancial support from Pfizer, MSD and BMS, personal fees from GSK, Novartis amd Chiesi, personal fees and nonfinancial support from Roche, grants and personal fees from Boehringer Ingelheim, personal fees and nonfinancial support from Abbvie, grants, personal fees and nonfinancial support from CSL Behring, personal fees from Lekam, grants, personal fees and nonfinancial support from CelonPharma, and personal fees from Takeda, outside the submitted work. Conflict of interest: C. Clarenbach reports consulting fees and travel support from Vifor Pharma, and consulting or speaker fees from Boehringer, Novartis, GSK, AstraZeneca and Sanofi, outside the submitted work. Conflict of interest: L. Corda has nothing to disclose. Conflict of interest: A.G. Corsico has nothing to disclose. Conflict of interest: I. Ferrarotti has nothing to disclose. Conflict of interest: C. Esquinas has nothing to disclose. Conflict of interest: C. Gouder has nothing to disclose. Conflict of interest: A. Hecimovic reports speaker fees from Roche, Boehringer Ingelheim, Novartis and MSD, outside the submitted work. Conflict of interest: A. Ilic has nothing to disclose. Conflict of interest: Y. Ivanov has nothing to disclose. Conflict of interest: S. Janciauskiene has nothing to disclose. Conflict of interest: W. Janssens reports receiving research grants and money for consultancy activities from Behring, AstraZeneca, Boerhinger Ingelheim, GSK, Chiesi, outside the submitted work. He is co-founder of ArtiQ, a spinoff company in healthcare. Conflict of interest: M. Kohler reports advisor fees from Novartis, Boehringer Ingelheim, AstraZeneca, GSK and Bayer, outside the submitted work. Conflict of interest: A. Krams reports personal fees and nonfinancial support from AstraZeneca, Berlin-Chemie/Menarini and Boehringer Ingelheim, personal fees from GlaxoSmithKline and Merck Serono, personal fees and nonfinancial support from Norameda (represents Chiesi in Baltic countries), personal fees from Novartis, and nonfinancial support from Mylan, outside the submitted work. Conflict of interest: B. Lara has nothing to disclose. Conflict of interest: R. Mahadeva reports personal fees from Chiesi, Astra Zeneca and Boehringer Ingelheim, and grants from Pfizer Open Air, outside the submitted work. Conflict of interest: G. McElvaney has sat on advisory boards for CSL Behring, Grifols, Vertex and Chiesi. Conflict of interest: J-F. Mornex reports nonfinancial support from ADAAT, grants, personal fees and nonfinancial support from LFB and CSL Behring, nonfinancial support from Grifols, personal fees and nonfinancial support from Polyphor, during the conduct of the study; personal fees and nonfinancial support from Actelion, GSK, Roche and Novartis, nonfinancial support from Bayer, personal fees and nonfinancial support from BMS, nonfinancial support from Pfizer and MSD, personal fees from Ellivie, and nonfinancial support from Boehringer, outside the submitted work. Conflict of interest: K. O'Hara reports nonfinancial support from ELF/ERS and Mereo BioPharma Group PLC, grants from CSL Behring, and also receives donations from individuals/companies as a result of fundraising activities; personal fees and nonfinancial support from NICE and nonfinancial support from Alpha-1 Global, outside the submitted work. Conflict of interest: D. Parr reports advisory board and consultancy fees from CSL Behring and Kamada, outside the submitted work. Conflict of interest: E. Piitulainen has nothing to disclose. Conflict of interest: K. Schmid-Scherzer has nothing to disclose. Conflict of interest: N. Seersholm has nothing to disclose. Conflict of interest: R.A. Stockley reports a grant for a small, investigator-lead project and personal fees for a trial steering board from CSL Behring; a lecture fee from GSK; advisory board fees from Vertex, inhbrix, Mereobiopharma and Novartis; and lecture fees from Grifols, during the conduct of the study. He acts in an advisory manner for several companies with an interest in alpha1-antitrypsin deficiency. Conflict of interest: J. Stolk has nothing to disclose. Conflict of interest: M. Sucena reports lecture fees and travel pay from Grifols and CSL Behring, outside the submitted work. Conflict of interest: H. Tanash has nothing to disclose. Conflict of interest: A. Turner reports grants and personal fees from CSL Behring; personal fees and nonfinancial support from Boehringer Ingelheim; nonfinancial support from GSK; grants, personal fees and nonfinancial support from AstraZeneca; grants, personal fees and nonfinancial support from Chiesi; grants from Grifols biotherapeutics, outside the submitted work. Conflict of interest: R. Ulmeanu has nothing to disclose. Conflict of interest: M. Wilkens reports that as chairman of the patient organisation Alpha1 Deutschland e.V., he does not receive personally any donations but his organisation receives money from public funds as well as from the pharmaceutical industry, including grants and travelling costs. Conflict of interest: A. Yorgancioglu reports grants from MSD, and personal fees from GSK, AstraZeneca, Abdi Ibrahim, Chiesi, Novartis and Sandoz, outside the submitted work. Conflict of interest: A. Zaharie has nothing to disclose. Conflict of interest: M. Miravitlles reports speaker fees from AstraZeneca, Boehringer Ingelheim, Chiesi, Cipla, AstraZeneca, Menarini, Rovi, Bial, Zambon, CSL Behring, Grifols and Novartis, consulting fees from AstraZeneca, Boehringer Ingelheim, Chiesi, GlaxoSmithKline, Bial, Gebro Pharma, CSL Behring, Laboratorios Esteve, Ferrer, Mereo Biopharma, Verona Pharma, TEVA, pH Pharma, Novartis and Grifols, and research grants to his institution from GlaxoSmithKline and Grifols, outside the submitted work. EDAT- 2020/03/11 06:00 MHDA- 2020/03/11 06:01 PMCR- 2020/03/02 CRDT- 2020/03/11 06:00 PHST- 2019/07/20 00:00 [received] PHST- 2019/12/04 00:00 [accepted] PHST- 2020/03/11 06:00 [entrez] PHST- 2020/03/11 06:00 [pubmed] PHST- 2020/03/11 06:01 [medline] PHST- 2020/03/02 00:00 [pmc-release] AID - 00181-2019 [pii] AID - 10.1183/23120541.00181-2019 [doi] PST - epublish SO - ERJ Open Res. 2020 Mar 2;6(1):00181-2019. doi: 10.1183/23120541.00181-2019. eCollection 2020 Jan.