The European Alpha-1 Research Collaboration (EARCO): a new ERS Clinical Research Collaboration to promote research in alpha-1 antitrypsin deficiency

Miravitlles, Marc; Chorostowska‐Wynimko, Joanna; Ferrarotti, Ilaria; McElvaney, Noel G.; O’Hara, Karen; Stolk, Jan; Stockley, Robert A.; Turner, Alice; Wilkens, Marion; Greulich, Timm

doi:10.1183/13993003.00138-2019

Cited by 34 publications

(30 citation statements)

References 28 publications

(29 reference statements)

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…The ERS Assembly 5 (airway diseases, asthma and COPD) has created the European Alpha-1 Research Collaboration (EARCO) that is a clinical research collaboration with the aim to establish a collaborative effort that brings together multiple stakeholders, including researchers, healthcare providers, patients and industry, in order to advance understanding through clinical and scientific research and improve the quality of life of patients with AATD [61]. This pan-European initiative will enable a group of experienced and new researchers across Europe to answer fundamental questions about the epidemiology, genetics, physiopathology, clinical management and prognosis of lung disease associated with AATD.…”

Section: Ongoing Research and Future Treatmentsmentioning

confidence: 99%

α₁-Antitrypsin deficiency and chronic respiratory disorders

et al. 2020

View full text Add to dashboard Cite

α1-antitrypsin deficiency (AATD) is a hereditary disorder associated with a risk of developing liver disease and pulmonary emphysema, and other chronic respiratory disorders (mainly asthma and bronchiectasis); Z variant is the commonest deficient variant of AAT. Determining AAT concentration in serum or plasma and identifying allelic variants by phenotyping or genotyping are fundamental in the diagnosis of AATD. Initial evaluation and annual follow-up measurement of lung function, including post-bronchodilator forced expiratory volume in 1 s and gas transfer inform on disease progression. Lung densitometry is the most sensitive measure of emphysema progression, but must not be use in the follow-up of patients in routine clinical practice. The exogenous administration of purified human serum-derived AAT is the only approved specific treatment for AATD in PiZZ. AAT augmentation therapy is not recommended in PiSZ, PiMZ or current smokers of any protein phenotype, or in patients with hepatic disease. Lung volume reduction and endoscopic bronchial valve placement are useful in selected patients, whereas the survival benefit of lung transplant is unclear. There are several new lines of research in AATD to improve the diagnosis and evaluation of the response to therapy and to develop genetic and regenerative therapies and other treatments.

show abstract

Section: Ongoing Research and Future Treatmentsmentioning

confidence: 99%

α₁-Antitrypsin deficiency and chronic respiratory disorders

et al. 2020

View full text Add to dashboard Cite

show abstract

“…The collaboration aims to bring together a network of experienced and early career clinical researchers, experts in the field of AATD and industry sponsors to establish current and future research priorities, and to facilitate an increase in the number and quality of European clinical trials in AATD. 40 In addition, as part of EARCO's aim to include the patient's voice at the center of its objectives and activities, the collaboration will work closely with patient advisors via the European Lung Foundation (Figure 3). The steering committee includes two AATD patients, and patients and patients' representatives are welcome to the meetings and also participate in projects.…”

Section: Earco and The Earco Registrymentioning

confidence: 99%

“…The first project of EARCO is the foundation of a new pan-European AATD registry. 40 Since the discovery of AATD, national and international registries have been critical to increasing knowledge and understanding of the disease. For example, one of the earliest AATD registries in Sweden provided crucial insight into the prevalence of AATD through neonatal screening, 41 and has subsequently enabled assessment of health-status in these individuals through the first five decades of life.…”

Section: Earco and The Earco Registrymentioning

confidence: 99%

<p>New Patient-Centric Approaches to the Management of Alpha-1 Antitrypsin Deficiency</p>

Chorostowska‐Wynimko¹,

Barrecheguren²,

Ferrarotti³

et al. 2020

COPD

Self Cite

View full text Add to dashboard Cite

Alpha-1 antitrypsin deficiency (AATD) is a rare and underdiagnosed genetic predisposition for COPD and emphysema and other conditions, including liver disease. Although there have been improvements in terms of awareness of AATD and understanding of its treatment in recent years, current challenges center on optimizing detection and management of patients with AATD, and improving access to intravenous (IV) AAT therapythe only available pharmacological intervention that can slow disease progression. However, as an orphan disease with geographically dispersed patients, international cooperation is essential to address these issues. To achieve this, new European initiatives in the form of the European Reference Network for Rare Lung Diseases (ERN-LUNG) and the European Alpha-1 Research Collaboration (EARCO) have been established. These organizations are striving to address the current challenges in AATD, and provide a new platform for future research efforts in AATD. The first objectives of ERN-LUNG are to establish a quality control program for European AATD laboratories and create a disease management program for AATD, following the success of such programs in the United States. The main purpose of EARCO is to create a pan-European registry, with the aim of understanding the natural history of the disease and supporting the development of new treatment modalities in AATD and access to AAT therapy. Going further, other patient-centric initiatives involve improving the convenience of intravenous AAT therapy infusions through extended-interval dosing and self-administration. The present review will discuss the implementation of these initiatives and their potential contribution to the optimization of patient care in AATD.

show abstract

“…These have the aim of 1) promoting the exchange of research ideas among clinicians and affiliated scientists in Europe and/or globally; 2) building an infrastructure for prospective clinical research; 3) securing additional funding through national and European Union funding streams; and 4) facilitating the planning, implementation, evaluation and publication of clinical and translational studies at pan-European level and beyond. So far, there are currently 17 ongoing CRCs that cover eight major respiratory disease domains (airway diseases, interstitial lung diseases, pulmonary vascular diseases, sleep and breathing disorders, respiratory critical care, paediatric respiratory diseases, respiratory infections and thoracic oncology), all of them linked to one or more ERS assemblies [2][3][4][5][6][7][8][9][10][11][12]. CADSET, an acronym that stands for "Chronic Airway Diseases Early Stratification", is the latest addition to the list of ongoing CRCs (www.…”

Section: Introductionmentioning

confidence: 99%

Chronic Airway Diseases Early Stratification (CADSET): a new ERS Clinical Research Collaboration

et al. 2019

View full text Add to dashboard Cite

The European Alpha-1 Research Collaboration (EARCO): a new ERS Clinical Research Collaboration to promote research in alpha-1 antitrypsin deficiency

Cited by 34 publications

References 28 publications

α₁-Antitrypsin deficiency and chronic respiratory disorders

α₁-Antitrypsin deficiency and chronic respiratory disorders

<p>New Patient-Centric Approaches to the Management of Alpha-1 Antitrypsin Deficiency</p>

Chronic Airway Diseases Early Stratification (CADSET): a new ERS Clinical Research Collaboration

Contact Info

Product

Resources

About

The European Alpha-1 Research Collaboration (EARCO): a new ERS Clinical Research Collaboration to promote research in alpha-1 antitrypsin deficiency

Cited by 34 publications

References 28 publications

α1-Antitrypsin deficiency and chronic respiratory disorders

α1-Antitrypsin deficiency and chronic respiratory disorders

<p>New Patient-Centric Approaches to the Management of Alpha-1 Antitrypsin Deficiency</p>

Chronic Airway Diseases Early Stratification (CADSET): a new ERS Clinical Research Collaboration

Contact Info

Product

Resources

About

α₁-Antitrypsin deficiency and chronic respiratory disorders

α₁-Antitrypsin deficiency and chronic respiratory disorders