European Registry of Patients with Alpha-1 Antitrypsin Deficiency EARCO Enrolls Patients
Insufficient identification of patients with alpha-1 antitrypsin deficiency (AATD) and a lack of knowledge about this disease have led to the launch of the European Registry EARCO, which is currently enrolling patients. The aim is to better describe the pathophysiology of the disease and the prognosis of these patients, and to create a validated prognostic tool to aid clinical decision-making.
Reasons for Creating the Registry
Alpha-1 antitrypsin deficiency is a genetic disease that often remains undiagnosed. It affects approximately 1 in 2000–5000 Europeans and predisposes to the early development of emphysema and kidney disease. Despite ongoing research, unresolved questions remain about the natural history of this disease, its pathophysiology, genetics, and patient prognosis. Therefore, a pan-European registry called the European Alpha-1 Clinical Research Collaboration (EARCO) was established, bringing together scientists from European countries to create a standardized database.
Registry Characteristics
This is a non-interventional multicenter pan-European longitudinal observational cohort study, which prospectively includes patients of all age groups and disease severity with AATD. AATD is defined as a serum level of alpha-1 antitrypsin (AAT) < 11 µM or genotype ZZ, SZ, or other rare variants of the AAT gene associated with its deficiency.
The registry is open to all physicians treating patients with AATD. Patients in the registry are treated according to standard procedures without any intervention within this observational study. However, they must provide written informed consent for prospective data recording.
Monitored Parameters
Upon entry into the registry and then annually, sociodemographic data, results of pulmonary function tests (spirometry, whole-body plethysmography, and lung diffusion capacity), exercise capacity testing, blood lab tests, and liver ultrasound are recorded, along with comorbidities, exacerbations of chronic obstructive pulmonary disease (COPD), treatment information, and patient-reported data (symptoms, quality of life, and physical activity via questionnaires). To ensure proper data entry, specific investigator physicians and a clinical study participant record form are assigned. All entered data will be verified.
Project Goals
- To create a pan-European AATD registry.
- To standardize the process of data recording and ensure high-quality longitudinal data.
- To better understand the natural history and prognosis of AATD and create a prognostic tool for clinical decision-making.
- To evaluate the impact of AAT augmentation and other treatments on emphysema progression, lung function, clinical outcomes, quality of life, and mortality in real-world practice.
- To gain more insights into severe AATD with non-ZZ genotypes.
Conclusion
By the end of the third year of the registry's existence, it is expected to include > 3000 patients from more than 20 European countries, including the Czech Republic, which is already involved in the project.
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Source: Greulich T., Altraja A., Barrecheguren M. et al. Protocol for the EARCO Registry: a pan-European observational study in patients with α1-antitrypsin deficiency. ERJ Open Res 2020 Mar 2; 6 (1): 00181-2019, doi: 10.1183/23120541.00181-2019.
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