Baseline characteristics of patients enrolled in the EARCO prospective registry of alpha-1 antitrypsin deficiency: preliminary results

Le résumé fourni par la source

The European Alpha-1 antitrypsin deficiency Research Collaboration (EARCO) is a Clinical Research Collaboration (CRC) of the ERS with the main objective to develop a prospective registry of individuals with the deficiency to understand the natural history of the disease and the impact of therapies better. We aim to describe the baseline characteristics of patients enrolled during the first year of the registry. The registry (http://earco.estudinord.com) was launched on February 2020 and consists of baseline and yearly follow-up data. By February 2021 there were 34 centers from 10 countries ready to enroll and the data base included information from 235 patients from 5 countries. Phenotypes were: 110 Pi*ZZ, 79 Pi*SZ, 16 Pi*SS and 30 others. Globally, 54.5% were male, 75% index cases, with a mean age of 54.3 years (SD:14.8), 66% with lung and 9.8% with liver disease. Mean FEV1= 78% pred. (SD:32.4%) and KCO 76% (SD:22%), with a mean CAT of 11 (SD:8) and BODEx index of 1.3 (SD:1.8). Among the 110 Pi*ZZ individuals, the mean age was 56.1 years (SD: 12.7), 90% with lung and 15% with liver disease; mean FEV1= 65.4% (SD:31.6%) and KCO 69% (SD:20%) with a mean CAT of 13.6 (SD:8) and BODEx index of 2 (SD: 2), 66 (61%) of Pi*ZZ patients and 6 patients with other genotypes were on augmentation therapy. Despite the difficulties with the different interpretations of data protection laws across countries and the slow recruitment due to the COVID-19 pandemic, the EARCO group has been able to engage a large number of centers from 10 countries and demonstrated to be a valid platform to collect standardized prospective data from patients with AATD.

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