TY - JOUR T1 - Research priorities in α<sub>1</sub>-antitrypsin deficiency: results of a patients' and healthcare providers' international survey from the EARCO Clinical Research Collaboration JF - ERJ Open Research JO - erjor DO - 10.1183/23120541.00523-2020 VL - 6 IS - 4 SP - 00523-2020 AU - Miriam Barrecheguren AU - Karen O'Hara AU - Marion Wilkens AU - Jeanette Boyd AU - Ewa Kolda AU - Beatriz Lara AU - Joanna Chorostowska-Wynimko AU - Ilaria Ferrarotti AU - Jan Chlumský AU - Christian Clarenbach AU - Timm Greulich AU - Marc Miravitlles AU - Maria Sucena A2 - , Y1 - 2020/10/01 UR - http://openres.ersjournals.com/content/6/4/00523-2020.abstract N2 - α1-antitrypsin deficiency (AATD) is a rare and under-recognised genetic condition. Owing to its low prevalence, international initiatives are key for conducting high-quality research in the field.From July 2018 to December 2019, the European Alpha-1 Research Collaboration (EARCO) developed and conducted two surveys, one for healthcare providers and one for patients and caregivers, aiming to identify research priorities and barriers in access to treatment for AATD.A survey on 164 research questions was electronically sent to 230 AATD experts in Europe, and 94 completed surveys from 24 countries were received. The top research areas identified by healthcare providers were causes of variable progression and poor outcomes, improvement in diagnosis, initiation and optimal dosing of augmentation therapy and effectiveness of self-management interventions. During the same period, 438 surveys were completed by patients and caregivers from 26 countries. The top research areas identified were improving knowledge about AATD, in particular among general practitioners, access to AATD specialised centres and access to reliable, easy to understand information about living with AATD. Regarding barriers to treatment, participants from countries where augmentation therapy was reimbursed prioritised improving knowledge in AATD, while respondents in non-reimbursed countries regarded access to AATD augmentation therapy and to specialised centres as the most relevant.The main research and management priorities identified by healthcare providers and patients included understanding the natural history of AATD, improving information to physicians, improving access to specialised reference centres, personalising treatment and having equal opportunities for access to existing therapies.Surveys for patients and healthcare providers on research priorities in AATD, developed by the @EuroRespSoc CRC EARCO, show that international collaboration and engagement with patients are key development strategies needed for improvement https://bit.ly/2ItIzaW ER -