US Patient-Centered Research Priorities and Roadmap for Bronchiectasis

[摘要] Noncystic fibrosis bronchiectasis (bronchiectasis) is an increasingly common chronic lung disease that is difficult to manage because of a lack of evidence on which to base treatment decision-making. We sought to develop a practical list of US-based patient-centered research priorities and an associated roadmap to guide bronchiectasis research. We designed and administered a web-based patient needs assessment survey to establish broad research priorities, convened three stakeholder webinars to confirm the top priorities, obtained written stakeholder feedback, and completed a final consensus survey of objectives. The stakeholder panel consisted of clinical research experts in bronchiectasis, a seven-member patient advisory panel, and representatives from the two key patient advocacy organizations: COPD Foundation and NTM Info and Research Inc. Based on survey results from 459 patients with bronchiectasis, the stakeholder panel identified 27 patient-centered research priorities for bronchiectasis in the areas of bronchiectasis treatment and prevention of exacerbations, improving treatment of exacerbations and infections, improving health-related quality of life, predictors of poor prognosis, understanding the impact of underlying conditions, and conducting patient-centered clinical trials. These priorities should further inform the development and evaluation of both new and previously unproven therapies, with particular attention to the inclusion of patient-reported outcomes. We anticipate a great deal of progress will be made in the field of bronchiectasis in the next decade.

[学科分类] 呼吸医学

[关键词] bronchiectasis;disease progression;drug therapy;health-related quality of life;infection;BSI;Bronchiectasis Severity Index;CF;cystic fibrosis;EMBARC;European Multicentre Bronchiectasis Audit and Research Collaboration;HRQoL;health-related quality of life;NTM;nontuberculous mycobacteria;NTMir;NTM Info and Research Inc;QOL-B;Quality of Life-Bronchiectasis instrument