Current trial end-points | Problems identified | Possible solutions |
Exacerbations | Lack of definition Seasonal effects in short-term studies Variable patient reporting including underreporting | Validate EMBARC/BRR definition Study the impact of this on exacerbation reporting Patient diaries or symptom scales to capture nonreported exacerbations Develop an exacerbation prediction tool |
Microbiology | Unknown clinical benefit | Study the correlation of microbiome with clinical burden |
Sputum | Dependent on patient compliance Inter- and intra-day variation | Develop a scale indicating burden of expectoration rather than specific volumes, weights, etc. Alternative methods of measuring airway clearance, e.g. imaging |
Lung function | Multiple guidelines Patient and technician dependent Therapies are not designed to improve lung function Patients do not ask for improvement | Improve reporting and standardisation of methods Study the impact of lung function on quality of life Qualitative data gathering on what quality of life means to patients themselves Identify patient subgroups who show lung function improvements in response to therapies as in cystic fibrosis |
HRQoL (questionnaires) | Multiple variations Currently anchored to clinical markers with no validated significance in bronchiectasis patients Not shown to be responsive | Further validation required Further work may involve optimising existing questionnaires or developing new responsive scales Establish MCID specific for bronchiectasis |
Biomarkers | No direct clinical benefit | Remain as early phase end-point May be useful for selecting patients for entry into trials |
HRQoL: health-related quality of life; EMBARC: the European Bronchiectasis Registry; BRR: American Bronchiectasis Research Registry; MCID: minimal clinically important difference.