Potential solutions to problems found in the current end-points

Current trial end-pointsProblems identifiedPossible solutions
ExacerbationsLack 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
MicrobiologyUnknown clinical benefitStudy the correlation of microbiome with clinical burden
SputumDependent 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 functionMultiple 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
BiomarkersNo direct clinical benefitRemain 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.