Table 1 Outcome data for BEAT-CF participants

Demographic data

Date of birth

Y

Age is independently associated with baseline lung function in CF. There may be “cohort” effects whereby those born in more recent periods have better outcomes because of improvements in care

Sex

Y

Sex is independently associated with lung function in CF

Clinical

Genotype

Y

Validation of CF diagnosis

Sweat test results

Y

Validation of CF diagnosis

Pancreatic exocrine status

Y

Y

Pancreatic function is independently associated with lung function in CF

CF-related diabetes

Y

Y

Diabetes is independently associated with lung function in CF

Known drug allergies or contraindications

Y

Y

Drug allergies and contraindications constrain therapeutic options, and plausibly confounds or modifies treatment effects

Concomitant medications

Y

Y

Use of concomitant medications plausibly confounds or modifies treatment effects, including toxicities

Laboratory

Total IgE

Y^a

Y

Marker for allergic bronchopulmonary aspergillosis which is a cause of clinical deterioration and may confound PEx management

Aspergillus-specific RAST

Y^a

Y

Marker for allergic bronchopulmonary aspergillosis which is a cause of clinical deterioration and may confound PEx management

Date and details of any new, documented infection, or airway colonization

Y^a

Y

The presence of specific pathogens may influence therapeutic choices, plausibly confounds or modifies treatment effects. Selection of resistant pathogens may be an untoward outcome of antibiotic therapy

Date and details of any new, documented infection with Clostridium difficile

Y^a

Y

Clostridium difficile-related diarrhea is an important untoward outcome of antibiotic therapy

Patient-reported outcomes:

Cystic Fibrosis Questionnaire-revised (CFQ-R) for all participants ≥6 years old

Y

Y

Treatment of PEx plausibly impacts on quality-of-life (QoL) over time

Anthropometric

Height

Y

Y

Height is associated with FEV₁ and is essential for estimating the ppFEV₁

Weight

Y

Y

Treatment of PEx plausibly impacts on weight over time

Spirometry

FEV₁

Y^b

Y

Treatment of PEx plausibly impacts on lung function. The ppFEV₁, is strongly associated with morbidity and mortality in CF