Efficacy
CAYSTON improved respiratory symptoms, such as coughing, wheezing, and sputum production as measured by CFQ-R* score
- The CAYSTON Prescribing Information does not present quantitative data on changes in CFQ-R
- The FDA determined the CFQ-R was not validated in accordance with its Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims4
- CFQ-R score has not been shown to correlate directly with a change in FEV14
- Improvement in respiratory symptoms was noted for CAYSTON-treated patients vs placebo on the last day of drug treatment (9.7 points; 95% CI; 4.3, 15.1; P < 0.001)1
- Statistically significant improvements were seen in adults and pediatric patients, but were substantially smaller in adults
- A difference in respiratory symptoms between treatment groups was present 2 weeks after completion of treatment, although the difference was smaller (6.3 points; 95% CI: 1.2, 11.4; P = 0.015)1
*Cystic Fibrosis Questionnaire-Revised.
AIR-CF1: FOREST PLOT
Respiratory Symptoms Assessed in All Patients By Disease Severity and Age1
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 1/4:
Show Observed Data
How do you think patients with moderate lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 1/4:
Show Observed Data
How do you think patients with moderate lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 2/4:
Show Observed Data
How do you think patients with severe lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 2/4:
Show Observed Data
How do you think patients with severe lung disease taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 3/4:
Show Observed Data
How do you think patients younger than 18 years and taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 3/4:
Show Observed Data
How do you think patients younger than 18 years and taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 4/4:
Show Observed Data
How do you think patients 18 years and older taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Question 4/4:
Show Observed Data
How do you think patients 18 years and older taking CAYSTON rated their respiratory symptom improvement? (Use the + and - buttons to select a range.)
Mean Change From Baseline CFQ-R* Respiratory Scores (±SE) at Day 28
Placebo
CAYSTON
Figure is adapted from Retsch-Bogart et al 2009.1
Statistically significant improvements were seen in both adult and pediatric patients, but were substantially smaller in adult patients. Two weeks after completion of treatment, a difference in respiratory symptoms between treatment groups was still present, though the difference was smaller.
Disclaimer: These questions are from the CFQ-R version for patients 14 years and older; younger children used a different questionnaire or a parent questionnaire was used. Minimum clinically important difference was not established for the child/parent questionnaire.
*Inferences regarding the treatment effect and size of the treatment effect should not be made based on the Respiratory Symptoms Scale of the CFQ-R.
†Moderate disease: FEV1 >50% to ≤75% predicted; measured at screening.
‡Severe disease: FEV1 ≥25% to ≤50% predicted; measured at screening.
AIR-CF1: LUNG FUNCTION
CAYSTON SIGNIFICANTLY IMPROVED FEV1 LUNG FUNCTION
- Mean percent change in FEV1 (L) was a prespecified secondary endpoint in AIR-CF1.1 Click here to review the study design
- CAYSTON demonstrated a significant improvement in FEV1 (L) lung function with a placebo-adjusted treatment effect of 10% (95% CI: 6%, 14%) on Day 28
- Improvements in FEV1 were comparable between adult and pediatric patients1
“When I'm on CAYSTON, I notice a positive difference in my coughing and see a boost in my lung function.”
–Kevin, age 43, TREATED WITH CAYSTON
Individual results may vary.
AIR-CF1: DISEASE SEVERITY
AT DAY 28, 10% PLACEBO-ADJUSTED TREATMENT EFFECT IN FEV1 (L) WAS SEEN IN PATIENTS REGARDLESS OF BASELINE LUNG FUNCTION5
- At Day 28, there was a treatment effect of 10.3% (95% CI: 3%, 18%; P = 0.0061) in patients with FEV1 ≥25% to ≤50%
- At Day 28, there was a treatment effect of 10.1% (95% CI: 5%, 15%; P < 0.0001) in patients with FEV1 >50% to ≤75%
- Two weeks after completion of drug treatment, the difference in FEV1 between CAYSTON and placebo groups continued but decreased to 6% (95% CI: 2%, 9%)
AIR-CF1: ADULT/PEDIATRIC
IMPROVEMENTS IN FEV1 WERE COMPARABLE ACROSS ADULT AND PEDIATRIC PATIENTS ON DAY 28 OF TREATMENT5
- At Day 28, there was a treatment effect of 10.5% (95% CI: 6.4%,14.7%; P < 0.0001) in patients ≥18 years
- At Day 28, there was a treatment effect of 10.4% (95% CI: -1.3%, 22.1%; P = 0.0790) in patients <18 years
- Two weeks after completion of drug treatment, the difference in FEV1 between CAYSTON and placebo groups continued but decreased to 6% (95% CI: 2%, 9%)
