Summary of results for key secondary efficacy endpoints
| Mannitol (n=233) | Control (n=228) | |
|---|---|---|
| Time to first exacerbation (days) | ||
| Patients with event (%) | 160 (68.7) | 178 (78.1) |
| Median (95% CI) | 165 (124, 204) | 124 (107, 143) |
| p Value (stratified Log-rank Test) | 0.0214 | |
| HR (95% CI) | 0.78 (0.63 to 0.96) | |
| p Value | 0.0218 | |
| Duration (days) of exacerbations* | ||
| Mean days with GPE (any type) per year (95% CI) | 31.49 (25.54 to 38.82) | 35.74 (28.90 to 44.20) |
| Rate ratio (95% CI) | 0.88 (0.67 to 1.16) | |
| p Value | 0.3602 | |
| Days on antibiotics for treatment of pulmonary exacerbations* | ||
| Mean days on antibiotics for treatment of GPE (any type) per year (95% CI) | 19.88 (16.12 to 24.51) | 26.03 (21.11 to 32.09) |
| Rate ratio (95% CI) | 0.76 (0.58 to 1.00) | |
| p Value | 0.0496 | |
| Absolute change in SGRQ total score from baseline† | ||
| n used in analysis | 228 | 219 |
| All on-treatment period | ||
| LS mean (95% CI) | −10.98 (−12.78 to −9.18) | −8.58 (−10.43 to −6.72) |
| Difference (95% CI) | −2.40 (−4.76 to −0.05) | |
| p Value | 0.0457 | |
Rate ratio is calculated using the negative binomial regression model for mannitol versus control. Model includes treatment, region and baseline pulmonary exacerbation rate as predictors.
HR, estimated from Cox regression, is for mannitol versus control.
Log-rank test and Cox regression are stratified by region and baseline PE rate (≤2/year, >2/year).
LS Mean: Least Squares Mean Difference, estimated from mixed model, is for mannitol versus control. Model includes treatment, visit, treatment×visit, region and baseline SGRQ Total score.
*Negative binomial regression.
†Mixed model analysis.
GPE, graded pulmonary exacerbation (any type); SGRQ, St George's Respiratory Questionnaire.