Table 2.
Efficacy Results at Week 24.*
| Result | TRAFFIC | TRANSPORT | Pooled | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Placebo (N = 184) | LUM (600 mg/ day)–IVA (N = 183) | LUM (400 mg every 12 hr)–IVA (N = 182) | Placebo (N = 187) | LUM (600 mg/ day)–IVA (N = 185) | LUM (400 mg every 12 hr)–IVA (N = 187) | Placebo (N = 371) | LUM (600 mg/ day)–IVA (N = 368) | LUM (400 mg every 12 hr)–IVA (N = 369) | |
| Change in percentage of predicted FEV1 from baseline† | |||||||||
|
| |||||||||
| Difference vs. placebo in the absolute change — percentage points | |||||||||
|
| |||||||||
| Mean (95% CI) | — | 4.0 (2.6 to 5.4)‡ | 2.6 (1.2 to 4.0)‡ | — | 2.6 (1.2 to 4.1)‡ | 3.0 (1.6 to 4.4)‡ | — | 3.3 (2.3 to 4.3)‡ | 2.8 (1.8 to 3.8)‡ |
|
| |||||||||
| P value | <0.001 | <0.001 | <0.001 | <0.001 | <0.001 | <0.001 | |||
|
| |||||||||
| Difference vs. placebo in the relative change — % | |||||||||
|
| |||||||||
| Mean (95% CI) | — | 6.7 (4.3 to 9.2)‡ | 4.3 (1.9 to 6.8)‡ | — | 4.4 (1.9 to 7.0)‡ | 5.3 (2.7 to 7.8)‡ | — | 5.6 (3.8 to 7.3)‡ | 4.8 (3.0 to 6.6)‡ |
|
| |||||||||
| P value | <0.001 | <0.001 | <0.001 | <0.001 | <0.001 | <0.001 | |||
|
| |||||||||
| Difference vs. placebo in absolute change from baseline in BMI | |||||||||
|
| |||||||||
| Mean (95% CI) | — | 0.16 (−0.04 to 0.35) | 0.13 (−0.07 to 0.32) | — | 0.41 (0.23 to 0.59)‡ | 0.36 (0.17 to 0.54)‡ | — | 0.28 (0.15 to 0.41)‡ | 0.24 (0.11 to 0.37)‡ |
|
| |||||||||
| P value | 0.11 | 0.19 | <0.001 | <0.001 | <0.001 | <0.001 | |||
|
| |||||||||
| Difference vs. placebo in absolute change from baseline in CFQ-R respiratory domain | |||||||||
|
| |||||||||
| Mean (95% CI) — points | — | 3.9 (0.7 to 7.1) | 1.5 (−1.7 to 4.7) | — | 2.2 (−0.9 to 5.3) | 2.9 (−0.3 to 6.0) | — | 3.1 (0.8 to 5.3)‡ | 2.2 (0.0 to 4.5) |
|
| |||||||||
| P value | 0.02 | 0.36 | 0.17 | 0.07 | 0.007 | 0.05 | |||
|
| |||||||||
| Odds ratio for a relative increase of ≥5% from baseline in the percentage of predicted FEV1 | |||||||||
|
| |||||||||
| Odds ratio (95% CI) | — | 2.9 (1.9 to 4.6) | 2.1 (1.3 to 3.3) | — | 3.0 (1.9 to 4.6) | 2.4 (1.5 to 3.7) | — | 2.9 (2.1 to 4.0)‡ | 2.2 (1.6 to 3.1)‡ |
|
| |||||||||
| P value | <0.001 | 0.002 | <0.001 | <0.001 | <0.001 | <0.001 | |||
|
| |||||||||
| Pulmonary exacerbations§ | |||||||||
|
| |||||||||
| Events — no. (rate per 48 wk) | 112 (1.07) | 79 (0.77) | 73 (0.71) | 139 (1.18) | 94 (0.82) | 79 (0.67) | 251 (1.14) | 173 (0.80) | 152 (0.70) |
|
| |||||||||
| Rate ratio (95% CI) | — | 0.72 (0.52 to 1.00) | 0.66 (0.47 to 0.93) | — | 0.69 (0.52 to 0.92) | 0.57 (0.42 to 0.76) | — | 0.70 (0.56 to 0.87)‡ | 0.61 (0.49 to 0.76)‡ |
|
| |||||||||
| P value for the rate ratio | 0.05 | 0.02 | 0.01 | <0.001 | 0.001 | <0.001 | |||
*
Reported means are least-squares means. For individual studies, within each active-treatment group and between the active-treatment groups and the placebo group, a hierarchical testing procedure was performed to control for multiplicity across primary and key secondary end points; P≤0.0250 in the current test and all previous tests was required to claim significance in the hierarchy. CFQ-R denotes Cystic Fibrosis Questionnaire–Revised.
†
Changes in the percentage of predicted FEV1 are calculated by averaging the means at weeks 16 and 24.
‡
The difference versus placebo was significant.
§
The number of pulmonary exacerbations was reported through week 24 and is expressed as a rate over 48 weeks.