Table 3:
Efficacy results after treatment with LUM/IVA for 24 weeks in patients with ppFEV1 <70 vs ≥70 at screening
| Parameter | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/IVA 250 mg q12h | |||
|---|---|---|---|---|---|---|
| ppFEV1 at screening* | ||||||
| <70 (n=244) |
≥70 (n=109) |
<70 (n=241) |
≥70 (n=119) |
<70 (n=245) |
≥70 (n=114) |
|
| Absolute change in ppFEV1 | ||||||
| Within group LS mean (SE) | −0∙5 (0∙4) | 0∙1 (0∙8) | – | – | – | – |
| LS mean vs placebo (95% CI), percentage points† | – | – | 3∙3 (2∙1–4∙4) | 3∙3 (1∙3–5∙4) | 3∙3 (2∙1–4∙4) | 1∙9 (−0∙2–4∙0) |
| p value | – | – | <0∙001 | 0∙002 | <0∙001 | 0∙079 |
| Relative change in ppFEV1 | ||||||
| Within group LS mean (SE) | −0∙3 (0∙9) | 0∙7 (1∙1) | – | – | – | – |
| LS mean vs placebo (95% CI), %† | – | – | 6∙0 (3∙7–8∙2) | 4∙4 (1∙5–7∙4) | 5∙9 (3∙6–8∙2) | 2∙5 (−0∙5–5∙5) |
| p value | – | – | <0∙001 | 0∙003 | <0∙001 | 0∙103 |
| Relative increase of ≥5% from baseline in ppFEV1‡ | ||||||
| Odds ratio vs placebo (95% CI) | – | – | 2∙5 (1∙7–3∙7) | 3∙8 (2∙1–6∙8) | 2∙4 (1∙6–3∙5) | 1∙9 (1∙0–3∙4) |
| p value | – | – | <0∙001 | <0∙001 | <0∙001 | 0∙045 |
| Body mass index | ||||||
| Within group LS mean (SE) | 0∙1 (0∙1) | 0∙1 (0∙1) | – | – | – | – |
| LS mean vs placebo (95% CI), kg/m2 | – | – | 0∙2 (0∙0–0∙4) | 0∙4 (0∙2–0∙7) | 0∙2 (0∙0–0∙3) | 0∙3 (0∙1–0∙6) |
| p value | – | – | 0∙017 | <0∙001 | 0∙041 | 0∙006 |
| CFQ-R respiratory domain | ||||||
| Within group LS mean (SE) | 1∙5 (1∙1) | 1∙7 (1∙4) | – | – | – | – |
| LS mean vs placebo (95% CI), points | – | – | 4∙1 (1∙3–6∙9) | 1∙9 (−1∙9–5∙7) | 1∙9 (−0∙9–4∙7) | 3∙6 (−0∙3–7∙4) |
| p value | – | – | 0∙005 | 0∙326 | 0∙184 | 0∙071 |
*
Eighty-one patients had ppFEV1 that decreased to <40 between screening and baseline.
†
Assessed by averaging the mean values from weeks 16 and 24, according to the prespecified statistical analysis plan.
‡
Average relative increase from baseline at weeks 16 and 24.
CFQ-R=Cystic Fibrosis Questionnaire-Revised; CI=confidence interval; IVA=ivacaftor; LUM=lumacaftor; LS=least squares; ppFEV1=percent predicted forced expiratory volume in 1 second; SE=standard error; q12h=every 12 hours; qd=every day.