Table 2.
Clinical Outcomes and Change from Baseline by Visit, Stratified by Baseline Modulator Use
| Baseline Modulator Use* |
||||||
|---|---|---|---|---|---|---|
| Outcome | Visit | All | None | Iva | Two-Drug | |
| ppFEV1 | Baseline | n | 487 | 238 | 34 | 215 |
| Mean ± SD | 80.5 ± 22.7 | 80.2 ± 23.2 | 79.3 ± 20.8 | 81.1 ± 22.5 | ||
| 1 mo | n [n chg.]† | 412 [412] | 198 [198] | 32 [32] | 182 [182] | |
| Mean ± SD | 88.5 ± 22.6 | 88.5 ± 22.7 | 84.6 ± 24.1 | 89.1 ± 22.2 | ||
| Change | +8.80 | +10.10 | +5.55 | +7.96 | ||
| CI (chg.) | (8.01 to 9.59) | (8.93 to 11.26) | (3.24 to 7.86) | (6.78 to 9.13) | ||
| 3 mo | n [n chg.]† | 190 [190] | 94 [94] | 11 [11] | 85 [85] | |
| Mean ± SD | 87.6 ± 23.1 | 88.4 ± 22.8 | 74.7 ± 23.9 | 88.3 ± 23.2 | ||
| Change | +10.31 | +12.57 | +3.94 | +8.63 | ||
| CI (chg.) | (8.99 to 11.63) | (10.55 to 14.59) | (1.24 to 6.65) | (6.85 to 10.40) | ||
| 6 mo | n [n chg.]† | 356 [356] | 166 [166] | 24 [24] | 166 [166] | |
| Mean ± SD | 90.9 ± 21.5 | 91.0 ± 20.8 | 87.5 ± 21.9 | 91.3 ± 22.2 | ||
| Change | +9.76 | +10.84 | +6.14 | +9.21 | ||
| CI (chg.) | (8.76 to 10.76) | (9.32 to 12.35) | (3.34 to 8.94) | (7.76 to 10.66) | ||
| ppFVC | Baseline | n | 487 | 238 | 34 | 215 |
| Mean ± SD | 91.9 ± 18.2 | 92.1 ± 18.4 | 93.2 ± 19.0 | 91.4 ± 17.8 | ||
| 1 mo | n [n chg.]† | 412 [412] | 198 [198] | 32 [32] | 182 [182] | |
| Mean ± SD | 96.9 ± 16.3 | 97.3 ± 15.8 | 94.6 ± 18.3 | 96.9 ± 16.5 | ||
| Change | +5.95 | +6.94 | +2.52 | +5.48 | ||
| CI (chg.) | (5.21 to 6.69) | (5.82 to 8.06) | (0.50 to 4.54) | (4.40 to 6.56) | ||
| 3 mo | n [n chg.]† | 190 [190] | 94 [94] | 11 [11] | 85 [85] | |
| Mean ± SD | 96.9 ± 17.1 | 97.9 ± 16.8 | 91.0 ± 18.3 | 96.6 ± 17.4 | ||
| Change | +7.50 | +9.22 | +3.48 | +6.12 | ||
| CI (chg.) | (6.34 to 8.66) | (7.44 to 10.99) | (0.87 to 6.09) | (4.51 to 7.74) | ||
| 6 mo | n [n chg.]† | 356 [356] | 166 [166] | 24 [24] | 166 [166] | |
| Mean ± SD | 98.8 ± 16.1 | 99.2 ± 15.7 | 97.7 ± 19.2 | 98.5 ± 16.1 | ||
| Change | +6.93 | +7.60 | +3.85 | +6.71 | ||
| CI (chg.) | (6.03 to 7.83) | (6.20 to 9.00) | (1.27 to 6.44) | (5.43 to 7.99) | ||
| Sweat chloride, mmol/L | Baseline | n | 462 | 221 | 33 | 208 |
| Mean ± SD | 88.0 ± 18.4 | 95.6 ± 12.8 | 52.6 ± 23.0 | 85.6 ± 15.2 | ||
| 1 mo | n [n chg.]† | 399 [387] | 187 [180] | 30 [29] | 182 [178] | |
| Mean ± SD | 49.1 ± 19.6 | 57.3 ± 17.9 | 25.3 ± 15.0 | 44.5 ± 17.2 | ||
| Change | −38.95 | −39.15 | −25.31 | −40.97 | ||
| CI (chg.) | (−40.60 to −37.29) | (−41.68 to −36.62) | (−31.43 to −19.19) | (−43.17 to −38.76) | ||
| 6 mo | n [n chg.]† | 383 [369] | 174 [165] | 31 [30] | 178 [174] | |
| Mean ± SD | 45.7 ± 21.2 | 53.5 ± 19.6 | 26.2 ± 20.4 | 41.5 ± 19.5 | ||
| Change | −41.70 | −43.15 | −23.92 | −43.40 | ||
| CI (chg.) | (−43.80 to −39.60) | (−46.21 to −40.09) | (−31.03 to −16.81) | (−46.38 to −40.41) | ||
| CFQ-R RD | Baseline | n | 410 | 205 | 23 | 182 |
| Mean ± SD | 70.3 ± 18.2 | 69.1 ± 18.5 | 71.0 ± 18.9 | 71.4 ± 17.9 | ||
| 1 mo | n [n chg.]† | 380 [342] | 187 [170] | 28 [22] | 165 [150] | |
| Mean ± SD | 82.7 ± 15.0 | 82.2 ± 14.8 | 83.3 ± 17.8 | 83.3 ± 14.7 | ||
| Change | +12.54 | +12.81 | +10.35 | +12.55 | ||
| CI (chg.) | (10.32 to 14.76) | (9.53 to 16.09) | (0.52 to 20.19) | (9.37 to 15.74) | ||
| 3 mo | n [n chg.]† | 352 [319] | 166 [154] | 27 [21] | 159 [144] | |
| Mean ± SD | 87.5 ± 13.5 | 87.2 ± 13.2 | 87.9 ± 15.2 | 87.7 ± 13.7 | ||
| Change | +16.95 | +17.97 | +14.81 | +16.16 | ||
| CI (chg.) | (14.76 to 19.13) | (15.00 to 20.95) | (5.75 to 23.87) | (12.69 to 19.63) | ||
| 6 mo | n [n chg.]† | 302 [273] | 144 [137] | 25 [18] | 133 [118] | |
| Mean ± SD | 90.5 ± 11.3 | 90.1 ± 12.2 | 91.8 ± 11.4 | 90.7 ± 10.4 | ||
| Change | +20.39 | +22.51 | +18.52 | +18.22 | ||
| CI (chg.) | (18.28 to 22.50) | (19.47 to 25.54) | (7.63 to 29.40) | (15.22 to 21.22) | ||
| BMI‡, adults, kg/m2 | Baseline | n | 326 | 157 | 24 | 145 |
| Mean ± SD | 23.1 ± 4.0 | 22.8 ± 3.9 | 23.5 ± 2.7 | 23.5 ± 4.3 | ||
| 1 mo | n vis. [n chg.] | 272 [272] | 128 [128] | 22 [22] | 122 [122] | |
| Mean ± SD | 23.7 ± 4.2 | 23.4 ± 4.2 | 23.8 ± 3.1 | 24.0 ± 4.3 | ||
| Change | +0.42 | +0.47 | +0.39 | +0.37 | ||
| CI (chg.) | (0.33 to 0.50) | (0.34 to 0.59) | (0.11 to 0.68) | (0.25 to 0.49) | ||
| 3 mo | n vis. [n chg.] | 136 [136] | 65 [65] | 10 [10] | 61 [61] | |
| Mean ± SD | 24.1 ± 3.8 | 23.9 ± 3.8 | 23.5 ± 2.7 | 24.4 ± 3.9 | ||
| Change | +0.85 | +1.08 | +0.86 | +0.62 | ||
| CI (chg.) | (0.68 to 1.03) | (0.85 to 1.30) | (-0.05 to 1.76) | (0.34 to 0.90) | ||
| 6 mo | n vis. [n chg.] | 268 [268] | 121 [121] | 22 [22] | 125 [125] | |
| Mean ± SD | 24.5 ± 4.6 | 24.2 ± 4.6 | 24.6 ± 3.2 | 24.8 ± 4.7 | ||
| Change | +1.24 | +1.31 | +1.28 | +1.17 | ||
| CI (chg.) | (1.05 to 1.44) | (1.01 to 1.61) | (0.47 to 2.09) | (0.89 to 1.44) | ||
| BMI, peds§ (z-score) |
Baseline | n | 159 | 80 | 10 | 69 |
| Mean ± SD | 0.2 ± 0.9 | 0.1 ± 0.9 | 0.5 ± 0.7 | 0.2 ± 0.8 | ||
| 1 mo | n [n chg.]† | 140 [140] | 70 [70] | 10 [10] | 60 [60] | |
| Mean ± SD | 0.3 ± 0.8 | 0.2 ± 0.9 | 0.5 ± 0.7 | 0.3 ± 0.8 | ||
| Change | +0.11 | +0.11 | +0.05 | +0.12 | ||
| CI (chg.) | (0.07 to 0.15) | (0.05 to 0.18) | (-0.07 to 0.18) | (0.08 to 0.16) | ||
| 3 mo | n [n chg.]† | 55 [55] | 28 [28] | 2 [2] | 25 [25] | |
| Mean ± SD | 0.3 ± 0.7 | 0.0 ± 0.8 | −0.5 ± 0.4 | 0.6 ± 0.5 | ||
| Change | +0.24 | +0.28 | +0.14 | +0.20 | ||
| CI (chg.) | (0.15 to 0.33) | (0.12 to 0.45) | (−1.92 to 2.19) | (0.11 to 0.29) | ||
| 6 mo | n [n chg.]† | 139 [139] | 67 [67] | 10 [10] | 62 [62] | |
| Mean ± SD | 0.5 ± 0.8 | 0.5 ± 0.8 | 0.7 ± 0.9 | 0.4 ± 0.8 | ||
| Change | +0.30 | +0.37 | +0.20 | +0.23 | ||
| CI (chg.) | (0.22 to 0.37) | (0.24 to 0.50) | (−0.06 to 0.45) | (0.14 to 0.31) | ||
Definition of abbreviations: BMI = body mass index; CFQ-R RD = Cystic Fibrosis Questionnaire–Revised, Respiratory Domain; chg. = change; CI = confidence interval; Iva = ivacaftor monotherapy; n vis. = number of participants attending the study visit; ppFEV1 = percent predicted FEV1; ppFVC = percent predicted FVC; two-drug = lumacaftor/iva or tezacaftor/iva.
*
Prior use of modulators defined as any use within 90 days of the baseline measurement.
†
[n chg.] is the number of participants contributing to the change estimate (having both baseline and follow-up measured).
‡
Pregnant participants were excluded from analyses of BMI.
§
z-scores are based on Centers for Disease Control and Prevention percentiles.