TABLE 2.
Summary data of the clinical visits before and after patients started on ivacaftor.
| Repeated measures of values at each visit model controls for age at baseline | Repeated measures of change from visit 1 (baseline); model controls for baseline value and age at baseline | ||||||
|---|---|---|---|---|---|---|---|
| Baseline | Post-6 | Post-12 | Post-24 | Post-6—Baseline | Post-12—Baseline | Post-24—Baseline | |
| Estimate (n) | Estimate (95%CL) p-value | ||||||
| 95%CL | |||||||
| Primary Outcome | |||||||
| mREE/kcal/day | 1466 (18) | 1500 (16) | 1513 (13) | 1576 (14) | 42 (−77; 160) | 43 (−79; 164) | 108 (−12; 228) |
| 1347; 1585 | 1378; 1623 | 1384; 1643 | 1449; 1703 | 0.5 | 0.5 | 0.076 | |
| mREE/kcal/day/kg | 30.3 (18) | 28.5 (16) | 27.6 (12) | 27.1 (13) | −1.8 (−3.7; 0.2) | −2.9 (−5.0;-0.8) | −3.1 (−5.2;-1.1) |
| 27.3; 33.2 | 25.5; 31.5 | 24.4; 30.7 | 23.9; 30.2 | 0.075 | 0.009 | 0.005 | |
| mREE, % predicted | 111.4 (18) | 110.3 (16) | 102.9 (12) | 104.7 (13) | −1.5 (−9.5; 6.6) | −8.3 (−17.7; 1.1) | −6.0 (−15.0; 3.1) |
| 102.2; 120.6 | 100.7; 120.0 | 91.9; 113.8 | 94.1; 115.3 | 0.7 | 0.080 | 0.18 | |
| Secondary Outcomes | |||||||
| Anthropometric Measures | |||||||
| BMI z-score | 0.50 (18) | 0.90 (18) | 0.88 (16) | 0.73 (15) | 0.40 (0.26; 0.54) | 0.39 (0.24; 0.54) | 0.24 (0.09; 0.40) |
| 0.05; 0.94 | 0.46; 1.34 | 0.43; 1.33 | 0.29; 1.18 | <.0001 | <.0001 | 0.003 | |
| Body Composition (fat) | |||||||
| Fat mass (kg) | 12.3 (13) | 16.5 (11) | 16.8 (11) | 18.3 (8) | 4.1 (1.7; 6.6) | 4.8 (2.4; 7.3) | 6.5 (4.0; 9.0) |
| 5.8; 18.8 | 10.0; 23.1 | 10.3; 23.3 | 11.7; 24.9 | 0.003 | 0.0007 | 0.0001 | |
| Fat mass (kg) a | 6.7 (10) | 11.6 (8) | 11.5 (8) | 13.7 (5) | 5.7 (−0.6; 12.1) | 6.3 (−0.1; 12.6) | 8.6 (2.2; 14.9) |
| −2.9; 16.4 | 1.9; 21.4 | 1.8; 21.3 | 3.8; 23.5 | 0.072 | 0.052 | 0.013 | |
| Fat % | 21.1 (13) | 25.0 (11) | 25.7 (11) | 26.4 (8) | 3.7 (1.5; 5.9) | 4.2 (2.0; 6.4) | 5.0 (2.6; 7.3) |
| 14.9; 27.4 | 18.7; 31.3 | 19.4; 32.0 | 20.1; 32.8 | 0.003 | 0.0008 | 0.0004 | |
| Fat % a | 15.1 (10) | 19.4 (8) | 19.6 (8) | 21.5 (5) | 4.7 (0.3; 9.0) | 4.9 (0.6; 9.1) | 6.9 (2.5; 11.2) |
| 6.4; 23.9 | 10.5; 28.2 | 10.8; 28.5 | 12.6; 30.4 | 0.037 | 0.030 | 0.0056 | |
| Body Composition (lean) | |||||||
| Fat free mass (kg) | 42.5 (13) | 43.2 (11) | 43.9 (11) | 45.0 (8) | 0.6 (−1.1; 2.3) | 1.2 (−0.4; 2.9) | 2.3 (0.5; 4.1) |
| 34.9; 50.1 | 35.5; 50.8 | 36.2; 51.5 | 37.3; 52.7 | 0.5 | 0.12 | 0.01 | |
| Fat free mass (kg) a | 50.6 (10) | 50.6 (8) | 50.8 (8) | 50.2 (5) | 0.0 (−1.3; 1.3) | 0.2 (−1.1; 1.6) | −0.4 (−2.0; 1.1) |
| 40.1; 61.2 | 40.0; 61.2 | 40.3; 61.4 | 39.6; 60.8 | >0.9 | 0.7 | 0.5 | |
a
Age at baseline >17 years, mREE-measured resting energy expenditure.