Table 1.
Comparison of change in all variables over the treatment period.
| Characteristics | Baseline | After 4 Weeks | Baseline | After 4 Weeks | Baseline | After 4 Weeks |
|---|---|---|---|---|---|---|
| Subject 1 ° | Subject 2 ° | Subject 3 ° | ||||
| Sweat chloride (mEq/L) | 51 | 13 | 104 | 64 | 106 | 101 |
| FEV1 (%) | 80 | 87 | 61 | 70 | 91 | 81 |
| BMI (kg/m2) | 22.40 | 23.25 | 18.65 × | 18.66 | 14.11 ~ | 13.75 |
| 6MWT (m) | 630 | 657 | 551 | 570 | 550 | 580 |
| LCI2.5 ∧ | n.a | n.a | 7.23 | 6.69 | 6.54 | 6.43 |
| CFQ-R 14+ domain * | ||||||
| Physical functioning | 58.3 | 91.7 | 50.0 | 77.8 | 72.2 | 55.6 |
| Role perception | 50.0 | 75.0 | / | / | / | / |
| Vitality | 53.3 | 73.3 | / | / | / | / |
| Emotion | 88.9 | 100.0 | 83.3 | 87.5 | 79.2 | 79.2 |
| Social perception | 22.2 | 33.3 | 76.2 | 81.0 | 47.6 | 47.6 |
| Body image | 33.3 | 55.6 | 100.0 | 100.0 | 100.0 | 100.0 |
| Eating disturbance | 44.4 | 44.4 | 77.8 | 100.0 | 0.0 | 0.0 |
| Treatment burden | 55.6 | 66.7 | 22.2 | 44.4 | 55.6 | 55.6 |
| Health perception | 41.7 | 58.3 | / | / | / | / |
| Weight | 100.0 | 100.0 | / | / | / | / |
| Respiratory symptoms | 72.2 | 94.4 | 83.3 | 100.0 | 50.0 | 50.0 |
| Digestive symptoms | 55.6 | 77.8 | 66.7 | 100.0 | 66.7 | 66.7 |
° Patients took TEZ-IVA (subject 1), ELX/TEZ/IVA (subject 2), or LUM/IVA (subject 3) therapies; ∧ the predicted value for patients 2 and 3 was 6.55; × 65th percentile for age; ~ 11the percentile for age; * based on patient age (adult, 12–13 years, and 6–11 years, respectively). Abbreviations: FEV1: forced expiratory volume in the 1st second; BMI: body mass index; 6MWT: six-minute walk test; LCI: lung clearance index; CFQ-R: CF Questionnaire-Revised; n.a: not available.