Table 1.
Demographic data and clinical outcome parameters of the ETI group from baseline to initiation of ETI and at follow-up and of the non-ETI group from baseline to follow-up.
| ETI group (n = 21) | non-ETI group (n = 6) | Between-group comparison p-value | Within-group comparison p-value | ||||||
|---|---|---|---|---|---|---|---|---|---|
| Baseline | Start of ETI | Follow-up | Baseline | Follow-up | Baseline | Follow-up | ETI group | non-ETI group | |
| Male, n (%) | 14 (67) | 4 (67) | |||||||
| del F508 homozygous, n (%) | 13 (62) | 4 (67) | |||||||
| del F508 heterozygous (in %) | 6 (28) | 1 (17) | |||||||
| Pancreatic insufficiency, n (%) | 17 (80) | 6 (100) | |||||||
| Pseudomonas aeruginosa, n (%) | 11 (52) | 2 (33) | |||||||
| Time between clinical assessments (years) | 5.4 ± 0.8 (5.0/5.7) | 6.5 ± 0.5 (6.1/7.0) | 0.001 | ||||||
| Duration of ETI therapy (weeks) | 33 ± 25 (21.0/44.0) | ||||||||
| Age (years) | 25.9 ± 7.4 (22.5/29.2) | 31.0 ± 7.6 (27.5/34.4)††† | 24.0 ± 7.9 (15.7/32.3) | 30.3 ± 7.7 (22.2/38.4)† | 0.316 | 0.860 | < 0.001 | 0.023 | |
| Height (cm) | 171.2 ± 8.8 (167.0/175.2) | 175.0 ± 12.0 (162.4/187.6) | – | ||||||
| Weight (kg) | 58.7 ± 11.6 (53.7/64.0) | 59.4 ± 11.4 (53.7/65.0)### | 67.1 ± 11.9 (61.7/72.6)††† | 60.0 ± 7.2 (54.0, 66.0) | 62.3 ± 7.3 (56.2/68.4) | 0.484 | .502 | 0.001 | 0.150 |
| BMI (kg/m2) | 19.9 ± 2.8 (18.8 21.2) | 20.5 ± 3.0 (19.0/22.0)### | 22.8 ± 2.6 (21.6/23.9)††† | 19.7 ± 1.2 (18.7/20.7) | 20.5 ± 1.7 (19.1/22.0) | 0.521 | 0.122 | < 0.001 | 0.075 |
| ppFEV1 | 51.9 ± 16.5 (44.4/59.5) | 43.0 ± 16.1 (35.0, 51.6)**,### | 53.1 ± 17.9 (45.0/61.3) | 68.5 ± 21.6 (60.0/100.7) | 63.0 ± 26.9 (41.6/88.7) | 0.004 | 0.381 | < 0.001 | 0.123 |
| FEV1 z-score | −3.92 ± 1.3 (−4.5/−3.4) | −4.54 ± 1.2 (−5.12/−3.97)*,### | −3.0 ± 1.9 (−3.9/−2.1)† | −1.89 ± 1.75 (−3.73/−0.6) | −2.70 ± 1.68 (−4.46/0.94) | 0.012 | 0.771 | < 0.001 | 0.401 |
Values are number of patients (%) or mean ± standard deviation (95% confidence interval). BMI, body mass index; ETI, elexacaftor/tezacaftor/ivacaftor; ppFEV1, percent predicted forced expiratory volume in 1 s. Baseline, assessment of habitual physical activity; clinical assessment and testing; Follow-up, clinical assessment and testing between groups. Statistical testing: Kruskal–Wallis test (between groups); Mann–Whitney U-test (within groups); Friedman two-way analysis of variance by ranks test in the ETI group to compare the changes over time; Chi-Quadrat (2), χ² (post hoc tests).
Baseline vs. initiation of ETI: p < 0.05.
Baseline vs. initiation of ETI: p < 0.01.
Initiation of ETI vs. follow-up: p < 0.05.
Initiation of ETI vs. follow-up: p < 0.001.
Baseline vs. follow-up: p < 0.05.
Baseline vs. follow-up: p < 0.001.
Bold type indicates a significant result.