Table 1.
Demographic and clinical characteristics
| Characteristic | Overall (N = 209) |
G551D/IVA patient group (n = 72) |
F508del/SOC patient group (n = 137) |
P valuea |
|---|---|---|---|---|
| Age, mean (SD), years | 24.3 (12.1) | 23.9 (13.9) | 24.6 (11.1) | 0.70 |
| Range | 6–62 | 6–62 | 6–52 | |
| Missing, n | 6 | 3 | 3 | |
| Age group (years), n (%) | ||||
| 6–11 | 33 (15.8) | 12 (16.7) | 21 (15.3) | |
| 12–17 | 39 (18.7) | 18 (25.0) | 21 (15.3) | 0.19 |
| ≥18 | 137 (65.6) | 42 (58.3) | 95 (69.3) | |
| Sex, n (%)b | ||||
| Male | 108 (56.0) | 27 (39.7) | 81 (64.8) | < 0.01 |
| Female | 85 (44.0) | 41 (60.3) | 44 (35.2) | |
| Missing, n | 16 | 3 | 12 | |
| ppFEV1 (%), mean (SD) | 73.9c (28.0) | 79.8 (25.6) | 70.7d (28.8) | < 0.05 |
| ppFEV1 <70, n (%) | 85 (40.7)e | 24 (33.3) | 61 (44.5)e | 0.12 |
| ppFEV1 (%), n; mean (SD) | ||||
| <40 | 32; 29.3 (6.6) | 6; 28.8 (4.5) | 26; 29.3 (7.0) | 0.87 |
| 40–69 | 51; 54.5 (8.7) | 18; 57.4 (7.9) | 33; 52.9 (8.7) | 0.07 |
| 70–89 | 50; 79.2 (6.0) | 16; 78.6 (6.6) | 33; 79.5 (5.7) | 0.60 |
| ≥90 | 74; 103.0 (10.5) | 31; 103.4 (9.7) | 43; 102.6 (11.1) | 0.74 |
| Number of comorbidities, mean (SD) | 1.9f (1.2) | 1.5g (1.1) | 2.0 (1.2) | < 0.01 |
| Patients with ≥1 comorbidity, n (%) | 192 (92.3) | 60 (84.5) | 132 (96.4) | < 0.01 |
| History of lung transplant, n (%) | ||||
| Yes | 3f (1.4) | 0g (0.0) | 3 (2.2) | 0.55 |
| No | 205f (98.6) | 71g (100.0) | 134 (97.8) | |
| Waiting for lung transplant, n (%) | ||||
| Yes | 3f (1.4) | 0g (0.0) | 3 (2.2) | 0.55 |
| No | 205f (98.6) | 71g (100.0) | 134 (97.8) | |
| BMI | ||||
| Aged ≥19 years, n; mean (SD), kg/m2 | 126; 21.6 (3.3) | 36; 22.2 (3.2) | 90; 21.3 (3.3) | 0.16 |
| Aged <19 years, n; mean (SD) z-score | 59; 0.001 (0.87) | 25; 0.004 (0.82) | 34; −0.0009 (0.92) | 0.98 |
BMI Body mass index, IVA Ivacaftor, ppFEV1 Percent predicted forced expiratory volume in 1 s, SD Standard deviation, SOC Standard of care
aP values presented are for comparison of the G551D/IVA group with the F508del/SOC group
bPercentages are derived from the available sample
cn=207
dn=135
eBaseline data was missing for 2 patients
fn=208
gn=71