Table 1.
Demographics and Clinical Characteristics of the Patients at Baseline in Part B
| ELX/TEZ/IVA |
|||
|---|---|---|---|
| All Patients (N = 66)* | F/MF (N = 37) | F/F (N = 29) | |
| Sex, F, n (%) | 39 (59.1) | 22 (59.5) | 17 (58.6) |
| Age at baseline, mean (SD), yr | 9.3 (1.9) | 9.7 (1.8) | 8.8 (1.9) |
| Race, n (%)† | |||
| White | 58 (87.9) | 33 (89.2) | 25 (86.2) |
| Black or African American | 0 | 0 | 0 |
| Asian | 1 (1.5) | 1 (2.7) | 0 |
| American Indian or Alaska Native | 0 | 0 | 0 |
| Native Hawaiian or other Pacific Islander | 0 | 0 | 0 |
| Other | 0 | 0 | 0 |
| Not collected per local regulations | 8 (12.1) | 4 (10.8) | 4 (13.8) |
| Geographic region, n (%) | |||
| North America | 47 (71.2) | 27 (73.0) | 20 (69.0) |
| Europe and Australia | 19 (28.8) | 10 (27.0) | 9 (31.0) |
| Weight, mean (SD), kg | 30.0 (7.7) | 31.4 (7.9) | 28.2 (7.3) |
| Weight distribution, n (%) | |||
| <30 kg | 36 (54.5) | 20 (54.1) | 16 (55.2) |
| ⩾30 kg | 30 (45.5) | 17 (45.9) | 13 (44.8) |
| Weight-for-age z-score, mean (SD) | −0.22 (0.76) | −0.20 (0.87) | −0.23 (0.59) |
| Height, mean (SD), cm | 134.1 (12.3) | 136.9 (12.0) | 130.4 (11.9) |
| Height-for-age z-score, mean (SD) | −0.11 (0.98) | −0.01 (1.03) | −0.23 (0.91) |
| BMI, mean (SD), kg/m2 | 16.39 (1.69) | 16.50 (1.77) | 16.26 (1.61) |
| BMI-for-age z-score, mean (SD) | −0.16 (0.74) | −0.21 (0.84) | −0.10 (0.61) |
| ppFEV1, mean (SD)‡ | 88.8 (17.7) | 89.8 (17.5) | 87.3 (18.3) |
| ppFEV1 category, n (%) | |||
| <70 | 10 (15.2) | 5 (13.5) | 5 (17.2) |
| ⩾70–⩽90 | 22 (33.3) | 15 (40.5) | 7 (24.1) |
| >90 | 30 (45.5) | 17 (45.9) | 13 (44.8) |
| Missing data | 4 (6.1) | 0 | 4 (13.8) |
| Sweat chloride concentration, mean (SD), mmol/L§ | 102.2 (9.1) | 104.4 (7.2) | 99.3 (10.8) |
| CFQ-R respiratory domain score, mean (SD) points‖ | 80.3 (15.2) | 79.1 (17.3) | 81.8 (12.0) |
| LCI2.5, mean (SD), units¶ | 9.77 (2.68) | 9.34 (1.82) | 10.26 (3.36) |
Definition of abbreviations: BMI = body mass index; CFQ-R = Cystic Fibrosis Questionnaire–Revised; CFTR = cystic fibrosis transmembrane conductance regulator; ELX/TEZ/IVA = elexacaftor/tezacaftor/ivacaftor; F/F = homozygous for the F508del-CFTR mutation; F/MF = heterozygous for the F508del-CFTR mutation and a minimal function CFTR mutation; LCI2.5 = lung clearance index2.5; ppFEV1 = percentage of predicted FEV1.
Baseline was defined as the most recent nonmissing measurement before the first dose of study drug.
All patients in the full analysis set.
The race categories may sum to >100% because each patient was able to indicate more than one race.
n = 62 for all patients; n = 37 for F/MF; n = 25 for F/F.
n = 62 for all patients; n = 36 for F/MF; n = 26 for F/F.
n = 65 for all patients; n = 37 for F/MF; n = 28 for F/F. Scores on the CFQ-R range from 0 to 100, with higher scores indicating a higher patient-reported quality of life with regard to respiratory status.
n = 53 for all patients; n = 28 for F/MF; n = 25 for F/F.