Demographics and clinical characteristics of the study cohort
| CF (no modulator) (CF.U) | CF.IVA | CF.LUM/IVA | Non-CF | P value* | |
| BAL analytes | |||||
| Total number | 54 | 11 | 5 | 23 | |
| Sex: male, n (%) | 23 (42.6) | 8 (72.7) | 2 (40) | 18 (78.2) | 0.012 |
| Age: years, median (range) | 2.0 (0.53–6.46) | 4.0 (1.45–5.95) | 4.5 (2.26–6.19) | 3.47 (0.48–12.3) | 0.013 |
| Ethnicity: European, n (%) | 47 (87) | 11 (100) | 5 (100) | 19 (82.6) | 0.631 |
| CFTR mutation: Phe508 del-homozygote, n (%) | 26 (48.1) | 0 (0) | 5 (100) | <0.0001 | |
| CFTR mutation: Phe509 del-heterozygote, n (%) | 23 (42.6) | 11 (100) | 0 (0) | <0.0001 | |
| Diagnosis by newborn screening, n (%) | 39 (72.2) | 8 (72.2) | 4 (80) | >0.999 | |
| Bronchiectasis at the time of sample, n (%) | 16 (29.6) | 1 (9.1) | 1 (20) | 0.485 | |
| Antibiotics (any) at the time of sample, n data available, n yes (%) | 38, 22 (57.9) | 9, 3 (33.3) | 1, 0 (0) | 0.201 | |
| Azithromycin at the time of sample, n data available, n yes (%) | 38, 4 (10.52) | 9, 0 (0) | 1, 0 (0) | 0.609 | |
| BAL flow cytometry | |||||
| Total number | 30 | 2 | 6 | 8 | |
| Sex: male, n (%) | 17 (56.7) | 2 (100) | 3 (50) | 5 (62.5) | 0.824 |
| Age: years, median (range) | 2.0 (0.95–6.46) | 5.16 (5.09–5.24) | 4.74 (2.26–6.19) | 3.03 (0.48–10.26) | 0.03 |
| Ethnicity: European, n (%) | 24 (80) | 2 (100) | 6 (100) | 8 (100) | 0.492 |
| CFTR mutation: Phe508 del-homozygote, n(%) | 7 (23.3) | 0 (0) | 6 (100) | 0.0006 | |
| CFTR mutation: Phe509 del-heterozygote, n(%) | 19 (63.3) | 2 (100) | 0 (0) | 0.002 | |
| Diagnosis by newborn screening, n (%) | 21 (70) | 2 (100) | 5 (83.3) | 0.814 | |
| Bronchiectasis at the time of sample, n (%) | 7 (23.3) | 0 (0) | 2 (33.3) | 0.784 | |
| Antibiotics (any) at the time of sample, n data available, n yes (%) | 11, 3 (27.2) | 1, 0 (0) | 2, 0 (0) | >0.999 | |
| Azithromycin at the time of sample, n data available, n yes (%) | 11, 1 (9.1) | 1, 0 (0) | 2, 0 (0) | >0.999 | |
| Plasma analytes | |||||
| Total number | 24 | 3 | 4 | 32 | |
| Sex: male, n (%) | 13 (54.2) | 2 (66.6) | 2 (50) | 20 (62.5) | 0.874 |
| Age: years, median (range) | 1.98 (0.95–6.46) | 4.96 (1.45–4.96) | 4.26 (2.26–6.19) | 3.4 (0.48–6.5) | 0.06 |
| Ethnicity: European, n (%) | 21 (87.5) | 3 (100) | 4 (100) | 25 (78.1) | 0.733 |
| CFTR mutation: Phe508 del-homozygote, n(%) | 6 (25) | 0 (0) | 4 (100) | 0.007 | |
| CFTR mutation: Phe509 del-heterozygote, n(%) | 15 (62.5) | 3 (100) | 0 (0) | 0.012 | |
| Diagnosis by newborn screening, n (%) | 18 (75) | 1 (33.3) | 2 (50) | 0.261 | |
| Bronchiectasis at the time of sample, n (%) | 6 (25) | 1 (33.3) | 2 (50) | 0.648 | |
| Antibiotics (any) at the time of sample, n data available, n yes (%) | 10, 2 (20) | 2, 2 (100) | 2, 0 (0) | 0.131 | |
| Azithromycin at the time of sample, n data available, n yes (%) | 10, 1 (10) | 2, 0 (0) | 2, 0 (0) | >0.999 | |
*Categorical variables were compared using Fisher’s exact tests and continuous variables were compared using Kruskal-Wallis tests.
BAL, bronchoalveolar lavage; CF, cystic fibrosis; CFTR, Cystic Fibrosis Transmembrane Conductance Regulator; IVA, ivacaftor; LUM, lumacaftor.