Table 1:
Patient Demographics by site
| Clinical Site | |||||
|---|---|---|---|---|---|
| Overall N=255 N (%) |
B N=75 N (%) |
C N=65 N (%) |
D N=115 N (%) |
p values1 | |
| Age(years) | |||||
| Mean | 21.9 | 29.1 | 16.1 | 20.5 | |
| Std Dev | 12.7 | 12.2 | 10.3 | 12.1 | |
| Median | 21.6 | 26.2 | 14.5 | 22.0 | |
| Minimum | 1.0 | 8.3 | 1.0 | 1.0 | |
| Maximum | 68.2 | 68.2 | 50.0 | 61.2 | |
| Age(years) | 255 | 75 | 65 | 115 | <.0001 |
| 0–<6 | 39 (15%) | 0 (0%) | 14 (22%) | 25 (22%) | |
| 6–<13 | 21 (8%) | 4 (5%) | 13 (20%) | 4 (3%) | |
| 13–<18 | 25 (10%) | 6 (8%) | 9 (14%) | 10 (9%) | |
| 18–<25 | 77 (30%) | 22 (29%) | 18 (28%) | 37 (32%) | |
| 25–<30 | 44 (17%) | 19 (25%) | 5 (8%) | 20 (17%) | |
| 30+ | 49 (19%) | 24 (32%) | 6 (9%) | 19 (17%) | |
| Gender | 255 | 75 | 65 | 115 | 0.13 |
| Female | 115 (45%) | 29 (39%) | 36 (55%) | 50 (43%) | |
| Male | 140 (55%) | 46 (61%) | 29 (45%) | 65 (57%) | |
| F508del mutation | 251 | 74 | 63 | 114 | 0.001 |
| Homozygote | 131 (52%) | 29 (39%) | 45 (71%) | 57 (50%) | |
| Heterozygote | 99 (39%) | 34 (46%) | 15 (24%) | 50 (44%) | |
| None | 21 (8%) | 11 (15%) | 3 (5%) | 7 (6%) | |
| Pancreatic status | 255 | 75 | 65 | 115 | 0.61 |
| PI | 223 (87%) | 64 (85%) | 59 (91%) | 100 (87%) | |
| PS | 32 (13%) | 11 (15%) | 6 (9%) | 15 (13%) | |
| BMI | 235 | 74 | 65 | 96 | 0.01 |
| undernourished | 26 (11%) | 3 (4%) | 9 (14%) | 14 (15%) | |
| acceptable | 118 (50%) | 34 (46%) | 37 (57%) | 47 (49%) | |
| well nourished | 91 (39%) | 37 (50%) | 19 (29%) | 35 (36%) | |
| FEV1 % predicted GLI | 212 | 74 | 51 | 87 | 0.02 |
| <41% | 42 (20%) | 10 (14%) | 5 (10%) | 27 (31%) | |
| 41–80% | 115 (54%) | 48 (65%) | 28 (55%) | 39 (45%) | |
| >80% | 55 (26%) | 16 (22%) | 18 (35%) | 21 (24%) | |
| Sample Type | 255 | 75 | 65 | 115 | <0.0001 |
| BAL | 55 (22%) | 0 (0%) | 24 (37%) | 31 (27%) | |
| Sputum | 200 (78%) | 75 (100%) | 41 (63%) | 84 (73%) | |
| Chronic antibiotics | 173 (68%) | 58 (77%) | 43 (66%) | 72 (63%) | 0.10 |
| Flucloxacillin | 18 (7%) | 8 (11%) | 1 (2%) | 9 (8%) | 0.09 |
| Azithromycin | 127 (50%) | 44 (59%) | 37 (57%) | 46 (40%) | 0.02 |
| Inhaled antibiotics2 | 130 (52%) | 42 (56%) | 30 (47%) | 58 (52%) | 0.58 |
| Any mucolytic | 205 (80%) | 59 (79%) | 54 (83%) | 92 (80%) | 0.79 |
| DNAse | 164 (64%) | 54 (72%) | 43 (66%) | 67 (58%) | 0.15 |
| Hypertonic saline | 109 (43%) | 15 (20%) | 37 (57%) | 57 (50%) | <0.0001 |
|
Inhaled corticosteroids |
105 (41%) | 31 (41%) | 36 (55%) | 38 (33%) | 0.004 |
| Antacid3 | 129 (51%) | 32 (43%) | 41 (63%) | 56 (49%) | 0.05 |
| Insulin | 34 (13%) | 7 (9%) | 8 (12%) | 19 (17%) | 0.35 |
B=Belfast, Northern Ireland; C=Chapel Hill, USA; D=Dublin, Ireland. PS=pancreatic sufficiency. PI=pancreatic insufficiency. BMI=body mass index. FEV1-forced expiratory volume in 1 second expressed as % predicted based on GLI=global lung initiative reference values[45]. BAL=bronchoalveolar lavage AZM=azithromycin. Patients were continuously enrolled at each site, not necessarily reflecting the study site population.
1
: Comparisons across sites using chi- square, Mantel-Haenszel mean score chi-square, or Fisher’s exact test.
2
: tobramycin, colistin and aztreonam.
3
: antacids, H2-blockers and proton pump inhibitors.